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  • 1.
    Afram, G.
    et al.
    Karolinska Inst, Med, Stockholm, Sweden..
    Watz, E.
    ONK PAT, Ctr Apheresis, Stockholm, Sweden..
    Remberger, M.
    ONK PAT, Ctr Allogene Stem Cell Transplantat, Immunol, Stockholm, Sweden..
    Axdorph-Nygell, U.
    ONK PAT, Ctr Apheresis, Stockholm, Sweden..
    Sundin, M.
    Karolinska Inst, Pediat Haematol, Stockholm, Sweden..
    Hagglund, Hans
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Mattsson, J.
    Karolinska Inst, Ctr Stem Cell Transplantat, Stockholm, Sweden..
    Uhlin, M.
    Karolinska Inst, Ctr Stem Cell Transplantat, Stockholm, Sweden..
    Extracorporeal photopheresis as treatment for moderate-severe chronic graft-versus-host disease2016In: Bone Marrow Transplantation, ISSN 0268-3369, E-ISSN 1476-5365, Vol. 51, p. S138-S138Article in journal (Other academic)
  • 2.
    Afram, Gabriel
    et al.
    Karolinska Univ Hosp Huddinge, Dept Hematol, Stockholm, Sweden.
    Perez Simon, Jose Antonio
    Univ Seville, CSIC, Hosp Univ Virgen del Rocio, Dept Hematol,Inst Biomed Sevilla IBIS, Seville, Spain.
    Remberger, Mats
    Karolinska Univ Hosp Huddinge, Ctr Allogene Stem Cell Transplantat, Stockholm, Sweden.
    Caballero-Velazquez, Teresa
    Univ Seville, CSIC, Hosp Univ Virgen del Rocio, Dept Hematol,Inst Biomed Sevilla IBIS, Seville, Spain.
    Martino, Rodrigo
    Hosp Santa Creu & Sant Pau, Dept Hematol, Barcelona, Spain.
    Luis Pinana, Jose
    Hosp Santa Creu & Sant Pau, Dept Hematol, Barcelona, Spain;Hosp Clin Univ, Dept Hematol, Valencia, Spain.
    Ringden, Olle
    Karolinska Univ Hosp Huddinge, Ctr Allogene Stem Cell Transplantat, Stockholm, Sweden.
    Esquirol, Albert
    Hosp Santa Creu & Sant Pau, Dept Hematol, Barcelona, Spain.
    Lopez-Corral, Lucia
    Hosp Univ Salamanca IBSAL, Dept Hematol, Salamanca, Spain.
    Garcia, Irene
    Hosp Santa Creu & Sant Pau, Dept Hematol, Barcelona, Spain.
    Lopez-Godino, Oriana
    Hosp Univ Salamanca IBSAL, Dept Hematol, Salamanca, Spain.
    Sierra, Jordi
    Hosp Santa Creu & Sant Pau, Dept Hematol, Barcelona, Spain.
    Caballero, Dolores
    Hosp Univ Salamanca IBSAL, Dept Hematol, Salamanca, Spain.
    Ljungman, Per
    Vazquez, Lourdes
    Hosp Univ Salamanca IBSAL, Dept Hematol, Salamanca, Spain.
    Hägglund, Hans
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Reduced intensity conditioning increases risk of severe cGVHD: identification of risk factors for cGVHD in a multicenter setting2018In: Medical Oncology, ISSN 1357-0560, E-ISSN 1559-131X, Vol. 35, no 6, article id 79Article in journal (Refereed)
    Abstract [en]

    Chronic graft-versus-host disease (cGVHD) remains a major cause of morbidity and mortality after allogeneic hematopoietic stem cell transplantation (HSCT). Aim is to identify risk factors for the development of cGVHD in a multicenter setting. Patients transplanted between 2000 and 2006 were analyzed (n = 820). Donors were HLA-identical siblings (57%), matched unrelated donors (30%), and HLA-A, B or DR antigen mismatched (13%). Reduced intensity conditioning (RIC) was given to 65% of patients. Overall incidence of cGVHD was 46% for patients surviving more than 100 days after HSCT (n = 747). Older patient age [HR 1.15, p < 0.001], prior acute GVHD [1.30, p = 0.024], and RIC [1.36, p = 0.028] increased overall cGVHD. In addition, RIC [4.85, p < 0.001], prior aGVHD [2.14, p = 0.001] and female donor to male recipient [1.80, p = 0.008] increased the risk of severe cGVHD. ATG had a protective effect for both overall [0.41, p < 0.001] and severe cGVHD [0.20, p < 0.001]. Relapse-free survival (RFS) was impaired in patients with severe cGVHD. RIC, prior aGVHD, and female-to-male donation increase the risk of severe cGVHD. ATG reduces the risk of all grades of cGVHD without hampering RFS. GVHD prophylaxis may be tailored according to the risk profile of patients.

  • 3.
    Afram, Gabriel
    et al.
    Karolinska Univ Lab, Hematol Ctr, Stockholm, Sweden;Karolinska Inst, Div Hematol, Dept Med, Stockholm, Sweden.
    Watz, Emma
    Karolinska Univ Lab, Dept Clin Immunol & Transfus Med, Stockholm, Sweden;Karolinska Inst, Div Transplantat Surg, Dept Clin Sci Intervent & Technol, Stockholm, Sweden.
    Remberger, Mats
    Karolinska Univ Hosp, Ctr Allogene Stem Cell Transplantat, Stockholm, Sweden;Karolinska Inst, Dept Oncol Pathol, Stockholm, Sweden.
    Nygell, Ulla Axdorph
    Karolinska Univ Lab, Hematol Ctr, Stockholm, Sweden;Karolinska Univ Lab, Dept Clin Immunol & Transfus Med, Stockholm, Sweden;Karolinska Inst, Div Transplantat Surg, Dept Clin Sci Intervent & Technol, Stockholm, Sweden.
    Sundin, Mikael
    Karolinska Univ Hosp, Astrid Lindgren Childrens Hosp, Hematol Immunol SCT Sect, Stockholm, Sweden;Karolinska Inst, Div Pediat, Dept Clin Sci Intervent & Technol, Stockholm, Sweden.
    Hägglund, Hans
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Mattsson, Jonas
    Karolinska Univ Hosp, Ctr Allogene Stem Cell Transplantat, Stockholm, Sweden;Karolinska Inst, Dept Oncol Pathol, Stockholm, Sweden.
    Uhlin, Michael
    Karolinska Univ Lab, Dept Clin Immunol & Transfus Med, Stockholm, Sweden;Karolinska Inst, Div Transplantat Surg, Dept Clin Sci Intervent & Technol, Stockholm, Sweden.
    Higher response rates in patients with severe chronic skin graft-versus-host disease treated with extracorporeal photopheresis2019In: Central European Journal of Immunology, ISSN 1426-3912, E-ISSN 1644-4124, Vol. 44, no 1, p. 84-91Article in journal (Refereed)
    Abstract [en]

    Introduction: Different forms of graft-versus-host disease (GVHD) remain a major cause of morbidity and mortality after allogeneic hematopoietic stem cell transplantation (HSCT). The prognosis for steroid-refractory chronic GVHD (cGVHD) remains poor. Our aim was to evaluate extracorporeal photopheresis (ECP) treatment in cGVHD patients with different organ involvement to detect subgroups of patients with the best response.

    Material and methods: Thirty-four patients who underwent HSCT and developed moderate (n = 7) or severe (n = 27) steroid-refractory or steroid-dependent cGVHD treated with ECP were included in the analysis. A matched cGVHD control patient group untreated with ECP was collected for comparison.

    Results: Compared to the control group and the stable/progressive disease (SD/PD) patients, individuals with complete/partial remission have higher overall survival and lower transplant-related mortality. Furthermore, patients with complete and partial remission (CR/PR) had significantly higher levels of albumin and platelets after ECP treatment compared to patients with stable or progressive cGVHD (SD/PD). Corticosteroid treatment and other immunosuppressive agents could successfully be tapered in the CR/PR group compared to the SD/PD patients. In this study patients with skin cGVHD are those with the highest rate of CR/PR after ECP treatment.

    Conclusions: Our results suggest that ECP treatment is safe and effective for patients with predominantly skin, oral and liver cGVHD.

  • 4.
    Ajeganova, Sofia
    et al.
    Karolinska Inst, Dept Med Huddinge, S-14186 Stockholm, Sweden..
    Tesfa, Daniel
    Karolinska Inst, Ctr Hematol & Regenerat Med, Dept Med Huddinge, S-14186 Stockholm, Sweden.;Roche AB, Med Affairs, S-10074 Stockholm, Sweden..
    Hägglund, Hans
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Fadeel, Bengt
    Karolinska Inst, Inst Environm Med, Unit Mol Toxicol, S-17177 Stockholm, Sweden..
    Vedin, Inger
    Karolinska Inst, Ctr Hematol & Regenerat Med, Dept Med Huddinge, S-14186 Stockholm, Sweden..
    Zignego, Anna Linda
    Univ Florence, Ctr Syst Manifestat Hepatitis Viruses, Dept Internal Med, I-50134 Florence, Italy..
    Palmblad, Jan
    Karolinska Inst, Ctr Hematol & Regenerat Med, Dept Med Huddinge, S-14186 Stockholm, Sweden..
    Effect of FCGR polymorphism on the occurrence of late-onset neutropenia and flare-free survival in rheumatic patients treated with rituximab2017In: Arthritis Research & Therapy, ISSN 1478-6354, E-ISSN 1478-6362, Vol. 19, article id 44Article in journal (Refereed)
    Abstract [en]

    Background: The causes and mechanisms of late-onset neutropenia (LON) following rituximab treatment in patients with rheumatic diseases are not known. In this study, we aimed to investigate the role of established Fc gamma receptor gene (FCGR) polymorphisms and B-cell-activating factor (BAFF) gene promoter polymorphisms for the development of LON and for the efficacy of rituximab in patients with rheumatic diseases. Methods: A single-center case-control retrospective study was nested in a cohort of 214 consecutive patients with rheumatic diseases treated with rituximab. Eleven patients presented with LON. Fifty non-LON control subjects were matched by diagnosis, age, sex, and treatments. Single-nucleotide polymorphisms of FCGR (FCGR2A 131H/R, FCGR2B 232I/T, FCGR3A 158V/F) and BAFF promoter polymorphism -871C/T were analyzed with polymerase chain reaction-based techniques, and serum immunoglobulin M (IgM) and BAFF levels were analyzed by enzyme-linked immunosorbent assay. Flare-free survival was related to LON occurrence and polymorphisms. Results: The FCGR3A V allele, but not other FCGR polymorphisms, correlated with the occurrence of LON; each V allele conferred a fourfold increased OR for LON (p = 0.017). FCGR3A 158V/V and presentation with LON were associated with a longer flare-free survival (p = 0.023 and p = 0.031, respectively). FCGR3A 158V/V was related to lower IgM levels (p = 0.016). Serum BAFF levels showed no relationship with LON and BAFF -871C/T promoter polymorphism. There was a tendency toward longer flare-free survival in patients with the BAFF -871T/T allotype compared with the C/T or C/C allotypes (p = 0.096). Conclusions: The results of the present study suggest that presentation with LON may be a result of the intrinsic efficacy of rituximab in patients with rheumatic diseases. LON could indicate a longer biological and therapeutic activity of rituximab modulated by a certain genotypic polymorphism: the high-affinity FCGR3A V allele. This genotype and the occurrence of LON are both related to longer flare-free survival, suggestive of common mechanisms for LON and duration of response to rituximab. The role of the BAFF -871C/T promoter polymorphism in LON occurrence is unclear.

  • 5.
    Bergfelt, Emma
    et al.
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Kozlowski, Piotr
    Ahlberg, Lucia
    Hulegardh, Erik
    Hagglund, Hans
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Karlsson, Karin
    Markuszewska-Kuczymska, Alicja
    Tomaszewska-Toporska, Beata
    Smedmyr, Bengt
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Astrom, Maria
    Amini, Rose-Marie
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Immunology, Genetics and Pathology.
    Hallböök, Hélene
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Satisfactory outcome after intensive chemotherapy with pragmatic use of minimal residual disease (MRD) monitoring in older patients with Philadelphia-negative B cell precursor acute lymphoblastic leukaemia: a Swedish registry-based study2015In: Medical Oncology, ISSN 1357-0560, E-ISSN 1559-131X, Vol. 32, no 4, article id 135Article in journal (Refereed)
    Abstract [en]

    The introduction of minimal residual disease (MRD) monitoring, in the Swedish national guidelines for acute lymphoblastic leukaemia, was evaluated in 35 patients aged 46-79 years (median 61), who were diagnosed from 2007 to 2011 and treated with high-intensity, block-based chemotherapy (ABCDV/VABA induction). Both a high complete remission rate (91 %) and acceptable overall survival (OS) rate (47 %) at 5 years were achieved. MRD by flow cytometry was measured in 73 % of the patients reaching complete remission after the first course, but was omitted by the clinicians for eight patients who were either over 70 years of age or already met conventional high-risk criteria. Factors negatively influencing OS were age over 65 years and WHO status >= 2. MRD < 0.1 % after induction had positive impact on continuous complete remission but not on OS. Only five patients were allocated to allogeneic haematopoietic stem cell transplantation in first remission, mainly due to conventional high risk factors. Thus, use of intensive remission induction therapy is effective in a selection of older patients. In a population for whom the possibilities of treatment escalation are limited, the optimal role of MRD monitoring remains to be determined.

  • 6. Bjorklund, Andreas T.
    et al.
    Carlsten, Mattias
    Schaffer, Marie
    Liu, Lisa
    Cooley, Sarah A.
    Miller, Jeffrey S.
    Watz, Emma
    Palma, Marzia
    Hansson, Lotta
    Wahlin, Bjorn E.
    Mollgard, Lars
    Hägglund, Hans
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Blomberg, Pontus
    Ljungman, Per T.
    Hellstrom-Lindberg, Eva
    Ljunggren, Hans-Gustaf
    Malmberg, Karl-Johan
    Early and Transient Microchimerism Associated with Complete Remission after Adoptively Transferred Haploidentical NK Cells Against High Risk Myelodysplastic Syndrome and Refractory Acute Myeloid Leukemia2014In: Blood, ISSN 0006-4971, E-ISSN 1528-0020, Vol. 124, no 21Article in journal (Other academic)
  • 7. Broesby-Olsen, Sigurd
    et al.
    Dybedal, Ingunn
    Gülen, Theo
    Kielsgaard Kristensen, Thomas
    Boe Møller, Michael
    Ackermann, Leena
    Sääf, Maria
    Karlsson, Maria A
    Agertoft, Lone
    Brixen, Kim
    Hermann, Pernille
    Stylianou, Eva
    Mortz, Charlotte G
    Torfing, Trine
    Havelund, Troels
    Sander, Birgitta
    Bergström, Anna
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Dermatology and Venereology.
    Bendix, Marie
    Garvey, Lene H
    Weis Bjerrum, Ole
    Valent, Peter
    Bindslev-Jensen, Carsten
    Nilsson, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Vestergaard, Hanne
    Hägglund, Hans
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Multidisciplinary Management of Mastocytosis: Nordic Expert Group Consensus2016In: Acta Dermato-Venereologica, ISSN 0001-5555, E-ISSN 1651-2057, Vol. 96, no 5Article in journal (Refereed)
    Abstract [en]

    Mastocytosis is a heterogeneous group of diseases defined by an increased number and accumulation of mast cells, and often also by signs and symptoms of mast cell activation. Disease subtypes range from indolent to rare aggressive forms. Mastocytosis affects people of all ages and has been considered rare; however, it is probably underdiagnosed with potential severe implications. Diagnosis can be challenging and symptoms may be complex and involve multiple organ-systems. In general it is advised that patients should be referred to centres with experience in the disease offering an individualized, multidisciplinary approach. We present here consensus recommendations from a Nordic expert group for the diagnosis and general management of patients with mastocytosis.

  • 8.
    Burman, Joachim
    et al.
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Neuroscience, Neurology.
    Iacobaeus, Ellen
    Svenningsson, Anders
    Lycke, Jan
    Gunnarsson, Martin
    Nilsson, Petra
    Vrethem, Magnus
    Fredrikson, Sten
    Martin, Claes
    Sandstedt, Anna
    Uggla, Bertil
    Lenhoff, Stig
    Johansson, Jan-Erik
    Isaksson, Cecilia
    Hägglund, Hans
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Carlson, Kristina
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Fagius, Jan
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Neuroscience, Neurology.
    Autologous haematopoietic stem cell transplantation for aggressive multiple sclerosis: the Swedish experience2014In: Journal of Neurology, Neurosurgery and Psychiatry, ISSN 0022-3050, E-ISSN 1468-330X, Vol. 85, no 10, p. 1116-1121Article in journal (Refereed)
    Abstract [en]

    BACKGROUND: Autologous haematopoietic stem cell transplantation (HSCT) is a viable option for treatment of aggressive multiple sclerosis (MS). No randomised controlled trial has been performed, and thus, experiences from systematic and sustained follow-up of treated patients constitute important information about safety and efficacy. In this observational study, we describe the characteristics and outcome of the Swedish patients treated with HSCT for MS.

    METHODS: Neurologists from the major hospitals in Sweden filled out a follow-up form with prospectively collected data. Fifty-two patients were identified in total; 48 were included in the study and evaluated for safety and side effects; 41 patients had at least 1 year of follow-up and were further analysed for clinical and radiological outcome. In this cohort, 34 patients (83%) had relapsing-remitting MS, and mean follow-up time was 47 months.

    RESULTS: At 5 years, relapse-free survival was 87%; MRI event-free survival 85%; expanded disability status scale (EDSS) score progression-free survival 77%; and disease-free survival (no relapses, no new MRI lesions and no EDSS progression) 68%. Presence of gadolinium-enhancing lesions prior to HSCT was associated with a favourable outcome (disease-free survival 79% vs 46%, p=0.028). There was no mortality. The most common long-term side effects were herpes zoster reactivation (15%) and thyroid disease (8.4%).

    CONCLUSIONS: HSCT is a very effective treatment of inflammatory active MS and can be performed with a high degree of safety at experienced centres.

  • 9.
    Burman, Joachim
    et al.
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Neuroscience, Neurology.
    Tolf, Andreas
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Neuroscience, Neurology.
    Hägglund, Hans
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Askmark, Håkan
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Neuroscience, Neurology.
    Autologous haematopoietic stem cell transplantation for neurological diseases2018In: Journal of Neurology, Neurosurgery and Psychiatry, ISSN 0022-3050, E-ISSN 1468-330X, Vol. 89, no 2, p. 147-155Article, review/survey (Refereed)
    Abstract [en]

    Neuroinflammatory diseases such as multiple sclerosis, neuromyelitis optica, chronic inflammatory demyelinating polyneuropathy and myasthenia gravis are leading causes of physical disability in people of working age. In the last decades significant therapeutic advances have been made that can ameliorate the disease course. Nevertheless, many affected will continue to deteriorate despite treatment, and the costs associated with disease-modifying drugs constitute a significant fiscal burden on healthcare in developed countries. Autologous haematopoietic stem cell transplantation is a treatment approach that aims to ameliorate and to terminate disease activity. The erroneous immune system is eradicated using cytotoxic drugs, and with the aid of haematopoietic stem cells a new immune system is rebuilt. As of today, more than 1000 patients with multiple sclerosis have been treated with this procedure. Available data suggest that autologous haematopoietic stem cell transplantation is superior to conventional treatment in terms of efficacy with an acceptable safety profile. A smaller number of patients with other neuroinflammatory conditions have been treated with promising results. Herein, current data on clinical effect and safety of autologous haematopoietic stem cell transplantation for neurological disease are reviewed.

  • 10.
    Dahlin, J. S.
    et al.
    Karolinska Inst, Karolinska Univ Hosp, Dept Med, Stockholm, Sweden..
    Ungerstedt, J. S.
    Karolinska Inst, Dept Med Huddinge, Stockholm, Sweden.;Karolinska Univ Hosp, Hematol Ctr, Stockholm, Sweden..
    Grootens, J.
    Karolinska Inst, Karolinska Univ Hosp, Dept Med, Stockholm, Sweden..
    Sander, B.
    Karolinska Inst, Karolinska Univ Hosp, Div Pathol, Dept Lab Med, Stockholm, Sweden..
    Guelen, T.
    Karolinska Univ, Huddinge Hosp, Dept Resp Dis & Allergy, Stockholm, Sweden..
    Hägglund, Hans
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology. Univ Uppsala Hosp, Sect Hematol, Uppsala, Sweden..
    Nilsson, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology. Karolinska Inst, Karolinska Univ Hosp, Dept Med, Stockholm, Sweden..
    Detection of circulating mast cells in advanced systemic mastocytosis2016In: Leukemia, ISSN 0887-6924, E-ISSN 1476-5551, Vol. 30, no 9, p. 1954-+Article in journal (Refereed)
  • 11. Greco, Raffaella
    et al.
    Bondanza, Attilio
    Oliveira, Maria Carolina
    Badoglio, Manuela
    Burman, Joachim
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Neuroscience, Neurology.
    Piehl, Fredrik
    Hägglund, Hans
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Krasulova, Eva
    Simões, Belinda Pinto
    Carlson, Kristina
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Pohlreich, David
    Labopin, Myriam
    Saccardi, Riccardo
    Comi, Giancarlo
    Mancardi, Gian Luigi
    Bacigalupo, Andrea
    Ciceri, Fabio
    Farge, Dominique
    Autologous hematopoietic stem cell transplantation in neuromyelitis optica: A registry study of the EBMT Autoimmune Diseases Working Party2015In: Multiple Sclerosis, ISSN 1352-4585, E-ISSN 1477-0970, Vol. 21, no 2, p. 189-197Article in journal (Refereed)
    Abstract [en]

    BACKGROUND:

    Neuromyelitis optica (NMO) is an inflammatory autoimmune disorder of the central nervous system, hallmarked by pathogenic anti-aquaporin 4 antibodies. NMO prognosis is worse compared with multiple sclerosis.

    OBJECTIVE:

    The European Group for Blood and Marrow Transplantation (EBMT) Autoimmune Diseases Working Party (ADWP) conducted a retrospective survey to analyze disease outcome following autologous stem cell transplantation (ASCT).

    METHODS:

    This retrospective multicenter study assessed the efficacy and safety of ASCT in 16 patients suffering from refractory NMO reported to the EBMT registry between 2001 and 2011.

    RESULTS:

    Fifteen patients were successfully mobilized with cyclophosphamide (Cy) and G-CSF, one with G-CSF alone. All patients received an unmanipulated autologous peripheral blood stem cell graft, after conditioning with BEAM plus anti-thymocyte globulin (ATG, n = 9 patients), thiotepa-Cy (n = 3) or Cy (200 mg/kg) plus ATG (n = 4). After a median follow-up of 47 months, three of 16 cases were progression and treatment free, while in the remaining 13 patients further treatments were administered for disability progression or relapse after ASCT. Altogether, relapse-free survival at three and five years was 31% and 10%, respectively, while progression-free survival remained 48% at three and five years.

    CONCLUSIONS:

    In these NMO patients, highly resistant to conventional treatment, ASCT allows for temporary control of the disease, despite a tendency to progress or relapse in the long term.

  • 12. Gülen, T
    et al.
    Hägglund, Hans
    astocytosis Centre Karolinska, Karolinska University Hospital, Karolinska Institutet, Stockholm, Sweden.
    Dahlén, B
    Nilsson, G
    High prevalence of anaphylaxis in patients with systemic mastocytosis: a single-centre experience2014In: Clinical and Experimental Allergy, ISSN 0954-7894, E-ISSN 1365-2222, Vol. 44, no 1, p. 121-129Article in journal (Refereed)
    Abstract [en]

    BACKGROUND: Systemic mastocytosis (SM) is a clonal mast cells disorder characterized by the proliferation, accumulation and activation of mast cells in extracutaneous tissues. The clinical picture is heterogeneous and may range from asymptomatic to potentially fatal anaphylactic reactions due to excessive mast cell mediator release.

    OBJECTIVE: The aim of this study was to investigate the prevalence and trigger factors of anaphylactic reactions among adult SM patients. We also explored the clinical spectrum of mast cell mediator-related symptoms in patients with SM.

    METHODS: This descriptive study was performed among 84 consecutive adult (≥ 18 years) patients those were diagnosed with SM according to WHO criteria. Sixty-six of the patients also underwent a comprehensive allergy work-up.

    RESULTS: Sixty of 84 patients with SM (71%) had bone marrow mast cell aggregates and fulfilled the major criteria for SM and 76 patients (91%) had indolent disease. Simultaneous occurrence of cutaneous mastocytosis was observed in 59 patients (70%). Thirty-six patients (43%) had had at least one episode of an anaphylactic reaction. The clinical courses of the reactions were usually severe and patients often presented with syncope attacks (72%). Most patients reacted after hymenoptera venom stings (19/36; 53%). In 39% (14/36), a clear aetiology could not be determined. While males and females were equally frequent among the patients with SM, anaphylaxis patients showed a male predominance (61%). Anaphylactic reactions occurred more frequently in patients without cutaneous engagement. The rate of allergy sensitization was significantly higher in SM patients with anaphylaxis as compared with non-anaphylaxis SM patients, 70% vs. 23%, respectively (P = 0.0002).

    CONCLUSIONS AND CLINICAL RELEVANCE: Anaphylaxis is more prevalent in patients with SM, predominantly in patients with atopic SM. Hymenoptera venom-induced and idiopathic anaphylaxis were the most frequent elicitors. Our findings implicate that all mastocytosis patients with anaphylaxis should undergo detailed allergological assessment before considering treatment and preventive measures.

  • 13. Gülen, T
    et al.
    Hägglund, Hans
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Dahlén, B
    Nilsson, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Mastocytosis: the puzzling clinical spectrum and challenging diagnostic aspects of an enigmatic disease2016In: Journal of Internal Medicine, ISSN 0954-6820, E-ISSN 1365-2796, Vol. 279, no 3, p. 211-228Article, review/survey (Refereed)
    Abstract [en]

    Mastocytosis is a complex disorder characterized by the accumulation of abnormal mast cells (MC) in the skin, bone marrow and/or other visceral organs. The clinical manifestations result from MC-derived mediators and, less frequently, from destructive infiltration of MCs. Patients suffer from a variety of symptoms including pruritus, flushing and life-threatening anaphylaxis. Whilst mastocytosis is likely to be suspected in a patient with typical skin lesions [i.e. urticaria pigmentosa (UP)], the absence of cutaneous signs does not rule out the diagnosis of this disease. Mastocytosis should be suspected in cases of recurrent, unexplained or severe insect-induced anaphylaxis or symptoms of MC degranulation without true allergy. In rare cases, unexplained osteoporosis or unexplained haematological abnormalities can be underlying feature of mastocytosis, particularly when these conditions are associated with elevated baseline serum tryptase levels. The diagnosis is based on the World Health Organization criteria, in which the tryptase level, histopathological and immunophenotypic evaluation of MCs and molecular analysis are crucial. A somatic KIT mutation, the most common of which is D816V, is usually detectable in MCs and their progenitors. Once a diagnosis of systemic mastocytosis (SM) is made, it is mandatory to assess the burden of the disease, its activity, subtype and prognosis, and the appropriate therapy. Mastocytosis comprises seven different categories that range from indolent forms, such as cutaneous and indolent SM, to progressive forms, such as aggressive SM and MC leukaemia. Although prognosis is good in patients with indolent forms of the disease, patients with advanced categories have a poor prognosis.

  • 14. Gülen, T
    et al.
    Hägglund, Hans
    Mastocytosis Centre Karolinska, Karolinska University Hospital and Karolinska Institutet, Stockholm.
    Sander, B
    Dahlén, B
    Nilsson, Gunnar
    Department of Medicine Solna, Clinical Immunology and Allergy Research Unit, Karolinska Institutet, Stockholm.
    The presence of mast cell clonality in patients with unexplained anaphylaxis2014In: Clinical and Experimental Allergy, ISSN 0954-7894, E-ISSN 1365-2222, Vol. 44, no 9, p. 1179-1187Article in journal (Refereed)
    Abstract [en]

    BACKGROUND: The mechanisms by which mast cells in patients with unexplained anaphylaxis (UEA) are triggered remain elusive. Onset of episodes is unpredictable and often recurrent. The substantial overlap between the clinical manifestations of UEA and clonal mast cell disorders (CMD) suggests an association between these rare disorders. The two forms of CMD characterized to date are systemic mastocytosis (SM) and monoclonal mast cell activation syndrome (MMAS).

    OBJECTIVE: To examine the hypothesis that the pathogenesis of UEA reflects the presence of aberrant subpopulations of mast cells.

    METHODS: Thirty (14 men, 16 women) patients (≥ 18 years) suffering from UEA and with no signs of cutaneous mastocytosis were recruited. Patients underwent an initial complete allergy work-up to confirm the diagnosis of UEA. Level of baseline serum tryptase (sBT) and total IgE were determined. In addition, a bone marrow examination was performed on all 30 patients to investigate possible underlying CMD.

    RESULTS: Fourteen (47%) of our cases (nine men, five women) were diagnosed with CMD: 10 with SM and four with MMAS. Four of the 10 patients with SM had mast cell aggregates in their bone marrow. All patients with SM exhibited a sBT level > 11.4 ng/mL, whereas this level was elevated in only two of those with MMAS and four with UAE but not diagnosed with CMD. Total IgE levels were lower in the group of patients with CMD (P < 0.03).

    CONCLUSION AND CLINICAL RELEVANCE: The pathogenic mechanism underlying UEA could be explained by the presence of immunophenotypically aberrant mast cells with clonal markers in 47% of our subjects, indicating that clonal mast cell disorders are present in a substantial subset of these patients. Thus, the presence of CMD should be considered in patients with UEA if they have an elevated level of sBT (≥ 11.4 ng/mL) and cardiovascular symptoms such as syncope.

  • 15. Gülen, Theo
    et al.
    Hägglund, Hans
    Department of Hematology, Karolinska University Hospital Huddinge, Stockholm, Sweden.
    Dahlén, Sven-Erik
    Sander, Birgitta
    Dahlén, Barbro
    Nilsson, Gunnar
    Clinical Immunology and Allergy Research Unit, Department of Medicine Solna, Karolinska Institutet, Stockholm, Sweden.
    Flushing, fatigue, and recurrent anaphylaxis: a delayed diagnosis of mastocytosis2014In: The Lancet, ISSN 0140-6736, E-ISSN 1474-547X, Vol. 383, no 9928, p. 1608-Article in journal (Refereed)
  • 16. Hartmann, Karin
    et al.
    Escribano, Luis
    Grattan, Clive
    Brockow, Knut
    Carter, Melody C
    Alvarez-Twose, Ivan
    Matito, Almudena
    Broesby-Olsen, Sigurd
    Siebenhaar, Frank
    Lange, Magdalena
    Niedoszytko, Marek
    Castells, Mariana
    Oude Elberink, Joanna N G
    Bonadonna, Patrizia
    Zanotti, Roberta
    Hornick, Jason L
    Torrelo, Antonio
    Grabbe, Jürgen
    Rabenhorst, Anja
    Nedoszytko, Boguslaw
    Butterfield, Joseph H
    Gotlib, Jason
    Reiter, Andreas
    Radia, Deepti
    Hermine, Olivier
    Sotlar, Karl
    George, Tracy I
    Kristensen, Thomas K
    Kluin-Nelemans, Hanneke C
    Yavuz, Selim
    Hägglund, Hans
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Sperr, Wolfgang R
    Schwartz, Lawrence B
    Triggiani, Massimo
    Maurer, Marcus
    Nilsson, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Horny, Hans-Peter
    Arock, Michel
    Orfao, Alberto
    Metcalfe, Dean D
    Akin, Cem
    Valent, Peter
    Cutaneous manifestations in patients with mastocytosis: Consensus report of the European Competence Network on Mastocytosis; the American Academy of Allergy, Asthma & Immunology; and the European Academy of Allergology and Clinical Immunology.2015In: Journal of Allergy and Clinical Immunology, ISSN 0091-6749, E-ISSN 1097-6825Article in journal (Refereed)
    Abstract [en]

    Cutaneous lesions in patients with mastocytosis are highly heterogeneous and encompass localized and disseminated forms. Although a classification and criteria for cutaneous mastocytosis (CM) have been proposed, there remains a need to better define subforms of cutaneous manifestations in patients with mastocytosis. To address this unmet need, an international task force involving experts from different organizations (including the European Competence Network on Mastocytosis; the American Academy of Allergy, Asthma & Immunology; and the European Academy of Allergology and Clinical Immunology) met several times between 2010 and 2014 to discuss the classification and criteria for diagnosis of cutaneous manifestations in patients with mastocytosis. This article provides the major outcomes of these meetings and a proposal for a revised definition and criteria. In particular, we recommend that the typical maculopapular cutaneous lesions (urticaria pigmentosa) should be subdivided into 2 variants, namely a monomorphic variant with small maculopapular lesions, which is typically seen in adult patients, and a polymorphic variant with larger lesions of variable size and shape, which is typically seen in pediatric patients. Clinical observations suggest that the monomorphic variant, if it develops in children, often persists into adulthood, whereas the polymorphic variant may resolve around puberty. This delineation might have important prognostic implications, and its implementation in diagnostic algorithms and future mastocytosis classifications is recommended. Refinements are also suggested for the diagnostic criteria of CM, removal of telangiectasia macularis eruptiva perstans from the current classification of CM, and removal of the adjunct solitary from the term solitary mastocytoma.

  • 17. Hulegardh, E.
    et al.
    Hägglund, Hans
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Ahlberg, L.
    Karlsson, K.
    Karbach, H.
    Markuszewska, A.
    Persson, Inger
    Uppsala University, Disciplinary Domain of Humanities and Social Sciences, Faculty of Social Sciences, Department of Statistics.
    Astrom, M.
    Hallböök, Helene
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Outcome after HSCT in Philadelphia chromosome positive acute lymphoblastic leukemia in Sweden: a population-based study2014In: Medical Oncology, ISSN 1357-0560, E-ISSN 1559-131X, Vol. 31, no 8, p. 66-Article in journal (Refereed)
    Abstract [en]

    Even in the tyrosine kinase inhibitor era, allogeneic hematopoietic stem cell transplantation (HSCT) is regarded as standard care for adult Philadelphia (Ph) positive acute lymphoblastic leukemia (ALL). In this retrospective national study, we have reviewed the outcome after HSCT in Sweden for adult Ph-positive ALL between 2000 and 2009. In total, 51 patients with median age 42 (range 20-66) years underwent HSCT. Mainly allogeneic HSCT was performed (24 related donor, 24 unrelated donor and one cord blood), and only two patients were treated with an autologous HSCT. The 5-year OS was 51 (37-64) %. The probabilities of morphological relapse and non-relapse mortality (NRM) at 5 years were 36 (23-49) and 18 (9-29) %, respectively. For the allogeneic transplanted, the 5-year OS was for patients <40 years 70 (50-90) % and for patients >= 40 years 34 (16-52) %, p = 0.002. The 5-year probability of NRM was for patients <40 years 10 (2-28) % compared to 25 (11-42) % for patients >= 40 years (p = 0.04). Patients with chronic graft-versus-host disease (GVHD) had a 5-year morphological relapse probability of 20 (6-40) % compared to 59 (35-77) % for patients without chronic GVHD (p = 0.03). Age >= 40 years and the absence of chronic GVHD were confirmed as independent negative prognostic factors for relapse and non-relapse mortality in a multivariate analysis although the impact of chronic GVHD was significant only in the older age cohort.

  • 18.
    Hägglund, Hans
    et al.
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Remberger, M.
    Ringden, O.
    Twenty-year follow-up of a randomized trial comparing intraosseous and i.v. BM transplantation2014In: Bone Marrow Transplantation, ISSN 0268-3369, E-ISSN 1476-5365, Vol. 49, no 12, p. 1541-1542Article in journal (Refereed)
  • 19.
    Hägglund, Hans
    et al.
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Sander, B.
    Ahmadi, A.
    Gulen, T.
    Nilsson, Gunnar
    Analysis of V600E BRAF and D816V KIT mutations in systemic mastocytosis2014In: Medical Oncology, ISSN 1357-0560, E-ISSN 1559-131X, Vol. 31, no 8, p. 123-Article in journal (Refereed)
    Abstract [en]

    Most patients with systemic mastocytosis (SM) carry a D816 V KIT mutation causing a ligand-independent activation of the receptor. Down-stream of KIT is several components known to be mutated in different malignancies. RAF is among the most frequently mutated kinases, where BRAF V600E mutation occurs in most hairy cell leukemias (HCL) and half of malignant melanomas. We investigated BRAF mutations in 36 subjects with different forms of SM, but could not detect BRAF mutation in any of the cases, not even in the mast cell lineage of a patient with V600E BRAF-positive HCL. Thus, although BRAF is commonly mutated it appears not to be present in SM.

  • 20.
    Hägglund, Hans
    et al.
    Department of Hematology, Karolinska University Hospital Huddinge.
    Sander, Birgitta
    Gülen, Theo
    Lindelöf, Bernt
    Nilsson, Gunnar
    Clinical Immunology and Allergy Unit, Department of Medicine, Karolinska Institutet and Karolinska University Hospital Solna, SE-171 76 Stockholm, and Mastocytosis Center Karolinska, Karolinska University Hospital and Karolinska Institutet, Stockholm, Sweden.
    Increased risk of malignant melanoma in patients with systemic mastocytosis?2014In: Acta Dermato-Venereologica, ISSN 0001-5555, E-ISSN 1651-2057, Vol. 94, no 5, p. 583-584Article in journal (Refereed)
    Abstract [en]

    Mastocytosis, a group of rare disorders that occur in bothchildren and adults, is characterised by abnormal growthand pathological accumulation of mast cells in one or moreorgans, most commonly the skin (1). Urticaria pigmentosa(UP) is the most common cutaneous variant. In cases ofextracutaneous involvement, systemic mastocytosis (SM)can be diagnosed on the basis of the criteria formulatedby the WHO. The course of SM in most patients (90%) isindolent, with more aggressive presentation in only a few.The incidence of cutaneous melanoma is increasingand although this malignancy and mastocytosis originatefrom 2 different types of cells (melanocytes from theneural crest and mast cells from haematopoetic stem cells,respectively) they share certain similarities, includingexpression of the transcription factors MITF and STAT3,and dependence of the growth factor receptor KIT and itsligand stem cell factor for their growth and development(2, 3). We have found 5 published case reports that suggesta relationship between these 2 pathologies. In the first,published in 1979, a patient with nodular mastocytosis de-veloped both melanocytoma and mastocytoma (4). In thesecond, UP and SM preceded a metastatic melanoma (5)and the third involved combined mastocytoma-junctionalnaevus (6). In the fourth case, malignant melanoma wasdiagnosed prior to SM (7). And finally, a patient withtelangiectasia macularis eruptive perstans (TEMP), arare form of cutaneous mastocytosis, was found to havea malignant melanoma (8).Here, we describe our 4 additional cases and discusspossible associations between these 2 diseases.

  • 21. Håkansson, Irene
    et al.
    Sandstedt, Anna
    Lundin, Fredrik
    Askmark, Håkan
    Pirskanen, Ritva
    Carlson, Kristina
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Piehl, Fredrik
    Hägglund, Hans
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Successful autologous haematopoietic stem cell transplantation for refractory myasthenia gravis - a case report2017In: Neuromuscular Disorders, ISSN 0960-8966, E-ISSN 1873-2364, Vol. 27, no 1, p. 90-93Article in journal (Refereed)
    Abstract [en]

    Myasthenia gravis (MG) is an autoimmune disease, with immune reactivity against the post-synaptic endplate of the neuromuscular junction. Apart from symptomatic treatment with choline esterase blockers, many patients also require immunomodulatory treatment. Despite existing treatment options, some patients are treatment refractory. We describe a patient with severe MG refractory to corticosteroids, four oral immunosuppressants, cyclophosphamide, rituximab and bortezomib who was treated with autologous haematopoietic stem cell transplantation. Two years after this, the patient has significantly improved in objective tests and in quality of life and leads an active life. Diplopia is her only remaining symptom and she is completely free of medication for MG. We believe that autologous haematopoietic stem cell transplantation can be an effective therapeutic option for carefully selected cases of severe, treatment refractory MG.

  • 22. Kozlowski, Piotr
    et al.
    Astrom, Maria
    Ahlberg, Lucia
    Bernell, Per
    Hulegardh, Erik
    Hägglund, Hans
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Karlsson, Karin
    Markuszewska-Kuczymska, Alicja
    Tomaszewska-Toporska, Beata
    Smedmyr, Bengt
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Amini, Rose-Marie
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Immunology, Genetics and Pathology, Molecular and Morphological Pathology.
    Hallböök, Helene
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    High relapse rate of T cell acute lymphoblastic leukemia in adults treated with Hyper-CVAD chemotherapy in Sweden2014In: European Journal of Haematology, ISSN 0902-4441, E-ISSN 1600-0609, Vol. 92, no 5, p. 377-381Article in journal (Refereed)
    Abstract [en]

    Background Hyper-CVAD is widely used to treat acute lymphoblastic leukemia (ALL) and aggressive lymphomas. This multicenter, population-based study assessed the efficacy of Hyper-CVAD as first-line therapy in patients with T-cell ALL (T-ALL). Patients and methods Between October 2002 and September 2006, 24 patients were diagnosed with T-ALL in Sweden; 19 were eligible for treatment with the protocol. Results The median age was 32yr (range 18-72yr). Complete remission (CR) was obtained in 17 of 19 (89%) patients, and the treatment was relatively well tolerated. Allogeneic stem cell transplantation (SCT) was recommended in high-risk disease and was performed in four patients upfront. Two- and 5-yr leukemia-free survivals (LFS) in 17 patients with CR achievement were identical, at 29% (95% confidence interval [CI]: 8-51). Two- and 5-yr overall survival (OS) in whole cohort was 63% (95% CI: 42-85) and 47% (95% CI: 26-69), respectively. The 5-yr LFS for 15 patients who did not receive allogeneic SCT upfront were 20% (95% CI: 0-40), although 14 of 15 completed the protocol (eight cycles). Relapse occurred in 2 of 4 upfront-transplanted patients and in 12 of 15 patients treated with chemotherapy alone, six of whom received allogeneic SCT in CR2. Age >= 35yr influenced OS negatively in univariate analysis (HR 5.1, 95% CI: 1.55-16.7). Conclusions Hyper-CVAD treatment resulted in a high CR rate and appeared safe, but it showed poor efficacy at preventing relapse. Therefore, this treatment is no longer recommended for adults with T-ALL in Sweden.

  • 23. Kozlowski, Piotr
    et al.
    Astrom, Maria
    Ahlberg, Lucia
    Bernell, Per
    Hulegardh, Erik
    Hägglund, Hans
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Karlsson, Karin
    Markuszewska-Kuczymska, Alicja
    Tomaszewska-Toporska, Beata
    Smedmyr, Bengt
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Hallböök, Helene
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    High curability via intensive reinduction chemotherapy and stem cell transplantation in young adults with relapsed acute lymphoblastic leukemia in Sweden 2003-20072012In: Haematologica, ISSN 0390-6078, E-ISSN 1592-8721, Vol. 97, no 9, p. 1414-1421Article in journal (Refereed)
    Abstract [en]

    Background A minority of patients with adult acute lymphoblastic leukemia who relapse are rescued. The aim of this population-based study was to assess the results of reinduction treatment and allogeneic stem cell transplantation in patients in second complete remission.

    Design and Methods Between 2003-2007, 76 adults (<66 years) with relapsed acute lymphoblastic leukemia (Burkitt's leukemia excluded) were prospectively reported to The Swedish Adult Acute Leukemia Registry and later evaluated.

    Results Reinduction with: (i) mitoxantrone, etoposide, and cytarabine (MEA); (ii) fludarabine, cytarabine, pegylated-asparaginase plus granulocyte colony-stimulating factor (FLAG-Asp); and (iii) cytarabine, betamethasone, cyclophosphamide, daunorubicin, and vincristine (ABCDV) resulted in complete remission in 6/9 (67%), 10/16 (63%) and 9/21 (43%) of the patients, respectively. Allogeneic stem cell transplantation was performed during second complete remission in 29 patients. Multivariate analysis regarding overall survival after relapse revealed that age over 35 years at diagnosis and relapse within 18 months were negative prognostic factors. Overall survival rates at 3 and 5 years were 22% (95% CI: 13-32) and 15% (95% CI: 7-24). Of 19 patients less than 35 years at diagnosis who underwent allogeneic stem cell transplantation in second remission, ten (53%) are still alive at a median of 5.5 years (range, 4.2-8.3) after relapse, whereas all patients over 35 years old at diagnosis have died.

    Conclusions Allogeneic stem cell transplantation remains the treatment of choice for young adults with relapsed acute lymphoblastic leukemia. Both (i) mitoxantrone, etoposide, and cytarabine and (ii) fludarabine, cytarabine, pegylated-asparaginase plus granulocyte colony-stimulating factor seem effective as reinduction treatments and should be further evaluated. New salvage strategies are needed, especially for patients over 35 years old at diagnosis.

  • 24.
    Källström, Miikka
    et al.
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences.
    Soveri, Inga
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Renal Medicine.
    Oldgren, Jonas
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Medicinska och farmaceutiska vetenskapsområdet, centrumbildningar mm, UCR-Uppsala Clinical Research Center. Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Cardiology.
    Laukkanen, Jari
    Faculty of Sport and Health Sciences and Central Finland Health Care District, Department of Internal Medicine, University of Jyväskylä, Jyväskylä, Finland; Institute of Public Health and Clinical Nutrition, University of Eastern Finland, Kuopio, Finland.
    Ichiki, Tomoko
    Cardiovascular Medicine, Mayo Clinic and Cardiology, International University of Health and Welfare, Rochester, Minnesota; Cardiology, International University of Health and Welfare, Narita, Japan.
    Tei, Chuwa
    Waon Therapy Research Institute, Tokyo, Japan.
    Timmerman, Mark
    Department of Family Medicine, River Valley Clinic, Spring Green, Wisconsin.
    Berglund, Lars
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Medicinska och farmaceutiska vetenskapsområdet, centrumbildningar mm, UCR-Uppsala Clinical Research Center. Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Public Health and Caring Sciences, Geriatrics.
    Hägglund, Hans
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Effects of sauna bath on heart failure: A systematic review and meta-analysis2018In: Clinical Cardiology, ISSN 0160-9289, E-ISSN 1932-8737, Vol. 41, no 11, p. 1491-1501Article, review/survey (Refereed)
    Abstract [en]

    BACKGROUND:

    Sauna bath has potential as a lifestyle treatment modality for heart failure (HF). It is important to analyze the current evidence to help suggest paths of future study and potential for clinical application.

    HYPOTHESIS:

    Sauna bath has a positive effect on HF patients.

    METHODS:

    PubMed, Cochrane Library, and CINAHL databases were searched to identify randomized and nonrandomized controlled studies to compare effects of sauna bath with no sauna bath. Studies were searched for both infrared sauna bath and Finnish sauna bath. The strength of evidence was rated using a modified GRADE approach. Out of 1444 studies, nine met the inclusion criteria and were included in this review. Seven of these nine studies were included in the meta-analysis. Only studies with infrared sauna bath met the inclusion criteria.

    RESULTS:

    In the meta-analysis, exposure to an infrared sauna bath in 60°C for 15 minutes, followed by a 30-minute rest in warm environment, five times a week for 2 to 4 weeks, was associated with a significant reduction in B-type natriuretic peptide, cardiothoracic ratio, and an improvement in left-ventricular ejection fraction. There was no significant effect on left-ventricular end-diastolic diameter, left atrial diameter, systolic blood pressure, or diastolic blood pressure. The strength of evidence varied from moderate to insufficient.

    CONCLUSION:

    Infrared sauna bath was associated with short-term improvement in cardiac function. More evidence is needed about long-term effects of sauna bath and the effects of a Finnish sauna on cardiovascular health among patients with HF or other cardiovascular diseases.

  • 25. Lazarevic, Vladimir
    et al.
    Remberger, Mats
    Hägglund, Hans
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Juliusson, Gunnar
    Omar, Hamdy
    Halböök, Helene
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Kimby, Eva
    Malm, Claes
    Wahlin, Anders
    Johansson, Jan-Erik
    Long-term survival after allogeneic stem cell transplant for relapsed large B cell lymphomas: a retrospective study2012In: Leukemia and Lymphoma, ISSN 1042-8194, E-ISSN 1029-2403, Vol. 53, no 3, p. 503-505Article in journal (Refereed)
  • 26. Lyberg, Katarina
    et al.
    Ali, Hani Abdulkadir
    Grootens, Jennine
    Kjellander, Matilda
    Tirfing, Malin
    Arock, Michel
    Hägglund, Hans
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Nilsson, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Ungerstedt, Johanna
    Histone deacetylase inhibitor SAHA mediates mast cell death and epigenetic silencing of constitutively active D816V KIT in systemic mastocytosis.2017In: OncoTarget, ISSN 1949-2553, E-ISSN 1949-2553, Vol. 8, no 6, p. 9647-9659Article in journal (Refereed)
    Abstract [en]

    Systemic mastocytosis (SM) is a clonal bone marrow disorder, where therapeutical options are limited. Over 90% of the patients carry the D816V point mutation in the KIT receptor that renders this receptor constitutively active. We assessed the sensitivity of primary mast cells (MC) and mast cell lines HMC1.2 (D816V mutated), ROSA (KIT WT) and ROSA (KIT D816V) cells to histone deacetylase inhibitor (HDACi) treatment. We found that of four HDACi, suberoyl anilide hydroxamic acid (SAHA) was the most effective in killing mutated MC. SAHA downregulated KIT, followed by major MC apoptosis. Primary SM patient MC cultured ex vivo were even more sensitive to SAHA than HMC1.2 cells, whereas primary MC from healthy subjects were less affected. There was a correlation between cell death and SM disease severity, where cell death was more pronounced in the case of aggressive SM, with almost 100% cell death among MC from the mast cell leukemia patient. Additionally, ROSA (KIT D816V) was more affected by HDACi than ROSA (KIT WT) cells. Using ChIP qPCR, we found that the level of active chromatin mark H3K18ac/H3 decreased significantly in the KIT region. This epigenetic silencing was seen only in the KIT region and not in control genes upstream and downstream of KIT, indicating that the downregulation of KIT is exerted by specific epigenetic silencing. In conclusion, KIT D816V mutation sensitized MC to HDACi mediated killing, and SAHA may be of value as specific treatment for SM, although the specific mechanism of action requires further investigation.

  • 27.
    Machaczka, M.
    et al.
    Karolinska Inst, Dept Med Huddinge, Stockholm, Sweden.;Karolinska Univ Hosp, Hematol Ctr Karolinska, Stockholm, Sweden.
    Hägglund, Hans
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology. Karolinska Inst, Dept Med Huddinge, Uppsala, Sweden.
    Staver, E.
    Karolinska Inst, Dept Med Huddinge, Stockholm, Sweden.
    Joks, M.
    Poznan Univ Med Sci, Dept Hematol & Bone Marrow Transplantat, Poznan, Poland.
    Hassan, M.
    Karolinska Inst, Dept Lab Med, Clin Res Ctr, Expt Canc Med, Stockholm, Sweden; Karolinska Univ Hosp, Dept Clin Res Ctr, Stockholm, Sweden.
    Wahlin, B. E.
    Karolinska Inst, Dept Med Huddinge, Stockholm, Sweden; Karolinska Univ Hosp, Hematol Ctr Karolinska, Stockholm, Sweden.
    Nygell, U. Axdorph
    Karolinska Inst, Dept Oncol & Pathol, Stockholm, Sweden; Karolinska Univ Hosp, Dept Clin Immunol & Transfus Med, Stockholm, Sweden.
    G-CSF mobilized peripheral blood stem cell collection for allogeneic transplantation in healthy donors: Analysis Of Factors Affecting Yield2017In: Bone Marrow Transplantation, ISSN 0268-3369, E-ISSN 1476-5365, Vol. 52, no Supplement: 1, p. S139-S139Article in journal (Other academic)
  • 28.
    Machaczka, Maciej
    et al.
    Karolinska Univ Hosp, Karolinska Inst, Dept Med Huddinge, Stockholm, Sweden.;Karolinska Univ Hosp, Hematol Ctr Karolinska, M54, SE-14186 Stockholm, Sweden..
    Hägglund, Hans
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology. Karolinska Univ Hosp, Karolinska Inst, Dept Med Huddinge, Stockholm, Sweden.;Karolinska Univ Hosp, Hematol Ctr Karolinska, Stockholm, Sweden..
    Staver, Emma
    Karolinska Univ Hosp, Karolinska Inst, Dept Med Huddinge, Stockholm, Sweden.;Karolinska Univ Hosp, Hematol Ctr Karolinska, M54, SE-14186 Stockholm, Sweden..
    Joks, Monika
    Poznan Univ Med Sci, Dept Hematol & Bone Marrow Transplantat, Poznan, Poland..
    Hassan, Moustapha
    Karolinska Univ Hosp, Karolinska Inst, Dept Lab Med, Expt Canc Med,Clin Res Ctr, Stockholm, Sweden.;Karolinska Univ Hosp, Clin Res Ctr, Stockholm, Sweden..
    Wahlin, Bjorn Engelbrekt
    Karolinska Univ Hosp, Karolinska Inst, Dept Med Huddinge, Stockholm, Sweden.;Karolinska Univ Hosp, Hematol Ctr Karolinska, M54, SE-14186 Stockholm, Sweden..
    Nygell, Ulla Axdorph
    Karolinska Univ Hosp, Karolinska Inst, Dept Med Huddinge, Stockholm, Sweden.;Karolinska Univ Hosp, Hematol Ctr Karolinska, M54, SE-14186 Stockholm, Sweden.;Karolinska Univ Hosp, Karolinska Inst, Dept Oncol & Pathol, Stockholm, Sweden.;Karolinska Univ Hosp, Dept Clin Immunol & Transfus Med, Stockholm, Sweden..
    G-CSF mobilized peripheral blood stem cell collection for allogeneic transplantation in healthy donors: Analysis of factors affecting yield2017In: Journal of clinical apheresis, ISSN 0733-2459, E-ISSN 1098-1101, Vol. 32, no 6, p. 384-391Article in journal (Refereed)
    Abstract [en]

    Mobilized PBSC are the main source for allogeneic HSCT. We aimed to evaluate factors that affect CD34+ cell yield including the donor's age, gender, BSA, processed blood volume and the method of G-CSF dose calculation. Data from 170 healthy donors were analyzed. The concentration of CD34+ cells in the peripheral blood (PB) and the processed volume of blood were significantly correlated to CD34+ cells yield (P<.00005 and P<.001, respectively). The G-CSF dose per m(2) was significantly correlated to the concentration of CD34+ cells in the PB (P=.0003) and in the product (P=.01). Smaller BSA and less processed volume were found among female donors, who were given lesser G-CSF dose perm(2), and showed lower yield compared to men. However, multivariate analysis of the yield showed that only the concentration of CD34+ cells in the PB and the processed volume remained independent significant.

  • 29. Machaczka, Maciej
    et al.
    Johansson, Jan-Erik
    Remberger, Mats
    Hallböök, Helene
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Lazarevic, Vladimir Lj
    Wahlin, Björn Engelbrekt
    Omar, Hamdy
    Wahlin, Anders
    Juliusson, Gunnar
    Kimby, Eva
    Hägglund, Hans
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    High incidence of chronic graft-versus-host disease after myeloablative allogeneic stem cell transplantation for chronic lymphocytic leukemia in Sweden: graft-versus-leukemia effect protects against relapse2013In: Medical Oncology, ISSN 1357-0560, E-ISSN 1559-131X, Vol. 30, no 4, p. 762-Article in journal (Refereed)
    Abstract [en]

    Allogeneic hematopoietic stem cell transplantation (allo-SCT) is a potentially curative treatment option for eligible patients with chronic lymphocytic leukemia (CLL). However, it is known that cure of CLL is only possible if a graft-versus-leukemia effect is present. Between 1994 and 2007, 48 adults underwent allo-SCT for poor-risk CLL in Sweden. Of these, ten (21%) patients aged 24-53 years (median: 46 years) received myeloablative conditioning (MAC), based on TBI and cyclophosphamide. All MAC patients had refractory, poorly controlled CLL before allo-SCT (partial remission in 9/10 patients and progressive disease in one). The cumulative incidence of acute graft-versus-host disease (GVHD) grades II-IV was 30%. Nine patients developed chronic GVHD; extensive in four. Rates of nonrelapse mortality at 1, 3 and 10 years were 0, 10 and 20%, respectively. Two patients relapsed 36 and 53 months after transplantation. Six patients were still alive after a median follow-up time of 11.5 years (range 5.9-13.7). The probabilities of relapse-free and overall survival from 1, 3 and 5 years after transplantation were 100, 90 and 70%, and 100, 90 and 80%, respectively. Nevertheless, our analysis of long-term outcome after MAC allo-SCT for CLL suggests that younger patients with poorly controlled CLL may benefit from MAC allo-SCT.

  • 30. Machaczka, Maciej
    et al.
    Johansson, Jan-Erik
    Remberger, Mats
    Hallböök, Helene
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Malm, Claes
    Lazarevic, Vladimir Lj
    Wahlin, Anders
    Omar, Hamdy
    Juliusson, Gunnar
    Kimby, Eva
    Hägglund, Hans
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Allogeneic hematopoietic stem cell transplant with reduced-intensity conditioning for chronic lymphocytic leukemia in Sweden: does donor T-cell engraftment 3 months after transplant predict survival?2012In: Leukemia and Lymphoma, ISSN 1042-8194, E-ISSN 1029-2403, Vol. 53, no 9, p. 1699-1705Article in journal (Refereed)
    Abstract [en]

    Thirty-eight adult patients with chronic lymphocytic leukemia (CLL) underwent reduced-intensity conditioning (RIC) allogeneic stem cell transplant (allo-SCT) in Sweden between 1999 and 2007. The cumulative incidences of acute graft-versus-host disease (GVHD) grades II-IV and chronic GVHD were 29% and 47%, respectively. Rates of non-relapse mortality, progression-free survival (PFS) and overall survival (OS) were 18%, 47% and 74% at 1 year, and 21%, 25% and 45% at 5 years, respectively. T-cell chimerism after transplant was measured in 31 out of 34 patients (91%) surviving beyond day + 100. Seventeen patients achieved > 90% donor T-cell engraftment at 3 months after allo-SCT and, compared with the 12 patients with <= 90% donor T-cell engraftment, they showed favorable PFS at 1 year (82% vs. 33%, p = 0.002) and better long-term PFS and OS (p = 0.002 and 0.046, respectively). Donor T-cell engraftment of > 90% at 3 months after RIC allo-SCT for CLL seems to predict favorable short-term and long-term outcome.

  • 31. Machaczka, Maciej
    et al.
    Kämpe Björkvall, Cecilia
    Wieremiejczyk, Joanna
    Paucar Arce, Martin
    Myhr-Eriksson, Kristina
    Klimkowska, Monika
    Hägglund, Hans
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Svenningsson, Per
    Impact of imiglucerase supply shortage on clinical and laboratory parameters in norrbottnian patients with Gaucher disease type 3.2015In: Archivum Immunologiae et Therapiae Experimentalis, ISSN 0004-069X, E-ISSN 1661-4917, Vol. 63, no 1, p. 65-71Article in journal (Refereed)
    Abstract [en]

    A viral contamination of the production plant producing imiglucerase (Cerezyme™) resulted in an unpredicted worldwide shortage of global supplies during 2009-2010. The aim of the study was to describe the effects of dose reduction of enzyme replacement therapy (ERT) in adults with Norrbottnian form of Gaucher disease type 3 (N-GD3). There were ten adults with N-GD3 treated with imiglucerase in the county of Norrbotten in June 2009. Analyzed variables included plasma chitotriosidase activity and concentration of CCL18/PARC, whole blood hemoglobin concentration (Hb) and platelet count (PLT), as well as patients' body weight, subjective complaints and health status measured by the EuroQoL-5D questionnaire. The median duration of ERT shortage lasted for 14 months (10-20 months). The median percentage reduction of imiglucerase dose was 36 % (26-59 %). Hb decreased in four patients, PLT decreased in three patients, chitotriosidase increased in three patients (max. +22 % of baseline), and CCL18/PARC increased in six patients (+14 % to +57 %). The body weight was moderately decreased in one patient. No new bone events were noted. Self-assessment of individual patient's health status was stable in all but one patient. Our results suggest that moderate reduction of ERT dosage lasting for relatively short period of time can lead to worsening in biomarkers of adults with N-GD3. However, this worsening is infrequently translated to clinical worsening of patients. It is possible that CCL18/PARC has a higher sensitivity than chitotriosidase in monitoring of ERT dosing in GD3.

  • 32.
    Pahnke, Simon
    et al.
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Egeland, Torstein
    Univ Oslo, Oslo Univ Hosp, Inst Immunol, Rikshosp, Oslo, Norway;Univ Oslo, Inst Clin Med, Oslo, Norway.
    Halter, Jorg
    Univ Hosp Basel, Dept Hematol, Basel, Switzerland;Univ Basel, Basel, Switzerland.
    Hägglund, Hans
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Shaw, Bronwen E.
    Med Coll Wisconsin, Ctr Int Blood & Bone Marrow Transplant Res, Milwaukee, WI 53226 USA.
    Woolfrey, Ann E.
    Fred Hutchinson Canc Res Ctr, Div Clin Res, 1124 Columbia St, Seattle, WA 98104 USA;Univ Washington, Dept Med, Seattle, WA USA.
    Szer, Jeff
    Royal Melbourne Hosp, Victorian Comprehens Canc Ctr, Integrated Haematol Dept, Melbourne, Vic, Australia.
    Current use of biosimilar G-CSF for haematopoietic stem cell mobilisation2019In: Bone Marrow Transplantation, ISSN 0268-3369, E-ISSN 1476-5365, Vol. 54, no 6, p. 858-866Article in journal (Refereed)
    Abstract [en]

    Despite biosimilars of the granulocyte-colony stimulating factor (G-CSF) filgrastim being approved by the European Medicines Agency since 2008, there is still some debate regarding their use in related and unrelated healthy haematopoietic stem cell donors. We present a review of published experiences using biosimilar filgrastim for healthy donor mobilisation as well as the results of a survey by the World Marrow Donor Association (WMDA) of its current use by register-associated transplant and collection centres for both related and unrelated donors. A total of 1287 healthy donors and volunteers are included in the reviewed studies. The pharmacokinetics and pharmacodynamics studies show a high degree of similarity to the reference product Neupogen. Mobilisation of CD34 + cells as well as reported adverse events are also found to be comparable, although there is still a lack of long-term follow up for both Neupogen and filgrastim biosimilars. No evidence is found of a higher risk of filgrastim antibody formation using filgrastim biosimilars. Based on this increased experience, the WMDA therefore recommend that Stem Cell Donor Registries can use filgrastim biosimilars for the mobilisation of peripheral blood progenitor cells in healthy donors, provided that they are approved by national and/or regional agencies.

  • 33.
    Pahnke, Simon
    et al.
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology. Falun Cty Hosp, Haematol, Falun, Sweden..
    Fischer-Nielsen, A.
    Copenhagen Univ Hosp, Clin Immunol, Copenhagen, Denmark..
    Haastrup, E.
    Copenhagen Univ Hosp, Clin Immunol, Copenhagen, Denmark..
    Heldal, D.
    Oslo Univ Hosp, Haematol, Oslo, Norway..
    Itala-Remes, M.
    Turku Univ Hosp, Stem Cell Transplantat Unit, FIN-20520 Turku, Finland..
    Kauppila, M.
    Turku Univ Hosp, Haematol, FIN-20520 Turku, Finland..
    Larfors, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology. Univ Uppsala Hosp, Haematol, Uppsala, Sweden..
    Niittyvuopio, R.
    Helsinki Univ Hosp, Stem Cell Transplantat Unit, Helsinki, Finland..
    Hagglund, Hans
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology. Univ Uppsala Hosp, Haematol, Uppsala, Sweden..
    Differences in side effects, sick leave and the will to donate again: the Nordic Register of Haematopoietic Stem Cell Donors2016In: Bone Marrow Transplantation, ISSN 0268-3369, E-ISSN 1476-5365, Vol. 51, p. S328-S329Article in journal (Other academic)
  • 34.
    Pahnke, Simon
    et al.
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Larfors, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Axdorph-Nygell, Ulla
    Karolinska University Hospital, Stockholm, Sweden .
    Fischer-Nielsen, Anne
    Copenhagen University Hospital, Copenhagen, Denmark.
    Haastrup, Eva
    Copenhagen University Hospital, Copenhagen, Denmark.
    Heldal, Dag
    Oslo University Hospital, Oslo, Norway.
    Itälä-Remes, Maija
    Turku University Hospital, Turku, Finland.
    Johansson, Jan-Erik
    University of Gothenburg, Gothenburg, Sweden.
    Kauppila, Marjut
    Turku University Hospital, Turku, Finland.
    Lenhoff, Stig
    Skåne University Hospital, Lund, Sweden.
    Ljungman, Per
    Karolinska Institutet, Stockholm, Sweden.
    Niittyvuopio, Riita
    Helsinki University Hospital, Helsinki, Finland.
    Sandstedt, Anna
    Linköping University Hospital, Linköping, Sweden.
    Hägglund, Hans
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Short-term side effects and attitudes towards second donation: A comparison of related and unrelated haematopoietic stem cell donors2018In: Journal of clinical apheresis, ISSN 0733-2459, E-ISSN 1098-1101, Vol. 33, no 3, p. 226-235Article in journal (Refereed)
    Abstract [en]

    The Nordic Register of Haematopoietic Stem Cell Donors (NRHSD) has registered related and unrelated donors from 10 transplant centres in Sweden, Norway, Finland and Denmark since 1998. We present a prospective, observational study of 1,957 donors, focusing mainly on the differences between related and unrelated donors. Related donors are reported to have more comorbidities, but similar side effects compared with unrelated donors. Side effects after BM or PBSC donation are generally of short duration and in this study no deaths, myocardial infarctions, splenic ruptures, or thromboembolic events are reported. Interestingly, related donors express more hesitancy towards donating again when asked 1 month after donation.

  • 35. Ruutu, Tapani
    et al.
    Juvonen, Eeva
    Remberger, Mats
    Remes, Kari
    Volin, Liisa
    Mattsson, Jonas
    Nihtinen, Anne
    Hägglund, Hans
    Centre for Allogeneic Stem Cell Transplantation, Departments of Clinical Immunology and Medicine, Karolinska Hospital, Huddinge University Hospital, Huddinge, Sweden.
    Ringdén, Olle
    Improved survival with ursodeoxycholic acid prophylaxis in allogeneic stem cell transplantation: long-term follow-up of a randomized study2014In: Biology of blood and marrow transplantation, ISSN 1083-8791, E-ISSN 1523-6536, Vol. 20, no 1, p. 135-138Article in journal (Refereed)
    Abstract [en]

    We report the long-term results of a prospective randomized study on the use of ursodeoxycholic acid (UDCA) for prevention of hepatic complications after allogeneic stem cell transplantation. Two hundred forty-two patients, 232 with malignant disease, were randomized to receive (n = 123) or not to receive (n = 119) UDCA from the beginning of the conditioning until 90 days post-transplantation. The results were reported after 1-year follow-up. UDCA administration reduced significantly the proportion of patients developing high serum bilirubin levels as well as the incidence of severe acute graft-versus-host disease (GVHD), liver GVHD, and intestinal GVHD. In the UDCA prophylaxis group, nonrelapse mortality (NRM) was lower and overall survival better than in the control group. After a 10-year follow-up, the difference in the survival and NRM in favor of the UDCA-treated group, seen at 1 year, was maintained (survival 48% versus 38%, P = .037; NRM 28% versus 41%, P = .01). A landmark analysis in patients surviving at 1 year post-transplantation showed no significant differences between the study groups in the long-term follow-up in chronic GVHD, relapse rate, NRM, disease-free survival, or overall survival. These long-term results continue to support the useful role of UDCA in the prevention of transplant-related complications in allogeneic transplantation.

  • 36.
    Simonson, Oscar E.
    et al.
    Karolinska Inst, Dept Mol Med & Surg, Karolinska Univ Hosp, Stockholm, Sweden.;Karolinska Inst, Dept Cardiothorac Surg & Anesthesia, Karolinska Univ Hosp, Stockholm, Sweden.;Karolinska Inst, Dept Lab Med, Karolinska Univ Hosp, Stockholm, Sweden..
    Mougiakakos, Dimitrios
    Univ Erlangen Nurnberg, Dept Internal Med Hematol & Oncol, D-91054 Erlangen, Germany..
    Heldring, Nina
    Karolinska Inst, Dept Lab Med, Karolinska Univ Hosp, Stockholm, Sweden..
    Bassi, Giulio
    Univ Verona, Dept Med, Sect Hematol, Stem Cell Res Lab, I-37100 Verona, Italy..
    Johansson, Henrik J.
    Karolinska Inst, Dept Oncol Pathol, Canc Prote Mass Spectrometry, Sci Life Lab, Stockholm, Sweden..
    Dalen, Magnus
    Karolinska Inst, Dept Mol Med & Surg, Karolinska Univ Hosp, Stockholm, Sweden.;Karolinska Inst, Dept Cardiothorac Surg & Anesthesia, Karolinska Univ Hosp, Stockholm, Sweden..
    Jitschin, Regina
    Karolinska Inst, Dept Lab Med, Karolinska Univ Hosp, Stockholm, Sweden..
    Rodin, Sergey
    Karolinska Inst, Dept Med Biochem & Biophys, Stockholm, Sweden..
    Corbascio, Matthias
    Karolinska Inst, Dept Mol Med & Surg, Karolinska Univ Hosp, Stockholm, Sweden.;Karolinska Inst, Dept Cardiothorac Surg & Anesthesia, Karolinska Univ Hosp, Stockholm, Sweden.;Vaccine & Gene Therapy Inst Florida, Ctr Dis Aging, Port St Lucie, FL USA..
    El Andaloussi, Samir
    Karolinska Inst, Dept Lab Med, Karolinska Univ Hosp, Stockholm, Sweden..
    Wiklander, Oscar P. B.
    Karolinska Inst, Dept Lab Med, Karolinska Univ Hosp, Stockholm, Sweden..
    Nordin, Joel Z.
    Karolinska Inst, Dept Lab Med, Karolinska Univ Hosp, Stockholm, Sweden..
    Skog, Johan
    Exosome Diagnost Inc, New York, NY USA..
    Romain, Charlotte
    Exosome Diagnost Inc, New York, NY USA..
    Koestler, Tina
    Exosome Diagnost Inc, New York, NY USA..
    Johansson Hellgren, Laila
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Surgical Sciences, Thoracic Surgery.
    Schiller, Petter
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Surgical Sciences, Thoracic Surgery.
    Joachimsson, Per-Olof
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Surgical Sciences, Anaesthesiology and Intensive Care.
    Hägglund, Hans
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology. Univ Uppsala Hosp, Dept Hematol, Uppsala, Sweden..
    Mattsson, Mattias
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology. Univ Uppsala Hosp, Dept Hematol, Uppsala, Sweden..
    Lentio, Janne
    Faridani, Omid R.
    Ludwig Inst Canc Res, S-10401 Stockholm, Sweden..
    Sandberg, Rickard
    Karolinska Inst, Dept Cell & Mol Biol, Stockholm, Sweden.;Ludwig Inst Canc Res, S-10401 Stockholm, Sweden..
    Korsgren, Olle
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Immunology, Genetics and Pathology, Clinical Immunology.
    Krampera, Mauro
    Univ Verona, Dept Med, Sect Hematol, Stem Cell Res Lab, I-37100 Verona, Italy..
    Weiss, Daniel J.
    Univ Vermont, Dept Med, Hlth Sci Res Facil, Burlington, VT USA..
    Grinnemo, Karl-Henrik
    Karolinska Inst, Dept Mol Med & Surg, Karolinska Univ Hosp, Stockholm, Sweden.;Karolinska Inst, Dept Cardiothorac Surg & Anesthesia, Karolinska Univ Hosp, Stockholm, Sweden.;Vaccine & Gene Therapy Inst Florida, Ctr Dis Aging, Port St Lucie, FL USA..
    Le Blanc, Katarina
    Karolinska Inst, Dept Lab Med, Karolinska Univ Hosp, Stockholm, Sweden..
    In Vivo Effects of Mesenchymal Stromal Cells in Two Patients With Severe Acute Respiratory Distress Syndrome2015In: Stem Cells Translational Medicine, ISSN 2157-6564, E-ISSN 2157-6580, Vol. 4, no 10, p. 1199-1213Article in journal (Refereed)
    Abstract [en]

    Mesenchymal stromal cells (MSCs) have been investigated as a treatment for various inflammatory diseases because of their immunomodulatory and reparative properties. However, many basic questions concerning their mechanisms of action after systemic infusion remain unanswered. We performed a detailed analysis of the immunomodulatory properties and proteomic profile of MSCs systemically administered to two patients with severe refractory acute respiratory distress syndrome (ARDS) on a compassionate use basis and attempted to correlate these with in vivo anti-inflammatory actions. Both patients received 2 x 10(6) cells per kilogram, and each subsequently improved with resolution of respiratory, hemodynamic, and multiorgan failure. In parallel, a decrease was seen in multiple pulmonary and systemic markers of inflammation, including epithelial apoptosis, alveolar-capillary fluid leakage, and proinflammatory cytokines, microRNAs, and chemokines. In vitro studies of the MSCs demonstrated a broad anti-inflammatory capacity, including suppression of T-cell responses and induction of regulatory phenotypes in T cells, monocytes, and neutrophils. Some of these in vitro potency assessments correlated with, and were relevant to, the observed in vivo actions. These experiences highlight both the mechanistic information that can be gained from clinical experience and the value of correlating in vitro potency assessments with clinical effects. The findings also suggest, but do not prove, a beneficial effect of lung protective strategies using adoptively transferred MSCs in ARDS. Appropriate randomized clinical trials are required to further assess any potential clinical efficacy and investigate the effects on in vivo inflammation. STEM CELLS TRANSLATIONAL MEDICINE 2015;4:1199-1213

  • 37.
    Ustun, Celalettin
    et al.
    Univ Minnesota, Div Hematol Oncol & Transplantat, Minneapolis, MN 55455 USA..
    Gotlib, Jason
    Stanford Univ, Div Hematol, Stanford, CA USA..
    Popat, Uday
    Univ Texas MD Anderson Canc Ctr, Dept Stem Cell Transplantat, Houston, TX USA..
    Artz, Andrew
    Univ Chicago, Dept Med, Hematol Oncol Sect, Chicago, IL USA..
    Litzow, Mark
    Mayo Clin, Dept Internal Med, Div Hematol, Minneapolis, MN USA..
    Reiter, Andreas
    Univ Med Ctr Mannheim, Dept Hematol & Oncol, Mannheim, Germany..
    Nakamura, Ryotaro
    City Hope Med Ctr, Dept Hematol Hematopoiet Cell Transplantat, Duarte, CA USA..
    Kluin-Nelemans, Hanneke C.
    Univ Groningen, Univ Med Ctr Groningen, Dept Hematol, Groningen, Netherlands..
    Verstovsek, Srdan
    Univ Texas MD Anderson Canc Ctr, Dept Leukemia, Houston, TX USA..
    Gajewskil, James
    Oregon Hlth & Sci Univ, Dept Hematol, Portland, OR USA..
    Perales, Miguel-Angel
    Mem Sloan Kettering Canc Ctr, Dept Med, Div Hematol Oncol, New York, NY USA..
    George, Tracy
    Univ New Mexico, Dept Pathol, Albuquerque, NM USA..
    Shore, Tsiporah
    Weill Cornell Med Coll, Div Hematol & Oncol, New York, NY USA..
    Sperr, Wolfgang
    Med Univ Vienna, Dept Internal Med 1, Div Hematol & Hemostaseol & Ludwig Boltzmann Clus, Vienna, Austria..
    Saber, Wael
    Med Coll Wisconsin, Ctr Int Blood & Marrow Transplant Res, Dept Med, Milwaukee, WI USA..
    Kota, Vamsi
    Emory Univ, Winship Canc Inst, Dept Hematol & Oncol, Div Hematol, Atlanta, GA USA..
    Yavuz, Akif Selim
    Istanbul Univ, Istanbul Med Sch, Div Hematol, Istanbul, Turkey..
    Pullarkat, Vinod
    Rogosheske, John
    Univ Minnesota, Div Hematol Oncol & Transplantat, Minneapolis, MN 55455 USA..
    Hogan, William
    Mayo Clin, Dept Internal Med, Div Hematol, Minneapolis, MN USA..
    Van Besien, Koen
    Weill Cornell Med Coll, Dept Med, New York, NY USA..
    Hägglund, Hans
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Damaj, Gandhi
    Univ Basse Normandie, Univ Hosp, Sch Med, Inst Hematol,Dept Hematol, Caen, France..
    Arock, Michel
    Ecole Normale Super, CNRS UMR 8113, Cellular & Mol Oncol Unit, Cachan, France.;Univ Pitie Salpetriere, Ctr Hosp, Hematol Lab, Paris, France..
    Horny, Hans-Peter
    LMU, Inst Pathol, Munich, Germany..
    Metcalfe, Dean D.
    NIAID, Mast Cell Biol Sect, Lab Allerg Dis, NIH, Bethesda, MD USA..
    Deeg, H. Joachim
    Univ Washington, Fred Hutchinson Canc Res Ctr, Seattle, WA USA.;Univ Washington, Sch Med, Seattle, WA USA..
    Devine, Steven
    Ohio State Univ, Dept Med, Div Hematol, Columbus, OH 43210 USA.;Ohio State Univ, Ctr Comprehens Canc, Columbus, OH 43210 USA..
    Weisdorfl, Daniel
    Univ Minnesota, Div Hematol Oncol & Transplantat, Minneapolis, MN 55455 USA..
    Akin, Cem
    Harvard Med Sch, Brigham & Womens Hosp, Div Rheumatol Immunol & Allergy, Boston, MA USA..
    Valent, Peter
    Consensus Opinion on Allogeneic Hematopoietic Cell Transplantation in Advanced Systemic Mastocytosis2016In: Biology of blood and marrow transplantation, ISSN 1083-8791, E-ISSN 1523-6536, Vol. 22, no 8, p. 1348-1356Article in journal (Other academic)
  • 38. Ustun, Celalettin
    et al.
    Reiter, Andreas
    Scott, Bart L
    Nakamura, Ryotaro
    Damaj, Gandhi
    Kreil, Sebastian
    Shanley, Ryan
    Hogan, William J
    Perales, Miguel-Angel
    Shore, Tsiporah
    Baurmann, Herrad
    Stuart, Robert
    Gruhn, Bernd
    Doubek, Michael
    Hsu, Jack W
    Tholouli, Eleni
    Gromke, Tanja
    Godley, Lucy A
    Pagano, Livio
    Gilman, Andrew
    Wagner, Eva Maria
    Shwayder, Tor
    Bornhäuser, Martin
    Papadopoulos, Esperanza B
    Böhm, Alexandra
    Vercellotti, Gregory
    Van Lint, Maria Teresa
    Schmid, Christoph
    Rabitsch, Werner
    Pullarkat, Vinod
    Legrand, Faezeh
    Yakoub-Agha, Ibrahim
    Saber, Wael
    Barrett, John
    Hermine, Olivier
    Hägglund, Hans
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology. Karolinska University Hospital, Stockholm, Sweden.
    Sperr, Wolfgang R
    Popat, Uday
    Alyea, Edwin P
    Devine, Steven
    Deeg, H Joachim
    Weisdorf, Daniel
    Akin, Cem
    Valent, Peter
    Hematopoietic stem-cell transplantation for advanced systemic mastocytosis2014In: Journal of Clinical Oncology, ISSN 0732-183X, E-ISSN 1527-7755, Vol. 32, no 29, p. 3264-3274Article in journal (Refereed)
    Abstract [en]

    PURPOSE: Advanced systemic mastocytosis (SM), a fatal hematopoietic malignancy characterized by drug resistance, has no standard therapy. The effectiveness of allogeneic hematopoietic stem-cell transplantation (alloHCT) in SM remains unknown.

    PATIENTS AND METHODS: In a global effort to define the value of HCT in SM, 57 patients with the following subtypes of SM were evaluated: SM associated with clonal hematologic non-mast cell disorders (SM-AHNMD; n = 38), mast cell leukemia (MCL; n = 12), and aggressive SM (ASM; n = 7). Median age of patients was 46 years (range, 11 to 67 years). Donors were HLA-identical (n = 34), unrelated (n = 17), umbilical cord blood (n = 2), HLA-haploidentical (n = 1), or unknown (n = 3). Thirty-six patients received myeloablative conditioning (MAC), and 21 patients received reduced-intensity conditioning (RIC).

    RESULTS: Responses in SM were observed in 40 patients (70%), with complete remission in 16 patients (28%). Twelve patients (21%) had stable disease, and five patients (9%) had primary refractory disease. Overall survival (OS) at 3 years was 57% for all patients, 74% for patients with SM-AHNMD, 43% for those with ASM, and 17% for those with MCL. The strongest risk factor for poor OS was MCL. Survival was also lower in patients receiving RIC compared with MAC and in patients having progression compared with patients having stable disease or response.

    CONCLUSION: AlloHCT was associated with long-term survival in patients with advanced SM. Although alloHCT may be considered as a viable and potentially curative therapeutic option for advanced SM in the meantime, given that this is a retrospective analysis with no control group, the definitive role of alloHCT will need to be determined by a prospective trial.

  • 39. Valent, P
    et al.
    Sotlar, K
    Sperr, W R
    Escribano, L
    Yavuz, S
    Reiter, A
    George, T I
    Kluin-Nelemans, H C
    Hermine, O
    Butterfield, J H
    Hägglund, Hans
    Hematology Center Karolinska, Karolinska University Hospital, Stockholm, Sweden.
    Ustun, C
    Hornick, J L
    Triggiani, M
    Radia, D
    Akin, C
    Hartmann, K
    Gotlib, J
    Schwartz, L B
    Verstovsek, S
    Orfao, A
    Metcalfe, D D
    Arock, M
    Horny, H-P
    Refined diagnostic criteria and classification of mast cell leukemia (MCL) and myelomastocytic leukemia (MML): a consensus proposal2014In: Annals of Oncology, ISSN 0923-7534, E-ISSN 1569-8041, Vol. 25, no 9, p. 1691-1700Article in journal (Refereed)
    Abstract [en]

    Mast cell leukemia (MCL), the leukemic manifestation of systemic mastocytosis (SM), is characterized by leukemic expansion of immature mast cells (MCs) in the bone marrow (BM) and other internal organs; and a poor prognosis. In a subset of patients, circulating MCs are detectable. A major differential diagnosis to MCL is myelomastocytic leukemia (MML). Although criteria for both MCL and MML have been published, several questions remain concerning terminologies and subvariants. To discuss open issues, the EU/US-consensus group and the European Competence Network on Mastocytosis (ECNM) launched a series of meetings and workshops in 2011-2013. Resulting discussions and outcomes are provided in this article. The group recommends that MML be recognized as a distinct condition defined by mastocytic differentiation in advanced myeloid neoplasms without evidence of SM. The group also proposes that MCL be divided into acute MCL and chronic MCL, based on the presence or absence of C-Findings. In addition, a primary (de novo) form of MCL should be separated from secondary MCL that typically develops in the presence of a known antecedent MC neoplasm, usually aggressive SM (ASM) or MC sarcoma. For MCL, an imminent prephase is also proposed. This prephase represents ASM with rapid progression and 5%-19% MCs in BM smears, which is generally accepted to be of prognostic significance. We recommend that this condition be termed ASM in transformation to MCL (ASM-t). The refined classification of MCL fits within and extends the current WHO classification; and should improve prognostication and patient selection in practice as well as in clinical trials.

  • 40.
    Valent, Peter
    et al.
    Med Univ Vienna, Dept Internal Med 1, Div Hematol & Hemostaseol, Vienna, Austria;Med Univ Vienna, Ludwig Boltzmann Inst Hematol & Oncol, Vienna, Austria.
    Elberink, Joanna N. G. Oude
    Univ Groningen, Univ Med Ctr Groningen, Dept Allergol, Groningen, Netherlands.
    Gorska, Aleksandra
    Med Univ Gdansk, Dept Allergol, Gdansk, Poland.
    Lange, Magdalena
    Med Univ Gdansk, Dept Dermatol Venereol & Allergol, Gdansk, Poland.
    Zanotti, Roberta
    Verona Univ Hosp, Dept Med, Sect Hematol, Verona, Italy.
    van Anrooij, Bjorn
    Univ Groningen, Univ Med Ctr Groningen, Dept Allergol, Groningen, Netherlands.
    Bonifacio, Massimiliano
    Verona Univ Hosp, Dept Med, Sect Hematol, Verona, Italy.
    Bonadonna, Patrizia
    Med Univ Vienna, Ludwig Boltzmann Inst Hematol & Oncol, Vienna, Austria;Verona Univ Hosp, Allergy Unit, Verona, Italy.
    Gleixner, Karoline V.
    Med Univ Vienna, Dept Internal Med 1, Div Hematol & Hemostaseol, Vienna, Austria;Med Univ Vienna, Ludwig Boltzmann Inst Hematol & Oncol, Vienna, Austria.
    Hadzijusufovic, Emir
    Med Univ Vienna, Dept Internal Med 1, Div Hematol & Hemostaseol, Vienna, Austria;Univ Vet Med Vienna, Clin Internal Med & Infect Dis, Dept Compan Anim & Horses, Vienna, Austria.
    Perkins, Cecelia
    Stanford Univ, Dept Med, Sch Med, Div Hematol,Stanford Canc Inst, Stanford, CA 94305 USA.
    Hartmann, Karin
    Univ Cologne, Dept Dermatol, Cologne, Germany;Univ Lubeck, Dept Dermatol, Lubeck, Germany.
    Illerhaus, Anja
    Univ Cologne, Dept Dermatol, Cologne, Germany.
    Merante, Serena
    Univ Pavia, Dept Mol Med, Pavia, Italy;Univ Pavia, Dept Hematol Oncol, Pavia, Italy;Fdn IRCCS Policlin San Matteo, Pavia, Italy.
    Elena, Chiara
    Univ Pavia, Dept Mol Med, Pavia, Italy;Univ Pavia, Dept Hematol Oncol, Pavia, Italy;Fdn IRCCS Policlin San Matteo, Pavia, Italy.
    Shoumariyeh, Khalid
    Univ Freiburg, Fac Med, Dept Hematol Oncol & Stem Cell Transplantat, Med Ctr, Freiburg, Germany.
    von Bubnoff, Nikolas
    Univ Freiburg, Fac Med, Dept Hematol Oncol & Stem Cell Transplantat, Med Ctr, Freiburg, Germany;German Canc Consortium DKTK Partner Site Freiburg, Freiburg, Germany.
    Parente, Roberta
    Univ Salerno, Div Allergy & Clin Immunol, Salerno, Italy.
    Triggiani, Massimo
    Univ Salerno, Div Allergy & Clin Immunol, Salerno, Italy.
    Schwaab, Juliana
    Heidelberg Univ, Univ Med Mannheim, Med Klin, Hamatol & Onkol 3, Mannheim, Germany.
    Jawhar, Mohamad
    Heidelberg Univ, Univ Med Mannheim, Med Klin, Hamatol & Onkol 3, Mannheim, Germany.
    Caroppo, Francesca
    Univ Padua, Dept Med, Pediat Dermatol Unit, Padua, Italy.
    Fortina, Anna Belloni
    Univ Padua, Dept Med, Pediat Dermatol Unit, Padua, Italy.
    Brockow, Knut
    Tech Univ Munich, Dept Dermatol & Allergy Biederstein, Munich, Germany.
    Fuchs, David
    Johannes Kepler Univ Linz, Kepler Univ Hosp, Dept Internal Med 3, Hematol & Oncol, Linz, Austria.
    Greul, Rosemarie
    Johannes Kepler Univ Linz, Kepler Univ Hosp, Dept Internal Med 3, Hematol & Oncol, Linz, Austria.
    Yavuz, Akif Selim
    Istanbul Univ, Istanbul Med Sch, Div Hematol, Istanbul, Turkey.
    Doubek, Michael
    Univ Hosp Brno, Brno, Czech Republic.
    Mattsson, Mattias
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology. Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Immunology, Genetics and Pathology, Experimental and Clinical Oncology.
    Hägglund, Hans
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Panse, Jens
    Univ Hosp RWTH Aachen, Dept Oncol Haematol Haemostaseol & Stem Cell Tran, Aachen, Germany.
    Sabato, Vito
    Univ Antwerp, Dept Immunol Allergol Rheumatol, Fac Med & Hlth Sci, Antwerp, Belgium;Antwerp Univ Hosp, Antwerp, Belgium.
    Aberer, Elisabeth
    Med Univ Graz, Dept Dermatol & Venereol, Graz, Austria.
    Al-Ali, Haifa Kathrin
    Univ Hosp Leipzig, Leipzig, Germany.
    Morren, Marie-Anne
    Univ Hosp Leuven, Dept Dermatol, Leuven, Belgium.
    Varkonyi, Judit
    Semmelweis Univ, Dept Hematol, Budapest, Hungary.
    Zink, Alexander
    Tech Univ Munich, Dept Dermatol & Allergy Biederstein, Munich, Germany.
    Niedoszytko, Marek
    Med Univ Gdansk, Dept Allergol, Gdansk, Poland.
    Niederwieser, Dietger
    Univ Hosp Leipzig, Leipzig, Germany.
    Malcovati, Luca
    Univ Pavia, Dept Mol Med, Pavia, Italy.
    Reiter, Andreas
    Heidelberg Univ, Univ Med Mannheim, Med Klin, Hamatol & Onkol 3, Mannheim, Germany.
    Kennedy, Vanessa
    Stanford Univ, Dept Med, Sch Med, Div Hematol,Stanford Canc Inst, Stanford, CA 94305 USA.
    Gotlib, Jason
    Stanford Univ, Dept Med, Sch Med, Div Hematol,Stanford Canc Inst, Stanford, CA 94305 USA.
    Lortholary, Olivier
    Paris Descartes Univ, Ctr Natl Reference Mastocytoses, Necker Pasteur Ctr Infect Dis & Trop Med, Inst Imagine, Paris, France;Paris Descartes Univ, Ctr Natl Reference Mastocytoses, Necker Enfants Malad, Inst Imagine, Paris, France.
    Hermine, Olivier
    Univ Paris 05, Hop Necker, APHP,Ctr Reference Mastocytoses, Imagine Inst,INSERM,U1123,Sorbonne Paris Cite,Dep, Paris, France.
    Arock, Michel
    Hop La Pitie Salpetriere, Lab Hematol, Paris, France.
    Kluin-Nelemans, Hanneke
    Univ Groningen, Univ Med Ctr Groningen, Dept Hematol, Groningen, Netherlands.
    Sperr, Wolfgang R.
    Med Univ Vienna, Dept Internal Med 1, Div Hematol & Hemostaseol, Vienna, Austria;Med Univ Vienna, Ludwig Boltzmann Inst Hematol & Oncol, Vienna, Austria.
    The Data Registry of the European Competence Network on Mastocytosis (ECNM): Set Up, Projects, and Perspectives2019In: Journal of Allergy and Clinical Immunology: In Practice, ISSN 2213-2198, E-ISSN 2213-2201, Vol. 7, no 1, p. 81-87Article, review/survey (Refereed)
    Abstract [en]

    Mastocytosis is a unique hematologic neoplasm with complex biology and pathology and a variable clinical course. The disease can essentially be divided into cutaneous mastocytosis (CM) and systemic mastocytosis (SM). In adults, SM is diagnosed in most cases and manifests as either indolent or advanced disease. Patients with advanced SM have an unfavorable prognosis with reduced survival. However, so far, little is known about the prevalence of various categories of SM and about prognostic factors. In an attempt to learn more about the behavior and evolution of various forms of CM and SM, the European Competence Network on Mastocytosis (ECNM) initiated a mastocytosis registry in 2012. In this article, the set up and start phase of this registry are described. Until 2018, more than 3000 patients from 12 countries and 25 centers have been enrolled. In a majority of all patients, robust follow-up data and relevant clinical end points are available. Using this data set, a series of registry projects have been launched, with the aim to validate previously identified diagnostic and prognostic variables and to identify new disease-related and patient-related parameters in various forms of mastocytosis. Moreover, the core data set of the registry will be useful to establish multiparametric scoring systems through which prognostication and individualized management of patients with mastocytosis should improve in the foreseeable future. (C) 2019 American Academy of Allergy, Asthma & Immunology

  • 41.
    Worel, Nina
    et al.
    Med Univ Vienna, Dept Blood Grp Serol & Transfus Med, A-1090 Vienna, Austria..
    Buser, Andreas
    Swiss Red Cross, Ctr Blood Transfus, Basel, Switzerland.;Univ Basel Hosp, Dept Hematol, CH-4031 Basel, Switzerland..
    Greinix, Hildegard T.
    Med Univ Graz, Div Hematol, Graz, Austria..
    Hagglund, Hans
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Navarro, Willis
    Natl Marrow Donor Program, Minneapolis, MN USA..
    Pulsipher, Michael A.
    Univ Utah, Div Hematol & Hematol Malignancies, Huntsman Canc Inst, Primary Childrens Hosp, Salt Lake City, UT USA..
    de Faveri, Grazia Nicoloso
    Swiss Blood Stem Cell, Bern, Switzerland..
    Bengtsson, Mats
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Immunology, Genetics and Pathology, Clinical Immunology. Swedish Bone Marrow Donors, Tobias Registry, Uppsala, Sweden..
    Billen, Annelies
    UCL Canc Inst, London, England..
    Espino, German
    Univ Hosp Caja del Seguro Social, Hematol & Bone Marrow Transplantat Sect, Dept Internal Med, Panama City, Panama..
    Fechter, Mirjam
    Leiden Univ, Med Ctr, Europdonor Fdn Leiden, Leiden, Netherlands..
    Giudice, Valeria
    S Orsola Malpighi Univ Hosp, Dept Immunohematol & Transfus Med, Bologna, Italy..
    Hoelig, Kristina
    Tech Univ Dresden, Univ Hosp Carl Gustav Cams, Dept Internal Med 1, D-01062 Dresden, Germany..
    Kanamori, Heiwa
    Kanagawa Canc Ctr, Dept Hematol, Yokohama, Kanagawa, Japan..
    Kodera, Yoshihisa
    Aichi Med Univ, Asia Pacific Blood & Marrow Transplantat Grp, Sch Med, Nagakute, Aichi 48011, Japan.;Aichi Med Univ, Dept Promot Blood & Marrow Transplantat, Sch Med, Nagakute, Aichi 48011, Japan..
    Leitner, Gerda
    Med Univ Vienna, Dept Blood Grp Serol & Transfus Med, A-1090 Vienna, Austria..
    Netelenbos, Tanja
    Leiden Univ, Dept Immunohematol & Blood Transfus, Med Ctr, Leiden, Netherlands..
    Niedervvieser, Dietger
    Univ Hosp Leipzig, Dept Hematol, Leipzig, Germany..
    van Walraven, Suzanna M.
    Leiden Univ, Med Ctr, Europdonor Fdn Leiden, Leiden, Netherlands.;World Marrow Donor Assoc, Eth Working Grp, Leiden, Netherlands.;Oxford Univ Hosp NHS Trust, British Bone Marrow Donor Registry, Oxford, England..
    Rocha, Vanderson
    NHS BT, Cord Blood Banks, Oxford, England..
    Torosian, Tigran
    Fdn DKMS Polska, Warsaw, Poland..
    Vergueiro, Carmen
    FCM Santa Casa de Selo Paulo, Disciplina Hematol & Oncol, Sao Paulo, Brazil..
    Weisdorf, Daniel
    Univ Minnesota, Bone Marrow Transplant Program, Minneapolis, MN 55455 USA..
    Yabe, Hiromasa
    Tokai Univ, Sch Med, Dept Cell Transplantat & Regenerat Med, Tokyo 151, Japan..
    Halter, Joerg P.
    Univ Basel Hosp, Dept Hematol, CH-4031 Basel, Switzerland..
    Suitability Criteria for Adult Related Donors: A Consensus Statement from the Worldwide Network for Blood and Marrow Transplantation Standing Committee on Donor Issues2015In: Biology of blood and marrow transplantation, ISSN 1083-8791, E-ISSN 1523-6536, Vol. 21, no 12, p. 2052-2060Article in journal (Refereed)
    Abstract [en]

    The number of allogeneic hematopoietic stem cell (HSC) transplants performed globally each year continues to increase. Advances in HLA typing, better supportive care, and administration of reduced-intensity conditioning regimens allow treatment of older patients with older sibling donors. Pretransplant donor assessment and testing are very important processes affecting the quality and safety of donation. For unrelated HSC donors detailed recommendations for health assessment have been published, allowing donation only if they are unrestrictedly healthy. Eligibility criteria for related donors are less strict and vary significantly between centers. In situations where a family donor does not meet the suitability criteria for unrelated donors, involved physicians often struggle with the decision whether the matched relative is suitable for donation or not. On behalf of the Worldwide Network for Blood and Marrow Transplantation Standing Committee on Donor Issues, we intended to develop a consensus document with recommendations for donor workup and final clearance of family donors who would not be able to serve as unrelated donors because of their age or pre-existing diseases. This article covers different topics intending to support decision-making, with the goal of minimizing medical risk to the donor and protection of the recipient from transmissible diseases.

1 - 41 of 41
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