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  • 1. Blimark, Cecilie Hveding
    et al.
    Turesson, Ingemar
    Genell, Anna
    Ahlberg, Lucia
    Björkstrand, Bo
    Carlson, Kristina
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Forsberg, Karin
    Juliusson, Gunnar
    Linder, Olle
    Mellqvist, Ulf-Henrik
    Nahi, Hareth
    Kristinsson, Sigurdur Y
    Outcome and survival of myeloma patients diagnosed 2008-2015. Real world data on 4904 patients from the Swedish Myeloma Registry (SMR)2018In: Haematologica, ISSN 0390-6078, E-ISSN 1592-8721, Vol. 103, no 3, p. 506-513Article in journal (Refereed)
    Abstract [en]

    Epidemiology and outcome of myeloma is mainly reported from large university centers and collaborative groups and do not represent real world patients. The Swedish Myeloma Registry is a prospective population-based registry documenting characteristics, treatments and outcome in newly diagnosed myeloma, including asymptomatic and localized forms, with the purpose to improve the management and outcome. This report presents information on patients diagnosed between 2008 and 2015, including data on first line treatment up to 2014, with a follow-up until December 2016. We present age-adjusted incidence, patient characteristics at baseline, treatment, response, and survival. Baseline data was available with a 97% coverage in 4,904 patients (median age 71 years, males 70 years, females 73 years, 72% were 65 years or older), and one-year follow-up of 3,558 patients with symptomatic disease (92% of patients initially reported). The age-adjusted incidence was 6.8 myeloma cases per 100 000 inhabitants and year. Among initially symptomatic patients (n=3,988), 77% had osteolytic lesions or compression fractures, 49% had anemia, 18% impaired kidney function, and 13% hypercalcemia. High-dose therapy with autologous stem cell transplantation was given to 77% of patients up to 66 years, and to 22% of patients 66-70 years. In the study period, 68% received bortezomib, thalidomide, and/or lenalidomide as part of the first line treatment, rising from 31% in 2008 to 81% 2014. In MM, the median relative survival of patients 65 years or younger was 7.7 years, and 3.4 years in 66 years and older. Patients diagnosed with myeloma in more recent years were associated with significantly higher rates of complete or very good partial remission (p<0.05), and with a significant higher overall survival with a HR of 0.84 (95% CI 0.77-0.92; p< 0.05). There was small, but significant survival benefit in patients treated in university hospitals (HR 0.93; 95% CI 0.87-0.99, p<0.05). Analysis of progression-free survival has to await collection of additional follow-up data. We here report on a near complete real world population of myeloma patients during an 8-year period, when newer drugs were implemented into standard practice. The overall incidence and median age were both higher than in most previous studies, indicating a more complete coverage of older patients. Myeloma survival in Sweden compare to other large registry studies and responses and survival improved during the study period.

  • 2.
    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.

  • 3.
    Burman, Joachim
    et al.
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Neuroscience, Neurology.
    Kirgizov, K
    Russian Childrens Res Hosp, BMT Dept, Moscow, Russia.
    Carlson, Kristina
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Badoglio, M
    Hop St Antoine, EBMT Paris Study Off, Paris, France.
    Mancardi, G L
    Univ Genoa, Dept Neurosci, Genoa, Italy.
    De Luca, G
    Univ G dAnnunzio, Multiple Sclerosis Ctr, Neurol Clin, Chieti, Italy.
    Casanova, B
    Hosp Univ & Politecninc La Fe, Neuroimmunol Unit, Valencia, Spain.
    Ouyang, J
    Nanjing Univ, Dept Hematol, Affiliated Drum Tower Hosp, Med Sch, Nanjing, Jiangsu, Peoples R China.
    Bembeeva, R
    Pirogov Russian Natl Res Med Univ, Neurosurg & Genet Pediat Fac, Dept Neurol, Moscow, Russia.
    Haas, J
    Jud Krankenhaus, Berlin, Germany.
    Bader, P
    GW Goethe Univ Hosp, Dept Children & Adolescents, Div Stem Cell Transplantat & Immunol, Frankfurt, Germany.
    Snowden, J
    Sheffield Teaching Hosp NHS Fdn Trust, Dept Haematol, Sheffield, S Yorkshire, England; Univ Sheffield, Sheffield, S Yorkshire, England.
    Farge, D
    Paris 7 Univ, INSERM U1160, Paris, France; Paris7 Denis Diderot Univ, St Louis Hosp, AP HP,UF 04, Unite Med Interne Malad Autoimmunes & Pathol Vasc, Paris, France.
    Autologous hematopoietic stem cell transplantation for pediatric multiple sclerosis: a registry-based study of the Autoimmune Diseases Working Party (ADWP) and Pediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT)2017In: Bone Marrow Transplantation, ISSN 0268-3369, E-ISSN 1476-5365, Vol. 52, no 8, p. 1133-1137Article in journal (Refereed)
    Abstract [en]

    Autologous hematopoietic stem cell transplantation (aHSCT) is a promising therapy for multiple sclerosis (MS), which has mainly been used in adults. The purpose of this study was to investigate efficacy and adverse events of aHSCT in the treatment of children with MS using data from the European Society for Blood and Marrow Transplantation registry. Twenty-one patients with a median follow-up time of 2.8 years could be identified. PFS at 3 years was 100%, 16 patients improved in expanded disability status scale score and only 2 patients experienced a clinical relapse. The procedure was generally well tolerated and only two instances of severe transplant-related toxicity were recorded. There was no treatment-related mortality, although one patient needed intensive care. aHSCT may be a therapeutic option for children with disease that does not respond to standard care.

  • 4.
    Burt, Richard K.
    et al.
    Northwestern Univ, Dept Med, Div Immunotherapy, Feinberg Sch Med, Chicago, IL 60611 USA.
    Balabanov, Roumen
    Northwestern Univ, Feinberg Sch Med, Dept Neurol, Chicago, IL 60611 USA.
    Burman, Joachim
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Neuroscience, Neurology.
    Sharrack, Basil
    Sheffield Teaching Hosp NHS Fdn Trust, Dept Neurosci, Sheffield, S Yorkshire, England;Sheffield Teaching Hosp NHS Fdn Trust, Sheffield NIHR Translat Neurosci BBC, Sheffield, S Yorkshire, England;Univ Sheffield, Sheffield, S Yorkshire, England.
    Snowden, John A.
    Univ Sheffield, Sheffield, S Yorkshire, England;Sheffield Teaching Hosp NHS Fdn Trust, Dept Haematol & Oncol, Sheffield, S Yorkshire, England;Sheffield Teaching Hosp NHS Fdn Trust, Dept Metab, Sheffield, S Yorkshire, England.
    Oliveira, Maria Carolina
    Univ Sao Paulo, Ctr Cell Based Therapy, Dept Internal Med, Ribeirao Preto Med Sch, Ribeirao Preto, Brazil.
    Fagius, Jan
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Neuroscience, Neurology.
    Rose, John
    Univ Utah, Dept Neurol, Salt Lake City, UT USA.
    Nelson, Flavia
    Univ Minnesota, Dept Neurol, Div Multiple Sclerosis, Minneapolis, MN 55455 USA.
    Barreira, Amilton Antunes
    Univ Sao Paulo, Ribeirao Preto Med Sch, Dept Neurosci & Behav Sci, Ribeirao Preto, Brazil.
    Carlson, Kristina
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Han, Xiaoqiang
    Northwestern Univ, Dept Med, Div Immunotherapy, Feinberg Sch Med, Chicago, IL 60611 USA.
    Moraes, Daniela
    Univ Sao Paulo, Ctr Cell Based Therapy, Dept Internal Med, Ribeirao Preto Med Sch, Ribeirao Preto, Brazil.
    Morgan, Amy
    Northwestern Univ, Dept Med, Div Immunotherapy, Feinberg Sch Med, Chicago, IL 60611 USA.
    Quigley, Kathleen
    Northwestern Univ, Dept Med, Div Immunotherapy, Feinberg Sch Med, Chicago, IL 60611 USA.
    Yaung, Kimberly
    Northwestern Univ, Dept Med, Div Immunotherapy, Feinberg Sch Med, Chicago, IL 60611 USA.
    Buckley, Regan
    Northwestern Univ, Dept Med, Div Immunotherapy, Feinberg Sch Med, Chicago, IL 60611 USA.
    Alldredge, Carri
    Northwestern Univ, Dept Med, Div Immunotherapy, Feinberg Sch Med, Chicago, IL 60611 USA.
    Clendenan, Allison
    Northwestern Univ, Dept Med, Div Immunotherapy, Feinberg Sch Med, Chicago, IL 60611 USA.
    Calvario, Michelle A.
    Northwestern Univ, Dept Med, Div Immunotherapy, Feinberg Sch Med, Chicago, IL 60611 USA.
    Henry, Jacquelyn
    Northwestern Univ, Dept Med, Div Immunotherapy, Feinberg Sch Med, Chicago, IL 60611 USA.
    Jovanovic, Borko
    Northwestern Univ, Feinberg Sch Med, Dept Prevent Med, Chicago, IL 60611 USA.
    Helenowski, Irene B.
    Northwestern Univ, Feinberg Sch Med, Dept Prevent Med, Chicago, IL 60611 USA.
    Effect of Nonmyeloablative Hematopoietic Stem Cell Transplantation vs Continued Disease-Modifying Therapy on Disease Progression in Patients With Relapsing-Remitting Multiple Sclerosis: A Randomized Clinical Trial2019In: Journal of the American Medical Association (JAMA), ISSN 0098-7484, E-ISSN 1538-3598, Vol. 321, no 2, p. 165-174Article in journal (Refereed)
    Abstract [en]

    IMPORTANCE Hematopoietic stem cell transplantation (HSCT) represents a potentially useful approach to slow or prevent progressive disability in relapsing-remitting multiple sclerosis (MS).

    OBJECTIVE To compare the effect of nonmyeloablative HSCT vs disease-modifying therapy (DMT) on disease progression.

    DESIGN, SETTING, AND PARTICIPANTS Between September 20, 2005, and July 7, 2016, a total of 110 patients with relapsing-remitting MS, at least 2 relapses while receiving DMT in the prior year, and an Expanded Disability Status Scale (EDSS; score range, 0-10 [10 = worst neurologic disability]) score of 2.0 to 6.0 were randomized at 4 US, European, and South American centers. Final follow-up occurred in January 2018 and database lock in February 2018.

    INTERVENTIONS Patients were randomized to receive HSCT along with cyclophosphamide (200mg/kg) and antithymocyte globulin (6mg/kg) (n = 55) or DMT of higher efficacy or a different class than DMT taken during the previous year (n = 55).

    MAIN OUTCOMES AND MEASURES The primary end point was disease progression, defined as an EDSS score increase after at least 1 year of 1.0 point or more (minimal clinically important difference, 0.5) on 2 evaluations 6 months apart, with differences in time to progression estimated as hazard ratios. RESULTS Among 110 randomized patients (73 [66%] women; mean age, 36 [SD, 8.6] years), 103 remained in the trial, with 98 evaluated at 1 year and 23 evaluated yearly for 5 years (median follow-up, 2 years; mean, 2.8 years). Disease progression occurred in 3 patients in the HSCT group and 34 patients in the DMT group. Median time to progression could not be calculated in the HSCT group because of too few events; it was 24 months (interquartile range, 18-48 months) in the DMT group (hazard ratio, 0.07; 95% CI, 0.02-0.24; P < .001). During the first year, mean EDSS scores decreased (improved) from 3.38 to 2.36 in the HSCT group and increased (worsened) from 3.31 to 3.98 in the DMT group (between-group mean difference,-1.7; 95% CI,-2.03 to -1.29; P < .001). There were no deaths and no patients who received HSCT developed nonhematopoietic grade 4 toxicities (such as myocardial infarction, sepsis, or other disabling or potential life-threatening events).

    CONCLUSIONS AND RELEVANCE In this preliminary study of patients with relapsing-remitting MS, nonmyeloablative HSCT, compared with DMT, resulted in prolonged time to disease progression. Further research is needed to replicate these findings and to assess long-term outcomes and safety.

  • 5.
    Cesaro, Simone
    et al.
    Azienda Osped Univ Integrata, Paediat Haematol & Oncol, Verona, Italy..
    de latour, Regis Peffault
    Univ Paris 07, Dept Haematol, BMT, Hop St Louis, Paris, France..
    Tridello, Gloria
    Azienda Osped Univ Integrata, Paediat Haematol & Oncol, Verona, Italy..
    Pillon, Marta
    Dipartimento Pediat, Clin Oncoematol Pediat, Padua, Italy..
    Carlson, Kristina
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Fagioli, Franca
    Regina Margherita Hosp, Paediat Haematol, Turin, Italy..
    Jouet, Jean-Pierre
    Hop Claude Huriez Serv Malad Sang, Lille, France..
    Koh, Mickey B. C.
    St George Hosp, Dept Haematol, London, England..
    Panizzolo, Irene Sara
    Azienda Osped Univ Integrata, Paediat Haematol & Oncol, Verona, Italy..
    Kyrcz-Krzemien, Slawomira
    Med Univ Silesia, Univ Dept Haematol, Katowice, Poland.;BMT, Katowice, Poland..
    Maertens, Johan
    Univ Hosp Gasthuisberg, Dept Haematol, Leuven, Belgium..
    Rambaldi, Alessandro
    Osped Riuniti Bergamo, Div Ematol, Bergamo, Italy..
    Strahm, Brigitte
    Univ Med Ctr, Dept Paediat & Adolescent Med, Paediat Haematol & Oncol, Freiburg, Germany..
    Blaise, Didier
    Inst Paoli Calmettes, Ctr Rech Cancerol Marseille, Programme Transplantat & Therapie Cellulaire, Marseille, France..
    Maschan, Alexei
    Fed Res Ctr Paediat Haematol Oncol & Immunol, Moscow, Russia..
    Marsh, Judith
    Kings Coll London, Kings Coll Hosp, Dept Haematol Med, London, England..
    Dufour, Carlo
    Inst G Gaslini, Paediat Haematol, Genoa, Italy..
    Second allogeneic stem cell transplant for aplastic anaemia: a retrospective study by the severe aplastic anaemia working party of the European society for blood and marrow transplantation2015In: British Journal of Haematology, ISSN 0007-1048, E-ISSN 1365-2141, Vol. 171, no 4, p. 606-614Article in journal (Refereed)
    Abstract [en]

    We analysed the outcome of a second allogeneic haematopoietic stem cell transplant (alloHSCT) in 162 patients reported to the European Society for Blood and Marrow Transplantation between 1998 and 2009. Donor origin was a sibling in 110 and an unrelated donor in 52 transplants, respectively. The stem cell source was bone marrow in 31% and peripheral blood in 69% of transplants. The same donor as for the first alloHSCT was used in 81% of transplants whereas a change in the choice of stem cell source was reported in 56% of patients, mainly from bone marrow to peripheral blood. Neutrophil and platelet engraftment occurred in 85% and 72% of patients, after a median time of 15 and 17days, respectively. Grade II-IV acute graft-versus-host disease (GVHD) and chronic GVHD occurred in 21% and 37% of patients, respectively. Graft failure (GF) occurred in 42 patients (26%). After a median follow-up of 3.5years, the 5-year overall survival (OS) was 60.7%. In multivariate analysis, the only factor significantly associated with a better outcome was a Karnofsky/Lansky score 80 (higher OS). We conclude that a second alloHSCT is feasible rescue option for GF in SAA, with a successful outcome in 60% of cases.

  • 6. Daikeler, Thomas
    et al.
    Labopin, Myriam
    Ruggeri, Annalisa
    Crotta, Alessandro
    Abinun, Mario
    Hussein, Ayad Ahmed
    Carlson, Kristina
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Cornillon, Jerome
    Diez-Martin, Jose L.
    Gandemer, Virginie
    Faraci, Maura
    Lindemans, Caroline
    O'Meara, Anne
    Mialou, Valerie
    Renard, Marleen
    Sedlacek, Petr
    Sirvent, Anne
    Socie, Gerard
    Sora, Federica
    Varotto, Stefania
    Sanz, Jaime
    Voswinkel, Jan
    Vora, Ajay
    Yesilipek, M. Akif
    Herr, Andree-Laure
    Gluckman, Eliane
    Farge, Dominique
    Rocha, Vanderson
    New autoimmune diseases after cord blood transplantation: a retrospective study of EUROCORD and the Autoimmune Disease Working Party of the European Group for Blood and Marrow Transplantation2013In: Blood, ISSN 0006-4971, E-ISSN 1528-0020, Vol. 121, no 6, p. 1059-1064Article in journal (Refereed)
    Abstract [en]

    To describe the incidence, risk factors, and treatment of autoimmune diseases (ADs) occurring after cord blood transplantation (CBT), we analyzed both CBT recipients reported to EUROCORD who had developed at least 1 new AD and those who had not. Fifty-two of 726 reported patients developed at least 1 AD within 212 days (range, 27-4267) after CBT. Cumulative incidence of ADs after CBT was 5.0% +/- 1% at 1 year and 6.6% +/- 1% at 5 years. Patients developing ADs were younger and had more nonmalignant diseases (P < .001). ADs target hematopoietic (autoimmune hemolytic anemia, n = 20; Evans syndrome, n = 9; autoimmune thrombocytopenia, n = 11; and immune neutropenia, n = 1) and other tissues (thyroiditis, n = 3; psoriasis, n = 2; Graves disease, n = 1; membranous glomerulonephritis, n = 2; rheumatoid arthritis, n = 1; ulcerative colitis, n = 1; and systemic lupus erythematosus, n = 1). Four patients developed 2 ADs (3 cases of immune thrombocytopenia followed by autoimmune hemolytic anemia and 1 Evans syndrome with rheumatoid arthritis). By multivariate analysis, the main risk factor for developing an AD was nonmalignant disease as an indication for CBT (P = .0001). Hematologic ADs were most often treated with steroids, rituximab, and cyclosporine. With a median follow-up of 26 months (range, 2-91), 6 of 52 patients died as a consequence of ADs. We conclude that CBT may be followed by potentially life-threatening, mainly hematologic ADs.

  • 7.
    Eshoj, Henrik Rode
    et al.
    Odense Univ Hosp, Dept Hematol, Qual Life Res Ctr, Odense, Denmark; Odense Univ Hosp, OPEN Odense Patient Data Explorat Network, Odense, Denmark.
    Nielsen, Lene Kongsgaard
    Odense Univ Hosp, Dept Hematol, Qual Life Res Ctr, Odense, Denmark.
    Schjesvold, Fredrik
    Oslo Univ Hosp, Dept Hematol, Oslo, Norway.
    Abildgaard, Niels
    Odense Univ Hosp, Dept Hematol, Qual Life Res Ctr, Odense, Denmark.
    Nahi, Hareth
    Karolinska Univ Hosp, Dept Hematol, Stockholm, Sweden.
    Andersen, Niels Frost
    Aarhus Univ Hosp, Dept Hematol, Aarhus, Denmark.
    Vangsted, Annette Juul
    Rigshosp, Dept Hematol, Copenhagen, Denmark.
    Helleberg, Carsten
    Herlev Hosp, Dept Hematol, Herlev, Denmark.
    Frølund, Ulf Christian
    Zealand Univ Hosp, Dept Hematol, Roskilde, Denmark.
    Axelsson, Per
    Helsingborg Hosp, Dept Hematol, Helsingborg, Sweden.
    Stromberg, Olga
    Karolinska Univ Hosp, Dept Hematol, Stockholm, Sweden.
    Blimark, Cecilie
    Sahlgrens Univ Hosp, Dept Hematol, Gothenburg, Sweden.
    Carlson, Kristina
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Waage, Anders
    St Olays Hosp NTNU, Dept Hematol, Trondheim, Norway.
    Remes, Kari
    Turku Univ Hosp, Dept Hematol, Turku, Finland.
    Peceliunas, Valdas
    Vilnius Univ Hosp, Dept Hematol, Vilnius, Lithuania.
    Guldbrandsen, Nina
    Oslo Univ Hosp, Dept Hematol, Oslo, Norway.
    Hansson, Markus
    Skane Univ Hosp, Dept Hematol, Lund, Sweden.
    Gregersen, Henrik
    Aalborg Univ Hosp, Dept Hematol, Aalborg, Denmark.
    Health-related quality of life in multiple myeloma patients with first relapse treated with Carfilzomib-based re-induction and salvage autologous stem cell transplantation: data from a Nordic phase II trial2018In: Quality of Life Research, ISSN 0962-9343, E-ISSN 1573-2649, Vol. 27, no Supplement 1, p. S137-S137Article in journal (Other academic)
  • 8. 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.

  • 9. 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.

  • 10.
    Kero, Tanja
    et al.
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Surgical Sciences, Radiology.
    Sorensen, Jens
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Surgical Sciences, Radiology.
    Antoni, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Pharmacy, Department of Medicinal Chemistry, Preparative Medicinal Chemistry.
    Wilking, Helena
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Surgical Sciences, Radiology.
    Carlson, Kristina
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Vedin, Ola
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Cardiology.
    Rosengren, Sara
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Wikstrom, Gerhard
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Cardiology.
    Lubberink, Mark
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Surgical Sciences, Radiology.
    Quantification of (11)C-PIB kinetics in cardiac amyloidosis2018In: Journal of Nuclear Cardiology, ISSN 1071-3581, EISSN 1532-6551Article in journal (Refereed)
    Abstract [en]

    BACKGROUND: The purpose of this work was to determine the optimal tracer kinetic model of (11)C-PIB and to validate the use of the simplified methods retention index (RI) and standardized uptake value (SUV) for quantification of cardiac (11)C-PIB uptake in amyloidosis. METHODS AND RESULTS: Single-tissue, reversible and irreversible two-tissue models were fitted to data from seven cardiac amyloidosis patients who underwent (11)C-PIB PET scans and arterial blood sampling for measurement of blood radioactivity and metabolites. The irreversible two-tissue model (2Tirr) best described cardiac (11)C-PIB uptake. RI and SUV showed high correlation with the rate of irreversible binding (Ki) from the 2Tirr model (r(2 )=0.95 and r(2 )=0.94). Retrospective data from 10 amyloidosis patients and 5 healthy controls were analyzed using RI, SUV, as well as compartment modelling with a population-average metabolite correction. All measures were higher in amyloidosis patients than in healthy controls (p=.001), but with an overlap between groups for Ki. CONCLUSION: An irreversible two-tissue model best describes the (11)C-PIB uptake in cardiac amyloidosis. RI and SUV correlate well with Ki from the 2Tirr model. RI and SUV discriminate better between amyloidosis patients and controls than Ki based on population-average metabolite correction.

  • 11.
    Machowcz, Rafal
    et al.
    Med Univ Warsaw, Warsaw, Poland..
    Suarez, Felipe
    Hop Necker Enfants Malad, Paris, France..
    Wiktor-Jedrzejczak, Wieslaw
    Med Univ Warsaw, Warsaw, Poland..
    Eikema, Diderik-Jan
    LUMC, Dept Med Stat & Bioinformat, Leiden, Netherlands..
    de Wreede, Liesbeth
    LUMC, Dept Med Stat & Bioinformat, Leiden, Netherlands..
    Blok, Henric-Jan
    LUMC, Dept Med Stat & Bioinformat, Leiden, Netherlands..
    Isaksson, Cecilia
    Umea Univ Hosp, Umea, Sweden..
    Poire, Xavier
    Clin Univ St Luc, Brussels, Belgium..
    Nikolousis, Manos
    Birmingham Heartlands Hosp, Birmingham, W Midlands, England..
    Kobbe, Guido
    Heinrich Heine Univ, Dusseldorf, Germany..
    Einsele, Herman
    Univ Klinikum Wurzburg, Wurzburg, Germany..
    Arnold, Renate
    Charite Univ Med Berlin, Berlin, Germany..
    Bonifazi, Francesca
    St Orsola Hosp, Bologna, Italy..
    McQuacker, Grant
    Gartnaval Gen Hosp, Glasgow, Lanark, Scotland..
    Lenhoff, Stig
    Skanes Univ Hosp, Lund, Sweden..
    Rohrlich, Pierre-Simon
    Hop ARCHET I, CHU Nice, Nice, France..
    Theobald, Matthias
    Univ Med Ctr Mainz, Mainz, Germany..
    Ljungman, Per
    Karolinska Univ Hosp, Stockholm, Sweden..
    Schaap, Nicolaas
    Radboud Univ Nijmegen, Med Ctr, Nijmegen, Netherlands..
    Collin, Matthew
    Freeman Rd Hosp, Newcastle Upon Tyne, Tyne & Wear, England..
    Albert, Michael
    Haunersches Kinderspital, Munich, Germany..
    Finke, Juergen
    Univ Freiburg, Freiburg, Germany..
    Ehninger, Gerhard
    Univ Klinikum Dresden, Dresden, Germany..
    Carlson, Kristina
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Halaburda, Kazimierz
    Inst Hematol & Transfus Med, Warsaw, Poland..
    Johansson, Jan-Erik
    Sahlgrens Univ Hosp, Gothenburg, Sweden..
    Zecca, Marco
    Fdn IRCCS Policlin San Matteo, Pavia, Italy..
    Diez-Martin, J.
    Hosp Gregorio Maranon, Madrid, Spain..
    Lehmberg, Kai
    Univ Hosp Eppendorf, Hamburg, Germany..
    Schoenland, Stefan
    Heidelberg Univ, Heidelberg, Germany..
    Lankester, Arjan
    Leiden Univ Hosp, Leiden, Netherlands..
    Gennery, Andrew
    Newcastle Univ, Inst Cellular Med, Newcastle Upon Tyne, Tyne & Wear, England..
    Kroger, Nicolaus
    Univ Hosp Eppendorf, Hamburg, Germany..
    Allogeneic Hematopoietic Stem Cell Transplantation Provides Cure for Adult Patients with Hemophagocytic Lymphohistiocytosis (HLH): A Retrospective Study of The Chronic Malignancies and Inborn ErrorsWorking Parties (CMWP and IEWP) of The EBMT2017In: Pediatric Blood & Cancer, ISSN 1545-5009, E-ISSN 1545-5017, Vol. 64, no S2, p. S23-S24Article in journal (Other academic)
  • 12.
    McLornan, Donal P.
    et al.
    Kings Coll London, Comprehens Canc Ctr, Div Haematol, London, England.
    Szydlo, Richard
    Hammersmith Hosp NHS Fdn Trust, London, England.
    Robin, Marie
    Hop St Louis, AP HP, Serv Hematol Greffe, Paris, France.
    van Biezen, Anja
    Univ Med Ctr, EBMT Data Off, Leiden, Netherlands.
    Koster, Linda
    Univ Med Ctr, EBMT Data Off, Leiden, Netherlands.
    Blok, Henrik J. P.
    Univ Med Ctr, EBMT Data Off, Leiden, Netherlands.
    Van Lint, Maria T.
    Osped San Martino Genova, Genoa, Italy.
    Finke, Juergen
    Univ Freiburg, Freiburg, Germany.
    Vitek, Antonin
    Inst Hematol & Blood Transfus, Prague, Czech Republic.
    Carlson, Kristina
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Griskevicius, Laimonas
    Vilnius Univ Hosp Santaros Klin, Haematol Oncol Transfus Ctr, Vilnius, Lithuania.
    Holler, Ernst
    Univ Regensburg, Dept Haematol & Oncol, Regensburg, Germany.
    Itala-Remes, Maija
    Turku Univ Hosp, Stem Cell Transplant Unit, Turku, Finland.
    Schaap, Michel
    Radboud Univ Nijmegen, Dept Haematol, Med Ctr, Nijmegen, Netherlands.
    Socie, Gerard
    Bay, Jacques-Olivier
    CHU ESTAING, Serv Hematol Clin Adulte & Pediat, Clermont, France.
    Beguin, Yves
    Univ Liege, Dept Haematol, Liege, Belgium.
    Bruno, Benedetto
    SSCVD Trapianto Cellule Staminali, AOU Citta Salute Sci Torino, Turin, Italy.
    Cornelissen, Jan J.
    Univ Med Ctr, Erasmus MC Canc Inst, Rotterdam, Netherlands.
    Gedde-Dahl, Tobias
    Oslo Univ Hosp, Rikshosp, Clin Canc Surg & Transplantat, Oslo, Norway.
    Ljungman, Per
    Karolinska Univ Hosp, Dept Haematol, Stockholm, Sweden.
    Rubio, Marie T.
    Hop Enfants, Dept Haematol, Nancy, France.
    Yakoub-Agha, Ibrahim
    Univ Lille, INSERM U995, CHU Lille, Lille, France.
    Klyuchnikov, Evgeny
    Univ Hosp Eppendorf, Hamburg, Germany.
    Olavarria, Eduardo
    Hammersmith Hosp NHS Fdn Trust, London, England.
    Chalandon, Yves
    Geneva Univ Hosp, Div Haematol, Geneva, Switzerland.
    Kroeger, Nicolaus
    Univ Hosp Eppendorf, Hamburg, Germany.
    Outcome of patients with Myelofibrosis relapsing after allogeneic stem cell transplant: a retrospective study by the Chronic Malignancies Working Party of EBMT2018In: British Journal of Haematology, ISSN 0007-1048, E-ISSN 1365-2141, Vol. 182, no 3, p. 418-422Article in journal (Refereed)
    Abstract [en]

    Allogeneic Haematopoietic Stem Cell Transplant (allo-HSCT) remains the only curative approach for Myelofibrosis (MF). Scarce information exists in the literature on the outcome and, indeed, management of those MF patients who relapse following transplant. We hereby report on the management and outcome of 202 patients who relapsed post allo-HSCT for MF.

  • 13. Mellqvist, Ulf-Henrik
    et al.
    Gimsing, Peter
    Hjertner, Oyvind
    Lenhoff, Stig
    Laane, Edward
    Remes, Kari
    Steingrimsdottir, Hlif
    Abildgaard, Niels
    Ahlberg, Lucia
    Blimark, Cecilie
    Dahl, Inger Marie
    Forsberg, Karin
    Gedde-Dahl, Tobias
    Gregersen, Henrik
    Gruber, Astrid
    Guldbrandsen, Nina
    Haukås, Einar
    Carlson, Kristina
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Kvam, Ann Kristin
    Nahi, Hareth
    Lindås, Roald
    Andersen, Niels Frost
    Turesson, Ingemar
    Waage, Anders
    Westin, Jan
    Bortezomib consolidation after autologous stem cell transplantation in multiple myeloma: a Nordic Myeloma Study Group randomized phase 3 trial2013In: Blood, ISSN 0006-4971, E-ISSN 1528-0020, Vol. 121, no 23, p. 4647-4654Article in journal (Refereed)
    Abstract [en]

    The Nordic Myeloma Study Group conducted an open randomized trial to compare bortezomib as consolidation therapy given after high-dose therapy and autologous stem cell transplantation (ASCT) with no consolidation in bortezomib-naive patients with newly diagnosed multiple myeloma. Overall, 370 patients were centrally randomly assigned 3 months after ASCT to receive 20 doses of bortezomib given during 21 weeks or no consolidation. The hypothesis was that consolidation therapy would prolong progression-free survival (PFS). The PFS after randomization was 27 months for the bortezomib group compared with 20 months for the control group (P = .05). Fifty-one of 90 patients in the treatment group compared with 32 of 90 controls improved their response after randomization (P = .007). No difference in overall survival was seen. Fatigue was reported more commonly by the bortezomib-treated patients in self-reported quality-of-life (QOL) questionnaires, whereas no other major differences in QOL were recorded between the groups. Consolidation therapy seemed to be beneficial for patients not achieving at least a very good partial response (VGPR) but not for patients in the ≥ VGPR category at randomization. Consolidation with bortezomib after ASCT in bortezomib-naive patients improves PFS without interfering with QOL. This trial was registered at www.clinicaltrials.gov as #NCT00417911.

  • 14.
    Nahi, Hareth
    et al.
    Karolinska Inst, Hematol, Stockholm, Sweden..
    Genell, Anna
    Reg Canc Ctr West, Gothenburg, Sweden..
    Walinder, Goran
    Karolinska Inst, Hematol, Stockholm, Sweden..
    Uttervall, Katarina
    Karolinska Inst, Hematol, Stockholm, Sweden..
    Juliusson, Gunnar
    Lund Univ, Hematol, Lund, Sweden..
    Karin, Forsberg
    Umea Univ Hosp, Hematol, Umea, Sweden..
    Hansson, Markus
    Lund Univ, Hematol, Lund, Sweden..
    Svensson, Ronald
    Linkoping Univ Hosp, Hematol, Linkoping, Sweden..
    Linder, Olle
    Orebro Univ Hosp, Hematol, Orebro, Sweden..
    Carlson, Kristina
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Bjorkstrand, Bo
    Karolinska Inst, Hematol, Stockholm, Sweden..
    Kristinsson, Sigurdur Y.
    Karolinska Inst, Hematol, Stockholm, Sweden..
    Mellqvist, Ulf Henrik
    South Elvsborg Hosp, Hematol, Boras, Sweden..
    Blimark, Cecilie
    Sahlgrens Univ Hosp, Hematol, Gothenburg, Sweden..
    Turesson, Ingemar
    Skane Univ Hosp, Hematol, Malmo, Sweden..
    Incidence, characteristics, and outcome of solitary plasmacytoma and plasma cell leukemia. Population-based data from the Swedish Myeloma Register2017In: European Journal of Haematology, ISSN 0902-4441, E-ISSN 1600-0609, Vol. 99, no 3, p. 216-222Article in journal (Refereed)
    Abstract [en]

    Solitary plasmacytoma (SP) and plasma cell leukemia (PCL) are uncommon (3-6%) types of plasma cell disease. The risk of progression to symptomatic multiple myeloma (MM) is probably important for the outcome of SP. PCL is rare and has a dismal outcome. In this study, we report on incidence and survival in PCL/SP, and progression to MM in SP, using the prospective observational Swedish Multiple Myeloma Register designed to document all newly diagnosed plasma cell diseases in Sweden since 2008. Both solitary bone plasmacytoma (SBP) (n=124) and extramedullary plasmacytoma (EMP) (n=67) have better overall survival (OS) than MM (n=3549). Progression to MM was higher in SBP than in EMP (35% and 7% at 2years, respectively), but this did not translate into better survival in EMP. In spite of treatment developments, the OS of primary PCL is still dismal (median of 11months, 0% at 5years). Hence, there is a great need for diagnostic and treatment guidelines as well as prospective studies addressing the role for alternative treatment options, such as allogeneic stem cell transplantation and monoclonal antibodies in the treatment of PCL.

  • 15.
    Raj, K.
    et al.
    GKT Sch Med, London, England.
    Olavarria, E.
    Hammersmith Hosp, London, England.
    Eikema, D-J
    Dept Med Stat & Bioinoformat, Leiden, Netherlands.
    Blok, H-J
    Dept Med Stat & Bioinoformat, Leiden, Netherlands.
    Bregante, S.
    Osped San Martino Genova, Genoa, Italy.
    Ciceri, F.
    Osped San Raffaele Srl, Segrate, Italy.
    Passweg, J.
    Univ Hosp, Basel, Switzerland.
    Ljungmann, P.
    Karolinska Univ Hosp, Stockholm, Sweden.
    Schaap, M.
    Radboud Univ Nijmegen, Nijmegen Med Ctr, Nijmegen, Netherlands.
    Carlson, Kristina
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Zuckerman, T.
    Rambam Med Ctr, Haifa, Israel.
    Volin, L.
    HUCH Comprehens Canc Ctr, Helsinki, Finland.
    Koc, Yener
    Med Pk Hosp, Antalya, Turkey.
    Diez-Martin, J.
    Hosp Gregorio Maranon, Madrid, Spain.
    Brossart, P.
    Univ Bonn, Bonn, Germany.
    Blaise, D.
    Inst Paoli Calmettes, Marseille, France.
    Natale, A.
    Osped Civile, Pescara, Italy.
    Vitek, A.
    Inst Hematol & Blood Transfus, Prague, Czech Republic.
    Mclornan, D.
    GKT Sch Med, London, England.
    Robin, M.
    Hop St Louis, Paris, France.
    Chalandon, Y.
    Hop Univ Geneve, Geneva, Switzerland.
    Kroger, N.
    Univ Hosp Eppendorf, Hamburg, Germany.
    Family mismatched allogeneic stem cell transplantationfor myelofibrosis: Report from the chronic malignancies working party of EBMT2017In: Bone Marrow Transplantation, ISSN 0268-3369, E-ISSN 1476-5365, Vol. 52, no Supplement: 1, p. S182-S183Article in journal (Other academic)
  • 16.
    Raj, Kavita
    et al.
    Guys & St Thomas NHS Fdn Trust, Dept Haematol Med, London, England.
    Eikema, Diderik-Jan
    EBMT Stat Unit, Dept Med Stat & Bioinformat, Leiden, Netherlands.
    McLornanl, Donal P.
    Guys & St Thomas NHS Fdn Trust, Dept Haematol Med, London, England.
    Olavarria, Eduardo
    Hammersmith Hosp London, Dept Haematol Med, London, England.
    Bloke, Henric-Jan
    EBMT Stat Unit, Dept Med Stat & Bioinformat, Leiden, Netherlands.
    Bregante, Stefania
    Osped San Martino Genova, Dept Haematol, Genoa, Italy.
    Ciceri, Fabio
    Carattere Sci San Raffaele Sci Inst, Dept Hematol & Hematopoiet, Milan, Italy.
    Passweg, Jakob
    Univ Hosp, Dept Internal Med, Basel, Switzerland.
    Ljungman, Per
    Karolinska Univ Hosp, Div Hematol, Stockholm, Sweden.
    Schaap, Nicolaas
    Radboud Univ Nijmegen, Med Ctr, Dept Hematol, Nijmegen, Netherlands.
    Carlson, Kristina
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Zuckerman, Tsila
    Rambam Med Ctr, Depatment Hematol & Bone Marrow Transplantat, Haifa, Israel.
    de Wreede, Liesbeth C.
    Deutsch Knochenmarkspenderdatei Clin Trials Unit, Dresden, Germany.
    Volin, Liisa
    Helsinki Univ Cent Hosp, Ctr Comprehens Canc, Dept Med, Helsinki, Finland.
    Koc, Yener
    Med Pk Hosp, Stem Cell Transplant Unit, Dept Oncol, Antalya, Turkey.
    Luis Diez-Martin, Jose
    Univ Complutense Med, Inst Invest Sanitaria Gregorio Maranon, Dept Hematol, Madrid, Spain.
    Brossart, Peter
    Univ Bonn, Dept Immunooncol & Rheumatol, Bonn, Germany.
    Wolf, Dominik
    Univ Bonn, Dept Immunooncol & Rheumatol, Bonn, Germany.
    Blaise, Didier
    Inst Paoli Calmettes, Transplantat & Cell Therapy Unit, Dept Oncohematol, Marseille, France.
    Di Bartolomeo, Paolo
    Osped Civile, Dept Hematol Transfus Med & Biotechnol, Marseille, France.
    Vitek, Antonin
    Inst Hematol & Blood Transfus, Prague, Czech Republic.
    Robin, Marie
    Hop St Louis, Dept Hematol & Bone Marrow Transplantat, Paris, France.
    Yakoub-Agha, Ibrahim
    Univ Lille, Lille Univ Hosp, INSERM U995, Dept Hematol, Lille, France.
    Chalandon, Yves
    Univ Geneva, Hematol Div, Hop Univ Geneve, Geneva, Switzerland.
    Kroger, Nicolaus
    Univ Hosp Eppendorf, Dept Stem Cell Transplantat, Hamburg, Germany.
    Family Mismatched Allogeneic Stem Cell Transplantation for Myelofibrosis: Report from the Chronic Malignancies Working Party of European Society for Blood and Marrow Transplantation2019In: Biology of blood and marrow transplantation, ISSN 1083-8791, E-ISSN 1523-6536, Vol. 25, no 3, p. 522-528Article in journal (Refereed)
    Abstract [en]

    This analysis included 56 myelofibrosis (MF) patients transplanted from family mismatched donor between 2009 and 2015 enrolled in the European Society for Blood and Marrow Transplantation database. The median age was 57 years (range, 38 to 72); 75% had primary MF and 25% had secondary MF. JAK2 V617F was mutated in 61%. Donors were HLA mismatched at 2 or more loci. Stem cells were sourced from bone marrow in 66% and peripheral blood in 34%. The median CD34(+) cell dose was 4.8 x 10(6)/kg (range, 1.7 to 22.9; n = 43). Conditioning was predominantly myeloablative in 70% and reduced intensity in the remainder. Regimens were heterogeneous with thiotepa, busulfan, fludarabine, and post-transplant cyclophosphamide used in 59%. The incidence of neutrophil engraftment by 28 days was 82% (range, 70% to 93%), at a median of 21 days (range, 19 to 23). At 2 years the cumulative incidence of primary graft failure was 9% (95% CI 1% to 16%) and secondary graft failure was 13% (95% CI 4% to 22%). The cumulative incidence of acute graft-versus-host disease (GVHD) grades II to IV and Ill to IV was 28% (95% CI 16% to 40%) and 9% (95% CI 2% to 17%) at 100 days. The cumulative incidence of chronic GVHD at 1 year was 45% (95% CI 32% to 58%), but the cumulative incidence of death without chronic GVHD by 1 year was 20% (95% CI 10% to 31%). With a median follow-up of 32 months, the 1- and 2-year overall survival was 61% (95% CI 48% to 74%) and 56% (95% CI 41% to 70%), respectively. The 1- and 2- year progression-free survival was 58% (95% CI 45% to 71%) and 43% (95% CI 28% to 58%), respectively, with a 2-year cumulative incidence of relapse of 19% 95% CI 7% to 31%). The 2-year nonrelapse mortality was 38% (95% CI 24% to 51%). This retrospective study of MF allo-SCT using family mismatched donors demonstrated feasibility of the approach, timely neutrophil engraftment in over 80% of cases, and acceptable overall and progression-free survival rates with relapse rates not dissimilar to the unrelated donor setting. However, strategies to minimize the risk of graft failure and the relatively high nonrelapse mortality need to be used, ideally in a multicenter prospective fashion.

  • 17.
    Rosengren, Sara
    et al.
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Mellqvist, U-H
    South Elvsborg Hosp, Dept Hematol, Boras, Sweden.
    Nahi, H
    Karolinska Inst, Dept Hematol, Stockholm, Sweden.
    Forsberg, K
    Norrlands Univ Hosp, Dept Hematol, Umeå, Sweden.
    Lenhoff, S
    Skåne Univ Hosp, Dept Hematol, Lund, Sweden.
    Strömberg, O
    Karolinska Inst, Dept Hematol, Stockholm, Sweden.
    Ahlberg, L
    Linköping Univ Hosp, Dept Hematol, Linköping, Sweden.
    Linder, O
    Örebro Univ Hosp, Dept Hematol, Örebro, Sweden.
    Carlson, Kristina
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Outcome of AL amyloidosis after high-dose melphalan and autologous stem cell transplantation in Sweden, long-term results from all patients treated in 1994-2009.2016In: Bone Marrow Transplantation, ISSN 0268-3369, E-ISSN 1476-5365, Vol. 51, no 12, p. 1569-1572Article in journal (Refereed)
    Abstract [en]

    High-dose melphalan and autologous stem cell transplantation (HDM/ASCT) is widely used in immunoglobulin light chain (AL) amyloidosis, but the benefit is debated mainly because of the high treatment-related mortality (24% in a randomised study comparing HDM/ASCT with oral melphalan/dexamethasone). We report here on the long-term outcome of all patients treated with HDM/ASCT for AL amyloidosis in Sweden between 1994 and 2009. Seventy-two patients were treated at eight Swedish centres. Median follow-up was 67.5 months. At least partial response (organ or haematological) was seen in 64% of the patients. Median overall survival was 98 months or 8.2 years, with 5-year survival 63.9% and 10-year survival 43.4%. In patients with cardiac involvement or multiple organ involvement, survival was significantly shorter, median overall survival 49 and 56 months, respectively. All mortality within 100 days from ASCT was 12.5% for all patients and 17.2% in the patients with cardiac involvement. For patients treated in the earlier time period (1994-2001), 100-day mortality was 23.8% compared with 7.8% in the later period (2002-2009). In conclusion, long survival times can be achieved in patients with AL amyloidosis treated with HDM/ASCT, also in smaller centres. Early mortality is high, but with a decreasing trend over time.

  • 18.
    Tolf, Andreas
    et al.
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Neuroscience, Landtblom: Neurology.
    Fagius, Jan
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Neuroscience, Landtblom: Neurology.
    Carlson, Kristina
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Åkerfeldt, Torbjörn
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Clinical Chemistry.
    Granberg, Tobias
    Karolinska Inst, Dept Clin Neurosci, Stockholm, Sweden; Karolinska Univ Hosp, Dept Radiol, Div Neuroradiol, Stockholm, Sweden.
    Larsson, Elna-Marie
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Surgical Sciences, Radiology.
    Burman, Joachim
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Neuroscience, Landtblom: Neurology.
    Sustained remission in multiple sclerosis after hematopoietic stem cell transplantation2019In: Acta Neurologica Scandinavica, ISSN 0001-6314, E-ISSN 1600-0404, Vol. 140, no 5, p. 320-327Article in journal (Refereed)
    Abstract [en]

    Objectives: To determine whether treatment with autologous hematopoietic stem cell transplantation (HSCT) can induce sustained complete remission in patients with multiple sclerosis (MS).

    Material and methods: Case series of patients with relapsing‐remitting MS (n = 10) treated at a single center between 2004 and 2007 and followed up for 10 years. The patients were treated with a BEAM/ATG conditioning regimen (n = 9) or a cyclophosphamide/ATG conditioning regimen (n = 1) followed by infusion of unmanipulated autologous hematopoietic stem cells. The primary endpoint was sustained complete remission. Sustained complete remission was defined as “no evidence of disease activity‐4,” sustained for a period of at least 5 years without any ongoing disease‐modifying treatment. Furthermore, MS was considered as “resolved” if intrathecal IgG production and cerebrospinal fluid neurofilament light levels were normalized as well.

    Results: Five out of 10 patients were in sustained complete remission at the end of the study. In three of them, MS was resolved.

    Conclusions: Our data demonstrate that sustained complete remission after autologous HSCT for MS is possible.

     

  • 19.
    Vaht, Krista
    et al.
    Sahlgrens Univ Hosp, Sect Hematol & Coagulat, Gothenburg, Sweden;Gothenburg Univ, Inst Med, Sahlgrenska Acad, Gothenburg, Sweden.
    Goransson, Magnus
    Sahlgrens Univ Hosp, Queen Silvia Childrens Hosp, Dept Pediat, Gothenburg, Sweden.
    Carlson, Kristina
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Isaksson, Cecilia
    Univ Hosp, Canc Ctr, Dept Hematol, Umea, Sweden.
    Lenhoff, Stig
    Lund Univ, Skane Univ Hosp, Dept Hematol, Lund, Sweden.
    Sandstedt, Anna
    Linkoping Univ Hosp, Dept Hematol, Linkoping, Sweden.
    Uggla, Bertil
    Orebro Univ, Fac Med & Hlth, Sect Hematol, Dept Med, Orebro, Sweden.
    Winiarski, Jacek
    Karolinska Univ Hosp, Astrid Lindgren Childrens Hosp, Huddinge, Sweden;Karolinska Inst, CLINTEC, Stockholm, Sweden.
    Ljungman, Per
    Karolinska Univ Hosp, Ctr Allogene Stem Cell Transplantat Unit CAST, Stockholm, Sweden;Karolinska Inst, Dept Med, Stockholm, Sweden.
    Andersson, Per-Ola
    Gothenburg Univ, Inst Med, Sahlgrenska Acad, Gothenburg, Sweden;Sodra Alvsborg Hosp Boras, Dept Med, Boras, Sweden.
    Brune, Mats
    Sahlgrens Univ Hosp, Sect Hematol & Coagulat, Gothenburg, Sweden;Gothenburg Univ, Inst Med, Sahlgrenska Acad, Gothenburg, Sweden.
    High Graft-versus-Host Disease-Free, Relapse/Rejection-Free Survival and Similar Outcome of Related and Unrelated Allogeneic Stem Cell Transplantation for Aplastic Anemia: A Nationwide Swedish Cohort Study2019In: Biology of blood and marrow transplantation, ISSN 1083-8791, E-ISSN 1523-6536, Vol. 25, no 10, p. 1970-1974Article in journal (Refereed)
    Abstract [en]

    Allogeneic stem cell transplantation (SCT) as primary treatment for aplastic anemia (AA) is being increasingly used. Yet, age, stem cell source, and donor type are important outcome factors. We have recently performed a nationwide cohort study of all patients with AA in Sweden diagnosed from 2000 to 2011 and now present outcome data on SCT patients. In total, 68 patients underwent SCT, and 63% of them had failed immunosuppressive therapy. We found that, with a median follow-up of 109 months (range, 35 to 192 months), 5-year overall survival (OS) for all patients was 86.8%, whereas graft-versus-host disease-free, relapse/rejection-free survival (GRFS) at 5 years was 69.1%. There was no survival impact regarding the donor type or stem cell source. Patients aged >= 40 years had a higher transplant-related mortality (29.4% versus 7.8%; P= .023), which translated into a lower 5-year OS: 70.6% versus 92.2% (A=.022) and a trend of lower GRFS (52.9% versus 74.5%; P = .069). In conclusion, we found in this real-world setting that both OS and GRFS were high, but SCT for patients with AA aged >= 40 years is problematic, and clinical trials addressing this issue are warranted. (C) 2019 American Society for Transplantation and Cellular Therapy. Published by Elsevier Inc.

  • 20.
    Vaht, Krista
    et al.
    Sahlgrens Univ Hosp, Sect Hematol & Coagulat, Gothenburg, Sweden.;Gothenburg Univ, Sahlgrenska Acad, Gothenburg, Sweden..
    Goransson, Magnus
    Sahlgrens Univ Hosp, Queen Silvia Childrens Hosp, Dept Pediat, Gothenburg, Sweden..
    Carlson, Kristina
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Isaksson, Cecilia
    Univ Hosp, Dept Hematol, Canc Ctr, Umea, Sweden..
    Lenhoff, Stig
    Lund Univ, Skane Univ Hosp, Dept Hematol, Lund, Sweden..
    Sandstedt, Anna
    Linkoping Univ Hosp, Dept Hematol, Linkoping, Sweden..
    Uggla, Bertil
    Orebro Univ, Fac Med & Hlth, Dept Med, Sect Hematol, Orebro, Sweden..
    Winiarski, Jacek
    Karolinska Univ Hosp, Astrid Lindgren Childrens Hosp, Stockholm, Sweden..
    Ljungman, Per
    Karolinska Univ Hosp Huddinge, Ctr Allogene Stem Cell Transplantat CAST, Stockholm, Sweden..
    Brune, Mats
    Sahlgrens Univ Hosp, Sect Hematol & Coagulat, Gothenburg, Sweden.;Gothenburg Univ, Sahlgrenska Acad, Gothenburg, Sweden..
    Andersson, Per-Ola
    South Alvsborg Hosp Boras, Boras, Sweden.;Gothenburg Univ, Sahlgrenska Acad, Gothenburg, Sweden..
    Incidence and outcome of acquired aplastic anemia: real-world data from patients diagnosed in Sweden from 2000-20112017In: Haematologica, ISSN 0390-6078, E-ISSN 1592-8721, Vol. 102, no 10, p. 1683-1690Article in journal (Refereed)
    Abstract [en]

    A plastic anemia is a rare life-threatening disease. However, since the introduction of immunosuppressive therapy and allogeneic stem cell transplantation, the outcome has improved considerably, and the 5-year survival is reported to be 70-80% in selected patient cohorts. Yet, contemporary population-based data on incidence and survival are lacking. We performed a national retrospective study to determine the incidence, treatment, and survival of patients with aplastic anemia diagnosed in Sweden from 2000-2011. Patients were included via the National Patient Registry, and diagnosed according to the Camitta criteria. In total, 257 confirmed cases were identified, with an overall incidence of 2.35 (95% CI: 2.06-2.64) cases per million inhabitants per year. Median age was 60 years (range: 2-92), and median follow up was 76 (0-193) months. Primary treatments included immunosuppressive therapy (63%), allogenic stem cell transplantation (10%), or single-agent cyclosporine/no specific therapy (27%). The 5-year survival was 90.7% in patients aged 0-18 years, 90.5% in patients aged 19-39 years, 70.7% in patients aged 40-59 years, and 38.1% in patients aged >= 60 years. Multivariate analysis showed that age (both 40-59 and >= 60 age groups), very severe aplastic anemia and single-agent cyclosporine/no specific therapy were independent risk factors for inferior survival. In conclusion, younger aplastic anemia patients experience a very good long-term survival, while that of patients >= 60 years in particular remains poor. Apparently, the challenge today is to improve the management of older aplastic anemia patients, and prospective studies to address this medical need are warranted.

  • 21.
    Vaht, Krista
    et al.
    Sahlgrens Univ Hosp, Sect Haematol & Coagulat, Gothenburg, Sweden;Gothenburg Univ, Sahlgrenska Acad, Inst Med, Gothenburg, Sweden.
    Goransson, Magnus
    Sahlgrens Univ Hosp, Dept Pediat, Queen Silvia Childrens Hosp, Gothenburg, Sweden.
    Carlson, Kristina
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Isaksson, Cecilia
    Univ Hosp, Dept Haematol, Ctr Canc, Umea, Sweden.
    Lenhoff, Stig
    Lund Univ, Dept Haematol, Skane Univ Hosp, Lund, Sweden.
    Sandstedt, Anna
    Linkoping Univ Hosp, Dept Haematol, Linkoping, Sweden.
    Uggla, Bertil
    Orebro Univ, Sect Haematol, Dept Med, Fac Med & Hlth, Orebro, Sweden.
    Winiarski, Jacek
    Karolinska Inst, Karolinska Univ Hosp, Astrid Lindgren Childrens Hosp, Stockholm, Sweden;CLINTEC, Stockholm, Sweden.
    Ljungman, Per
    Karolinska Inst, Karolinska Univ Hosp, Dept Med, Ctr Allogene Stem Cell Transplantat Unit CAST, Stockholm, Sweden.
    Brune, Mats
    Sahlgrens Univ Hosp, Sect Haematol & Coagulat, Gothenburg, Sweden;Gothenburg Univ, Sahlgrenska Acad, Inst Med, Gothenburg, Sweden.
    Andersson, Per-Ola
    Gothenburg Univ, Sahlgrenska Acad, Inst Med, Gothenburg, Sweden;Sodra Alvsborg Hosp Boras, Dept Med, Boras, Sweden.
    Low response rate to ATG-based immunosuppressive therapy in very severe aplastic anaemia A Swedish nationwide cohort study2018In: European Journal of Haematology, ISSN 0902-4441, E-ISSN 1600-0609, Vol. 100, no 6, p. 613-620Article in journal (Refereed)
    Abstract [en]

    ObjectivesAntithymocyte globulin (ATG)-based immunosuppression remains a cornerstone in aplastic anaemia (AA) treatment. However, most ATG studies are not population-based and knowledge about real-world results concerning response and outcome could offer important information for treating physicians. MethodsWe have recently performed a nationwide retrospective cohort study on all AA patients diagnosed in Sweden in 2000-2011 and now present treatment and outcome data on patients receiving first-line ATG. In total, 158 patients showed a 47.0% response rate which was similar in all age groups (range 41.5%-51.7%) with no difference regarding ATG formulation. The response was significantly associated with severity gradeespecially at time of treatment initiation: very severe (VSAA) 22.7%; severe (SAA) 54.5% (P<.001); and non-severe 88.5% (P<.001). A logistic regression-based predictive model indicated that VSAA patients with an absolute reticulocyte count <25x10(9)/L had only a 19% probability of response. In a multivariable analysis, age and VSAA at the time of treatment were the independent factors for inferior survival. ConclusionsReal-world VSAA patients respond poorly to ATG which indicates the need for a different treatment approach. Our findings suggest that age alone should not be a discriminating factor for administering ATG treatment.

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