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  • 1. Abelsson, J
    et al.
    Merup, M
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    WeisBjerrum, O
    Brinch, L
    Brune, M
    Johansson, P
    Kauppila, M
    Lenhoff, S
    Liljeholm, M
    Malm, C
    Remes, K
    Vindelöv, L
    Andréasson, B
    The outcome of allo-HSCT for 92 patients with myelofibrosis in the Nordic countries2012In: Bone Marrow Transplantation, ISSN 0268-3369, E-ISSN 1476-5365, Vol. 47, no 3, p. 380-386Article in journal (Refereed)
    Abstract [en]

    Between 1982 and 2009 a total of 92 patients with myelofibrosis (MF) in chronic phase underwent allo-SCT in nine Nordic transplant centers. Myeloablative conditioning (MAC) was given to 40 patients, and reduced intensity conditioning (RIC) was used in 52 patients. The mean age in the two groups at transplantation was 46±12 and 55±8 years, respectively (P<0.001). When adjustment for age differences was made, the survival of the patients treated with RIC was significantly better (P=0.003). Among the RIC patients, the survival was significantly (P=0.003) better for the patients with age <60 years (a 10-year survival close to 80%) than for the older patients. The type of stem cell donor did not significantly affect the survival. No significant difference was found in TRM at 100 days between the MAC- and the RIC-treated patients. The probability of survival at 5 years was 49% for the MAC-treated patients and 59% in the RIC group (P=0.125). Patients treated with RIC experienced significantly less aGVHD compared with patients treated with MAC (P<0.001). The OS at 5 years was 70, 59 and 41% for patients with Lille score 0, 1 and 2, respectively (P=0.038, when age adjustment was made). Twenty-one percent of the patients in the RIC group were given donor lymphocyte infusion because of incomplete donor chimerism, compared with none of the MAC-treated patients (P<0.002). Nine percent of the patients needed a second transplant because of graft failure, progressive disease or transformation to AML, with no significant difference between the groups. Our conclusions are (1) allo-SCT performed with RIC gives a better survival compared with MAC. (2) age over 60 years is strongly related to a worse outcome and (3) patients with higher Lille score had a shorter survival.

  • 2.
    Barbuil, Tiziano
    et al.
    Osped Giovanni 23, Div Hematol, Bergamo, Italy.
    Tefferi, Ayalew
    Mayo Clin, Dept Med, Div Hematol, Rochester, MN USA.
    Vannucchi, Alessandro M.
    Univ Florence, AOU Careggi, Ctr Res & Innovat Myeloproliferat Neoplasms, CRIMM, Florence, Italy.
    Passamonti, Francesco
    Univ Insubria, Osped Circolo, Dept Med & Surg, Div Hematol,ASST Sette Laghi, Varese, Italy.
    Silvers, Richard T.
    Weill Cornell Med, Div Hematol Oncol, New York, NY USA.
    Hoffman, Ronald
    Mt Sinai Sch Med, Dept Med, Tisch Canc Inst, New York, NY USA.
    Verstovsek, Srdan
    Univ Texas MD Anderson Canc Ctr, Dept Leukemia, Houston, TX 77030 USA.
    Mesa, Ruben
    UT Hlth San Antonio Canc Ctr, San Antonio, TX USA.
    Kiladjian, Jean-Jacques
    Univ Paris 07, Hop St Louis, AP HP, INSERM,Ctr Invest Clin CIC 1427, Paris, France.
    Hehlmann, Rudiger
    Heidelberg Univ, Univ Hosp Mannheim, Dept Hematol & Oncol, Mannheim, Germany.
    Reiter, Andreas
    Heidelberg Univ, Univ Hosp Mannheim, Dept Hematol & Oncol, Mannheim, Germany.
    Cervantes, Francisco
    Univ Barcelona, IDIBAPS, Hosp Clin, Barcelona, Spain.
    Harrison, Claire
    Guys & St Thomas NHS Fdn Trust, Dept Hematol, London, England.
    Mc Mullin, Mary Frances
    Queens Univ, Ctr Med Educ, Belfast, Antrim, North Ireland.
    Hasselbalch, Hans Carl
    Zealand Univ Hosp, Dept Hematol, Roskilde, Denmark.
    Koschmieder, Steffen
    Rhein Westfal TH Aachen, Fac Med, Dept Hematol Oncol Hemostaseol & Stem Cell Transp, Aachen, Germany.
    Marchetti, Monia
    Hosp Cardinal Massaia, Oncol SOC, Hematol Day Serv, Asti, Italy.
    Bacigalupo, Andrea
    Univ Cattolica Sacro Cuore, Fdn Policlin Univ Gemelli, Ist Ematol, Rome, Italy.
    Finazzil, Guido
    Osped Giovanni 23, Div Hematol, Bergamo, Italy.
    Kroeger, Nicolaus
    Univ Hosp Hamburg Eppendorf, Dept Stem Cell Transplantat, Hamburg, Germany.
    Griesshammer, Martin
    Univ Hannover, Acad Hosp, Johannes Wesling Med Ctr Minden, Dept Hematol & Oncol, Minden, Germany.
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Barosi, Giovanni
    IRCCS Policlin S Matteo Fdn, Ctr Study Myelofibrosis, Pavia, Italy.
    Philadelphia chromosome-negative classical myeloproliferative neoplasms: revised management recommendations from European LeukemiaNet2018In: Leukemia, ISSN 0887-6924, E-ISSN 1476-5551, Vol. 32, no 5, p. 1057-1069Article, review/survey (Refereed)
    Abstract [en]

    This document updates the recommendations on the management of Philadelphia chromosome-negative myeloproliferative neoplasms (Ph-neg MPNs) published in 2011 by the European LeukemiaNet (ELN) consortium. Recommendations were produced by multiple-step formalized procedures of group discussion. A critical appraisal of evidence by using Grades of Recommendation, Assessment, Development and Evaluation (GRADE) methodology was performed in the areas where at least one randomized clinical trial was published. Seven randomized controlled trials provided the evidence base; earlier phase trials also informed recommendation development. Key differences from the 2011 diagnostic recommendations included: lower threshold values for hemoglobin and hematocrit and bone marrow examination for diagnosis of polycythemia vera (PV), according to the revised WHO criteria; the search for complementary clonal markers, such as ASXL1, EZH2, IDH1/IDH2, and SRSF2 for the diagnosis of myelofibrosis (MF) in patients who test negative for JAK2V617, CALR or MPL driver mutations. Regarding key differences of therapy recommendations, both recombinant interferon alpha and the JAK1/JAK2 inhibitor ruxolitinib are recommended as second-line therapies for PV patients who are intolerant or have inadequate response to hydroxyurea. Ruxolitinib is recommended as first-line approach for MF-associated splenomegaly in patients with intermediate-2 or high-risk disease; in case of intermediate-1 disease, ruxolitinib is recommended in highly symptomatic splenomegaly. Allogeneic stem cell transplantation is recommended for transplant-eligible MF patients with high or intermediate-2 risk score. Allogeneic stem cell transplantation is also recommended for transplant-eligible MF patients with intermediate-1 risk score who present with either refractory, transfusion-dependent anemia, blasts in peripheral blood > 2%, adverse cytogenetics, or high-risk mutations. In these situations, the transplant procedure should be performed in a controlled setting.

  • 3. Barosi, G
    et al.
    Tefferi, A
    Besses, C
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Cervantes, F
    Finazzi, G
    Gisslinger, H
    Griesshammer, M
    Harrison, C
    Hehlmann, R
    Hermouet, S
    Kiladjian, J-J
    Kröger, N
    Mesa, R
    Mc Mullin, M F
    Pardanani, A
    Passamonti, F
    Samuelsson, J
    Vannucchi, A M
    Reiter, A
    Silver, R T
    Verstovsek, S
    Tognoni, G
    Barbui, T
    Clinical end points for drug treatment trials in BCR-ABL1-negative classic myeloproliferative neoplasms: consensus statements from European LeukemiaNET (ELN) and Internation Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT)2015In: Leukemia, ISSN 0887-6924, E-ISSN 1476-5551, Vol. 29, no 1, p. 20-26Article in journal (Refereed)
    Abstract [en]

    The discovery of somatic mutations, primarily JAK2V617F and CALR, in classic BCR-ABL1-negative myeloproliferative neoplasms (MPNs) has generated interest in the development of molecularly targeted therapies, whose accurate assessment requires a standardized framework. A working group, comprised of members from European LeukemiaNet (ELN) and International Working Group for MPN Research and Treatment (IWG-MRT), prepared consensus-based recommendations regarding trial design, patient selection and definition of relevant end points. Accordingly, a response able to capture the long-term effect of the drug should be selected as the end point of phase II trials aimed at developing new drugs for MPNs. A time-to-event, such as overall survival, or progression-free survival or both, as co-primary end points, should measure efficacy in phase III studies. New drugs should be tested for preventing disease progression in myelofibrosis patients with early disease in randomized studies, and a time to event, such as progression-free or event-free survival should be the primary end point. Phase III trials aimed at preventing vascular events in polycythemia vera and essential thrombocythemia should be based on a selection of the target population based on new prognostic factors, including JAK2 mutation. In conclusion, we recommended a format for clinical trials in MPNs that facilitates communication between academic investigators, regulatory agencies and drug companies.

  • 4. Bennett, Charles L.
    et al.
    McKoy, June M.
    Henke, Michael
    Silver, Samuel M.
    MacDougall, Iain C.
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences.
    Luminari, Stefano
    Casadevall, Nicole
    Schellekens, Huub
    Sartor, Oliver
    Lai, Stephen Y.
    Armitage, James O.
    Reassessments of ESAs for cancer treatment in the US and Europe2010In: Oncology, ISSN 0890-9091, Vol. 24, no 3, p. 260-268Article in journal (Refereed)
    Abstract [en]

    Anemia is a widely prevalent complication among cancer patients. At the time of diagnosis, 30% to 40% of patients with non-Hodgkin lymphoma or Hodgkin lymphoma and up to 70% of patients with multiple myeloma are anemic; rates are higher among persons with myelodysplastic syndromes. Among patients with solid cancers or lymphomas, up to half develop anemia following chemotherapy. For almost 2 decades, erythropoiesis-stimulating agents (ESAs) were the primary treatment for cancer-related anemia. However, reassessments of benefits and risks of ESAs for cancer-associated anemia have occurred internationally. We reviewed guidelines and notifications from regulatory agencies and manufacturers, reimbursement policies, and utilization for ESAs in the cancer and chronic kidney disease settings within the United States, Europe, and Canada. In 2008 the US Food and Drug Administration (FDA) restricted ESAs from cancer patients seeking cure. Reimbursement is limited to hemoglobin levels < 10 g/dL. In the United States, ESA usage increased 340% between 2001 and 2006, and decreased 60% since 2007. The European Medicines Agency (EMEA) recommended that ESA benefits do not outweigh risks. In Europe between 2001 and 2006, ESA use increased 51%; since 2006, use decreased by 10%. In 2009, Canadian manufacturers recommended usage based on patient preferences. In Canada in 2007, approximately 20% of anemic cancer patients received ESAs, a 20% increase since 2004. In contrast to Europe, where ESA use has increased over time, reassessments of ESA-associated safety concerns in the United States have resulted in marked decrements in ESA use among cancer patients.

  • 5.
    Besses, C.
    et al.
    Hosp del Mar IMIM, Serv Hematol, Barcelona, Spain..
    Bello-Lopez, J. L.
    Univ Santiago, Hosp Clin, E-15706 Santiago, Spain..
    De la Serna, J.
    Hosp Univ 12 Octubre, Madrid, Spain..
    Hernandez-Boluda, J. C.
    Hosp Clin Univ, Valencia, Spain..
    Loscertales, J.
    Hosp Univ La Princesa, Madrid, Spain..
    Griesshammer, M.
    Johannes Wesling Med Ctr, Serv Hematol & Oncol, Minden, Germany..
    Gugliotta, L.
    St Orsola Malpighi Hosp, Serv Hematol, Bologna, Italy..
    Harrison, C.
    Guys & St Thomas NHS Fdn Trust, Serv Hematol, London, England..
    Kiladjian, J. J.
    Hop St Louis, APHP, Ctr Invest Clin, Paris, France..
    Hamdani, M.
    Shire Pharmaceut, Global Biometr, Wayne, NJ USA..
    Achenbach, H.
    Shire AG, Res & Dev, Eysins, Switzerland..
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Treatment Of Essential Thrombocythemia In Europe: Observational Study Of 3649 Patients Of High Risk (Exels)2015In: Haematologica, ISSN 0390-6078, E-ISSN 1592-8721, Vol. 100, p. 29-29Article in journal (Other academic)
  • 6. Besses, Carlos
    et al.
    Kiladjian, Jean-Jacques
    Griesshammer, Martin
    Gugliotta, Luigi
    Harrison, Claire
    Coll, Ruth
    Smith, Jonathan
    Abhyankar, Brihad
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Cytoreductive treatment patterns for essential thrombocythemia in Europe. Analysis of 3643 patients in the EXELS study2013In: Leukemia research: a Forum for Studies on Leukemia and Normal Hemopoiesis, ISSN 0145-2126, E-ISSN 1873-5835, Vol. 37, no 2, p. 162-168Article in journal (Refereed)
    Abstract [en]

    EXELS is an ongoing phase IV non-interventional study; 3643 high-risk patients with essential thrombocythemia (ET) were recruited across 13 European countries. We report patient characteristics and cytoreductive treatment patterns of ET across Europe. Hydroxycarbamide (HC; 64.3%) and anagrelide (22.0%) were the two main cytoreductive treatments prescribed. The proportions of patients taking either HC or anagrelide varied across countries, as did the number of patients receiving anti-aggregatory therapy in addition to cytoreductive treatment. This real-world evidence demonstrates that, generally, treatment patterns of ET across Europe adhere to expert recommendations, with some notable variations between countries.

  • 7.
    Birgegard, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Does anything work for anaemia in myelofibrosis?2014In: Baillière's Best Practice & Research: Clinical Haematology, ISSN 1521-6926, E-ISSN 1532-1924, Vol. 27, no 2, p. 175-185Article in journal (Refereed)
    Abstract [en]

    Anaemia is a common finding at diagnosis in myelofibrosis, and becomes a symptomatic problem in most patients with time. There are several treatment options for specific anaemia treatment, none of which has been tested in large, randomized, controlled trials. However, as myelofibrosis is not a disease with spontaneous remissions, even non-randomized trials carry weight In this survey, the existing evidence will be analysed, both for the commonly used treatments like erythropoiesis-stimulating agents, androgens and thalidomide and for the new drugs in the area, and conclusions will be drawn concerning standard clinical anaemia treatment in myelofibrosis, which according to evidence from studies has a 40-50% chance of response in patients with not too advanced disease. (C) 2014 Elsevier Ltd. All rights reserved.

  • 8.
    Birgegard, Gunnar
    et al.
    Uppsala University, Medicinska vetenskapsområdet, Faculty of Medicine, Department of Medical Sciences.
    Eriksson, Ulf
    Department of Medical Cell Biology.
    Uppsalaprofilen i läkarutbildningen. Människokunskap och biologi förenas.1995In: Läkartidningen, Vol. 92, p. 299-302Article in journal (Other scientific)
  • 9.
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    15. Anemi och järn2012In: Blodets sjukdomar: lärobok i hematologi / [ed] Gösta Gahrton & Gunnar Juliusson, Lund: Studentlitteratur, 2012, 1. uppl, p. 195-213Chapter in book (Other academic)
  • 10.
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Advances and challenges in the management of essential thrombocythemia2015In: Therapeutic advances in hematology, ISSN 2040-6215, Vol. 6, no 3, p. 142-156Article in journal (Refereed)
    Abstract [en]

    The new World Health Organization (WHO) diagnostic criteria for essential thrombocythemia (ET) issued in 2008 made an important distinction between true ET and early myelofibrosis (MF), which has helped to identify a more homogenous population for the diagnosis with longer survival and much less transformation to overt MF. The recent finding of a new mutation (CALR), which is mutually exclusive with JAK2 and MPL mutations, adds to the characterization of ET patients, since there are important phenotypic differences between the mutation types. CALR patients are younger, have lower white blood cell counts (WBC) and a lower thrombosis incidence. A growing field of interest is the state of hypercoagulation due to dysfunction of hemostatic systems, cell-cell interaction and hereditary prothrombotic traits. Activation of platelets, WBC and endothelial cells has been found, making the whole intravascular milieu prothrombotic. Several risk score models, based on retrospective studies, have been developed lately, distinguishing patient groups with graded risk for complications and death. Even if these may be helpful in evaluating patients, they have not been validated in prospective studies and there are not enough data to support their use as a basis for treatment algorithms. The traditional risk factors age, previous thrombosis and platelets >1500 × 10(9)/l are still recommended for the distinction between high risk and low risk ET and the decision to give cytoreductive therapy. However, cardiovascular (CV) risk factors add to thrombosis risk and should be considered both for specific treatment in any risk group and for upgrading low risk patients with high CV risk to an intermediary group where active therapy with aspirin and cytoreduction may be considered. First-line cytoreductive therapy differs with age; in younger patients interferon (IFN) or anagrelide are preferable, in older patients hydroxycarbamide (HC). Second-line therapy for younger patients is HC, for older patients IFN or anagrelide (ANA). JAK2 inhibitors may be suitable in rare cases with symptoms not responding to other therapy.

  • 11.
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Järnbristanemi och sekundär anemi2011In: Internmedicin / [ed] Ulf Dahlström, Stergios Kechagias & Leif Stenke, Stockholm: Liber, 2011, 5. uppl, p. 365-371Chapter in book (Other academic)
  • 12.
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Pharmacological management of essential thrombocythemia2013In: Expert Opinion on Pharmacotherapy, ISSN 1465-6566, E-ISSN 1744-7666, Vol. 14, no 10, p. 1295-1306Article, review/survey (Refereed)
    Abstract [en]

    Introduction: Two factors have deeply influenced the area of essential thrombocythemia (ET). A gain-of-function mutation in the pseudokinase region of the JAK2 gene, which partly explains the pathophysiology of myeloproliferative neoplasms (MPNs), was discovered in 2005 and is present in 50 - 60% of ET patients. Furthermore, the 2008 WHO MPN classification outlined criteria for the separation of ET and early or prefibrotic primary myelofibrosis (PMF). However, these and other new findings have not yet changed the pharmacotherapy of ET, which is based on risk stratification for thrombohemorrhagic risk and aims to reduce thrombosis and bleeding. Areas covered: Studies on the basis for and the validation of the WHO classification as well as studies on possible new risk factors are covered. The most important drugs for ET treatment and consensus recommendations for management of ET are also presented. Expert opinion: The new WHO classification should be used for both ET studies and clinical practice, since true ET has a different prognosis than early PMF. The management of patients should be based on risk stratification. Age >60 years or previous throbosis (high risk) and platelet counts >1500 x 10(9)/l warrant cytoreductive treatment, and high risk patients and selected low risk patients should be given anti-aggregation therapy.

  • 13.
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    The Use of Anagrelide in Myeloproliferative Neoplasms, with Focus on Essential Thrombocythemia2016In: Current Hematologic Malignancy Reports, ISSN 1558-8211, E-ISSN 1558-822X, Vol. 11, no 5, p. 348-355Article, review/survey (Refereed)
    Abstract [en]

    Anagrelide (ANA) is a drug with specific platelet-lowering activity, used primarily in ET, registered as a second-line drug in essential thrombocythemia (ET) in Europe and in some countries as first-line therapy, in USA licensed by FDA for thrombocythemia in myeloproliferative neoplasms (MPN). The platelet-lowering efficacy is similar to that of hydroxycarbamide (HC), around 70 % complete response and 90 % partial response. Side effects are common, especially headache and tachycardia, but usually subside or disappear within a few weeks. Around 20 % of patients stop ANA therapy due to side effects or insufficient response. Studies of treatment patterns in Europe show that ANA is preferentially given to younger patients, probably because of the concern for a possible leukemogenic effect of the common first-line drug, HC. Only two randomized studies have compared the efficacy of ANA and HC in preventing thrombosis and haemorrhage, the larger of them showing a slightly better efficacy of HC, the other showing non-inferiority of ANA to HC. A recent observational 5-year study of 3600 patients shows a low and basically similar efficacy of ANA and other cytoreductive therapies in ET. ANA does not appear to inhibit fibrosis development, and probably due to its anticoagulation properties, the combination of ASA and ANA produces an increased rate of haemorrhage. Combination of ANA with HC or interferon (IFN) is feasible and effective in patients with insufficient platelet response to mono-therapy.

  • 14.
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Övriga myeloproliferativa Sjukdomar2011In: Internmedicin / [ed] Ulf Dahlström, Stergios Kechagias & Leif Stenke, Stockholm: Liber, 2011, 5. uppl, p. 410-418Chapter in book (Other academic)
  • 15.
    Birgegård, Gunnar
    et al.
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Besses, C.
    Hosp del Mar, IMIM, Dept Haematol, Barcelona, Spain..
    Griesshammer, M.
    Johannes Wesling Med Ctr, Hematol & Oncol, Minden, Germany..
    Gugliotta, L.
    St Orsola Malpighi Hosp, Dept Haematol, L & A Seragnoli, Bologna, Italy..
    Harrison, C.
    Guys & St Thomas NHS Fdn Trust, Dept Haematol, London, England..
    Hamdani, M.
    Shire Pharmaceut, Global Biometr, Wayne, NJ USA..
    Achenbach, H.
    Shire GmbH, Res & Dev, Zug, Switzerland..
    Kiladjian, J. -J
    RISK FACTORS FOR THROMBOHEMORRHAGIC AND TRANSFORMATION EVENTS IN 3649 HIGH-RISK PATIENTS WITH ESSENTIAL THROMBOCYTHEMIA: RESULTS FROM THE PROSPECTIVE LONG-TERM OBSERVATIONAL EXELS STUDY2015In: Haematologica, ISSN 0390-6078, E-ISSN 1592-8721, Vol. 100, p. 160-161Article in journal (Other academic)
  • 16.
    Birgegård, Gunnar
    et al.
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Besses, C.
    Hosp del Mar, IMIM, Dept Haematol, Barcelona, Spain..
    Griesshammer, M.
    Johannes Wesling Med Ctr, Hematol & Oncol, Minden, Germany..
    Gugliotta, L.
    St Orsola Malpighi Hosp, Dept Haematol, L&A Seragnoli, Bologna, Italy..
    Harrison, C.
    Guys & St Thomas NHS Fdn Trust, Dept Haematol, London, England..
    Hamdani, M.
    Shire Pharmaceut, Global Biometr, Wayne, PA USA..
    Achenbach, H.
    Shire AG, Res & Dev, Eysins, Switzerland..
    Kiladjian, J-J
    Hop St Louis, APHP, Ctr Invest Clin, Paris, France..
    Treatment of high risk ET: data from the EXELS study2016In: Leukemia research: a Forum for Studies on Leukemia and Normal Hemopoiesis, ISSN 0145-2126, E-ISSN 1873-5835, Vol. 44, p. S7-S8Article in journal (Other academic)
  • 17.
    Birgegård, Gunnar
    et al.
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Besses, Carlos
    Griesshammer, Martin
    Gugliotta, Luigi
    Harrison, Claire N.
    Hamdani, Mohamed
    Achenbach, Heinrich
    Kiladjian, Jean-Jacques
    Treatment of Essential Thrombocythemia in Europe: An Observational Study of 3649 High-Risk Patients in Exels2014In: Blood, ISSN 0006-4971, E-ISSN 1528-0020, Vol. 124, no 21Article in journal (Other academic)
  • 18.
    Birgegård, Gunnar
    et al.
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Folkvaljon, Y.
    Reg Canc Ctr, Uppsala, Sweden..
    Garmo, H.
    Reg Canc Ctr, Uppsala, Sweden..
    Besses, C.
    Hosp Mar IMIM, Dept Haematol, Barcelona, Spain..
    Griesshammer, M.
    Johannes Wesling Med Ctr, Hematol & Oncol, Minden, Germany..
    Gugliotta, L.
    St Orsola Malpighi Hosp, Dept Haematol L&A Seragnoli, Bologna, Italy..
    Harrison, C.
    Guys & St Thomas NHS Fdn Trust, Dept Haematol, London, England..
    Hamdani, M.
    Shire Pharmaceut, Global Biometr, Lexington, MA USA..
    Wu, J.
    Shire Pharmaceut, Global Biometr, Lexington, MA USA..
    Achenbach, H.
    Shire GmbH, Res & Dev, Zug, Switzerland..
    Kiladjian, J. J.
    Hop St Louis, APHP, Ctr Invest Clin, Paris, France..
    Rate Of Malignant Transformation In High Risk Et During 5 Years Of Follow-Up Of Cytoreductive Therapy2016In: Haematologica, ISSN 0390-6078, E-ISSN 1592-8721, Vol. 101, p. 96-96Article in journal (Other academic)
  • 19.
    Birgegård, Gunnar
    et al.
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Henry, David
    Penn Hosp, Joan Karnell Canc Ctr, Philadelphia, PA 19107 USA..
    Glaspy, John
    Univ Calif Los Angeles, Sch Med, Dept Med, Div Hematol Oncol, Los Angeles, CA 90024 USA..
    Chopra, Rakesh
    Indraprastha Apollo Hosp, New Delhi, India..
    Thomsen, Lars L.
    Pharmacosmos AS, Dept Clin & Nonclin Res, Holbaek, Denmark..
    Auerbach, Michael
    Georgetown Univ, Sch Med, Washington, DC USA..
    A Randomized Noninferiority Trial of Intravenous Iron Isomaltoside versus Oral Iron Sulfate in Patients with Nonmyeloid Malignancies and Anemia Receiving Chemotherapy: The PROFOUND Trial2016In: Pharmacotherapy, ISSN 0277-0008, E-ISSN 1875-9114, Vol. 36, no 4, p. 402-414Article in journal (Refereed)
    Abstract [en]

    Study ObjectiveA safe alternative to erythropoiesis-stimulating agents to treat anemia is warranted in patients with cancer and anemia; thus the objective of this trial was to compare the efficacy and safety of intravenous (IV) iron isomaltoside with oral iron in patients with cancer and anemia by testing the noninferiority of IV versus oral iron. DesignPhase III, prospective, open-label, comparative, randomized, noninferiority, multicenter trial. SettingForty-seven hospitals or private cancer clinics in Asia, the United States, and Europe. PatientsA total of 350 patients with cancer and anemia. InterventionPatients were randomized in a 2:1 ratio to either intravenous iron isomaltoside or oral iron sulfate. Patients in the iron isomaltoside group were then randomized into an infusion subgroup (single intravenous infusions of a maximum dose of 1000 mg over 15 min) or a bolus injection subgroup (bolus injections of 500 mg over 2 min). Measurements and Main ResultsThe primary efficacy outcome was change in hemoglobin concentration from baseline to week 4. Changes in other relevant hematology variables, effect on quality of life, and safety outcomes were also assessed. The primary efficacy outcome was tested for noninferiority, whereas the remaining outcomes were tested for superiority. Iron isomaltoside was noninferior to oral iron in change in hemoglobin concentration from baseline to week 4 (difference estimate 0.016, 95% confidence interval -0.26 to 0.29, p<0.001). A faster onset of the hemoglobin response was observed with infusion of iron isomaltoside (superiority test: p=0.03 at week 1), and a sustained effect on hemoglobin level was shown in both the iron isomaltoside and oral iron treatment groups until week 24. A significant mean decrease in fatigue score was observed from baseline to week 12 in the iron isomaltoside group (p<0.001) but not in the oral iron group (p=0.057). A higher proportion of patients treated with oral iron experienced adverse drug reactions (18.8% vs 6.6%, p<0.001) and discontinued the trial due to intolerance (8.0% vs 0.9%, p=0.001). Transient hypophosphatemia (phosphate level less than 2 mg/dl) was reported at similar low frequencies among the groups: 7.1% in the iron isomaltoside infusion subgroup versus 8.5% in the iron isomaltoside bolus injection subgroup versus 5.4% in the oral iron group. ConclusionThis trial demonstrated comparable sustained increases in hemoglobin concentration over time with both iron isomaltoside and oral iron. Iron isomaltoside was better tolerated than oral iron, and fatigue was significantly decreased with iron isomaltoside. Low rates of clinically insignificant hypophosphatemia were reported in patients receiving both treatments.

  • 20. Björkholm, Magnus
    et al.
    Derolf, Asa R
    Hultcrantz, Malin
    Kristinsson, Sigurdur Y
    Ekstrand, Charlotta
    Goldin, Lynn R
    Andreasson, Björn
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences.
    Linder, Olle
    Malm, Claes
    Markevärn, Berit
    Nilsson, Lars
    Samuelsson, Jan
    Granath, Fredrik
    Landgren, Ola
    Treatment-related risk factors for transformation to acute myeloid leukemia and myelodysplastic syndromes in myeloproliferative neoplasms2011In: Journal of Clinical Oncology, ISSN 0732-183X, E-ISSN 1527-7755, Vol. 29, no 17, p. 2410-2415Article in journal (Refereed)
    Abstract [en]

    PURPOSE:

    Patients with myeloproliferative neoplasms (MPNs), including polycythemia vera, essential thrombocythemia, and primary myelofibrosis, have a propensity to develop acute myeloid leukemia (AML) and myelodysplastic syndromes (MDSs). Using population-based data from Sweden, we assessed the role of MPN treatment and subsequent AML/MDS risk with special focus on the leukemogenic potential of hydroxyurea (HU).

    METHODS:

    On the basis of a nationwide MPN cohort (N = 11,039), we conducted a nested case-control study, including 162 patients (153 and nine with subsequent AML and MDS diagnosis, respectively) and 242 matched controls. We obtained clinical and MPN treatment data for all patients. Using logistic regression, we calculated odds ratios (ORs) as measures of AML/MDS risk.

    RESULTS:

    Forty-one (25%) of 162 patients with MPNs with AML/MDS development were never exposed to alkylating agents, radioactive phosphorous (P(32)), or HU. Compared with patients with who were not exposed to HU, the ORs for 1 to 499 g, 500 to 999 g, more than 1,000 g of HU were 1.5 (95% CI, 0.6 to 2.4), 1.4 (95% CI, 0.6 to 3.4), and 1.3 (95% CI, 0.5 to 3.3), respectively, for AML/MDS development (not significant). Patients with MPNs who received P(32) greater than 1,000 MBq and alkylators greater than 1 g had a 4.6-fold (95% CI, 2.1 to 9.8; P = .002) and 3.4-fold (95% CI, 1.1 to 10.6; P = .015) increased risk of AML/MDS, respectively. Patients receiving two or more cytoreductive treatments had a 2.9-fold (95% CI, 1.4 to 5.9) increased risk of transformation.

    CONCLUSION:

    The risk of AML/MDS development after MPN diagnosis was significantly associated with high exposures of P(32) and alkylators but not with HU treatment. Twenty-five percent of patients with MPNs who developed AML/MDS were not exposed to cytotoxic therapy, supporting a major role for nontreatment-related factors.

  • 21. Dignass, Axel U
    et al.
    Gasche, Christoph
    Bettenworth, Dominik
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Danese, Silvio
    Gisbert, Javier P
    Gomollon, Fernando
    Iqbal, Tariq
    Katsanos, Konstantinos
    Koutroubakis, Ioannis
    Magro, Fernando
    Savoye, Guillaume
    Stein, Jürgen
    Vavricka, Stephan
    European Consensus on the Diagnosis and Management of Iron Deficiency and Anaemia in Inflammatory Bowel Diseases2015In: Journal of Crohn's & Colitis, ISSN 1873-9946, E-ISSN 1876-4479, Vol. 9, no 3, p. 211-222Article in journal (Refereed)
  • 22.
    Ejerblad, Elisabeth
    et al.
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Kvasnicka, Hans M
    Thiele, Jürgen
    Andreasson, Björn
    Björkholm, Magnus
    Löfvenberg, Eva
    Markevärn, Berit
    Merup, Mats
    Nilssson, Lars
    Palmblad, Jan
    Samuelsson, Jan
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Diagnosis according to World Health Organization determines the long-term prognosis in patients with myeloproliferative neoplasms treated with anagrelide: Results of a prospective long-term follow-up2013In: Hematology, ISSN 1024-5332, E-ISSN 1607-8454, Vol. 18, no 1, p. 8-13Article in journal (Refereed)
    Abstract [en]

    OBJECTIVES:

    During long term follow-up of a cohort of patients with essential thrombocythemia (ET) and polycythemia vera (PV) a higher than expected incidence of myelofibrosis (MF) was noted. In order to test if the explanation could be found in the diagnostic criteria a re-evaluation of diagnosis using the 2008 WHO diagnostic criteria for ET and MF was performed.

    METHODS:

    This prospective study of 60 patients with ET and PV was set up in 1998 to evaluate the long-term efficacy and tolerability of anagrelide treatment. Bone marrow trephine biopsies were requested from study start, after 2 and 7 years of follow-up. A blinded re-evaluation of the bone marrow trephines was performed. The 2008 WHO bone marrow criteria were used for diagnosis and fibrosis grading.

    RESULTS:

    Of 40 patients with an initial diagnosis of ET, 21 were confirmed as 'true ET' whereas 17 were reclassified as primary myelofibrosis (PMF) (12 PMF-0, 3 PMF-1, 2 PMF-2) and 2 as myeloproliferative neoplasms of uncertain origin. After 7 years of follow-up, 19 of 21 patients with 'true ET' were alive, none had transformed to MF, leukemia, or myelodysplastic syndrome. In contrast, 4/17 patients reclassified as PMF had died, two patients transformed to myelodysplastic syndrome and 7 patients progressed to overt MF.

    DISCUSSION:

    We conclude that a blinded re-evaluation of bone marrow trephines from study start and after 7 years of follow-up using 2008 World Health Organization criteria was able to differentiate between true ET and PMF with a marked difference in follow-up outcome.

  • 23. Emanuel, Robyn M
    et al.
    Dueck, Amylou C
    Geyer, Holly L
    Kiladjian, Jean-Jacques
    Slot, Stefanie
    Zweegman, Sonja
    Te Boekhorst, Peter A W
    Commandeur, Suzan
    Schouten, Harry C
    Sackmann, Federico
    Kerguelen Fuentes, Ana
    Hernández-Maraver, Dolores
    Pahl, Heike L
    Griesshammer, Martin
    Stegelmann, Frank
    Doehner, Konstanze
    Lehmann, Thomas
    Bonatz, Karin
    Reiter, Andreas
    Boyer, Francoise
    Etienne, Gabriel
    Ianotto, Jean-Christophe
    Ranta, Dana
    Roy, Lydia
    Cahn, Jean-Yves
    Harrison, Claire N
    Radia, Deepti
    Muxi, Pablo
    Maldonado, Norman
    Besses, Carlos
    Cervantes, Francisco
    Johansson, Peter L
    Barbui, Tiziano
    Barosi, Giovanni
    Vannucchi, Alessandro M
    Passamonti, Francesco
    Andreasson, Bjorn
    Ferarri, Maria L
    Rambaldi, Alessandro
    Samuelsson, Jan
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Tefferi, Ayalew
    Mesa, Ruben A
    Myeloproliferative Neoplasm (MPN) Symptom Assessment Form Total Symptom Score: Prospective International Assessment of an Abbreviated Symptom Burden Scoring System Among Patients With MPNs2012In: Journal of Clinical Oncology, ISSN 0732-183X, E-ISSN 1527-7755, Vol. 30, no 33, p. 4098-4103Article in journal (Refereed)
    Abstract [en]

    PURPOSE

    Myeloproliferative neoplasm (MPN) symptoms are troublesome to patients, and alleviation of this burden represents a paramount treatment objective in the development of MPN-directed therapies. We aimed to assess the utility of an abbreviated symptom score for the most pertinent and representative MPN symptoms for subsequent serial use in assessing response to therapy.

    PATIENTS AND METHODS

    The Myeloproliferative Neoplasm Symptom Assessment Form total symptom score (MPN-SAF TSS) was calculated as the mean score for 10 items from two previously validated scoring systems. Questions focus on fatigue, concentration, early satiety, inactivity, night sweats, itching, bone pain, abdominal discomfort, weight loss, and fevers.

    RESULTS 

    MPN-SAF TSS was calculable for 1,408 of 1,433 patients with MPNs who had a mean score of 21.2 (standard deviation [SD], 16.3). MPN-SAF TSS results significantly differed among MPN disease subtypes (P < .001), with a mean of 18.7 (SD, 15.3), 21.8 (SD, 16.3), and 25.3 (SD, 17.2) for patients with essential thrombocythemia, polycythemia vera, and myelofibrosis, respectively. The MPN-SAF TSS strongly correlated with overall quality of life (QOL; r = 0.59; P < .001) and European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire C30 (EORTC QLQ-C30) functional scales (all P < .001 and absolute r ≥ 0.50 except social functioning r = 0.48). No significant trends were present when comparing therapy subgroups. The MPN-SAF TSS had excellent internal consistency (Cronbach's α = .83). Factor analysis identified a single underlying construct, indicating that the MPN-SAF TSS is an appropriate, unified scoring method.

    CONCLUSION

    The MPN-SAF TSS is a concise, valid, and accurate assessment of MPN symptom burden with demonstrated clinical utility in the largest prospective MPN symptom study to date. This new prospective scoring method may be used to assess MPN symptom burden in both clinical practice and trial settings.

  • 24. Emanuel, Robyn M.
    et al.
    Dueck, Amylou C.
    Geyer, Holly Lynn
    Kiladjian, Jean-Jacques
    Slot, Stefanie
    Zweegman, Sonja
    te Boekhorst, Peter
    Commandeur, Suzan
    Schouten, Harry C.
    Sackmann, Federico
    Kerguelen Fuentes, Ana
    Hernandez, Dolores
    Pahl, Heike L.
    Griesshammer, Martin
    Stegelmann, Frank
    Doehner, Konstanze
    Lehmann, Thomas
    Bonatz, Karin
    Reiter, Andreas
    Boyer, Francoise
    Etienne, Gabriel
    Ianotto, Jean-Christophe
    Ranta, Dana
    Roy, Lydia
    Cahn, Jean-Yves
    Harrison, Claire N.
    Radia, Deepti H.
    Muxi, Pablo J.
    Maldonado, Norman I.
    Besses, Carlos
    Cervantes, Francisco
    Johansson, Peter
    Barbui, Tiziano
    Barosi, Giovanni
    Vannucchi, Alessandro M.
    Passamonti, Francesco
    Andreasson, Bjorn
    Ferarri, Maria L.
    Rambaldi, Alessandro
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Xiao, Zhijian
    Xu, Zefeng
    Zhang, Yue
    Sun, Xiujuan
    Xu, Junqing
    Zhang, Peihong
    Gale, Robert Peter
    Tefferi, Ayalew
    Mesa, Ruben A.
    Essential Thrombocythemia (ET) and Polycythemia Vera (PV) Symptom Burden: Phenotypic Cluster Analysis Among an International Sample of 1,141 ET and PV Patients2012In: Blood, ISSN 0006-4971, E-ISSN 1528-0020, Vol. 120, no 21, p. 1726-Article in journal (Refereed)
  • 25. Geyer, H.
    et al.
    Emanuel, R.
    Dueck, A.
    Kiladjian, J. J.
    Xiao, Z.
    Slot, S.
    Zweegman, S.
    Sackman, F.
    Kerguelen Fuentes, A.
    Hernandez-Maraver, D.
    Dohner, K.
    Harrison, C.
    Radia, D.
    Muxi, P.
    Besses, C.
    Cervantes, F.
    Johansson, P.
    Andreasson, B.
    Rambaldi, A.
    Barbui, T.
    Vannucchi, A.
    Passamonti, F.
    Samuelsson, J.
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Bonatz, K.
    Reiter, A.
    Boyer, F.
    Etienne, G.
    Ianotto, J. C.
    Ranta, D.
    Roy, L.
    Cahn, J. Y.
    Maldonado, N.
    Barosi, G.
    Ferrari, M.
    Cannon, K.
    te Boekhorst, P. A.
    Klauke, K.
    Schouten, H.
    Pahl, H.
    Griesshammer, M.
    Stegelmann, F.
    Lehmann, T.
    Xu, Z.
    Zhang, Y.
    Sun, X.
    Xu, J.
    Zhang, P.
    Mesa, R.
    Gender Differences and MPN Symptom Burden: An Analysis by the MPN Quality of Life International Study Group (MPN-QOL ISG)2014In: Haematologica (online), ISSN 0390-6078, E-ISSN 1592-8721, Vol. 99, p. 396-397Article in journal (Other academic)
  • 26.
    Geyer, H.
    et al.
    Mayo Clin, Phoenix, AZ USA..
    Kosiorek, H.
    Mayo Clin, Phoenix, AZ USA..
    Dueck, A.
    Mayo Clin, Phoenix, AZ USA..
    Slot, S.
    Vrije Univ Amsterdam, Med Ctr, Amsterdam, Netherlands..
    Zweegman, S.
    Vrije Univ Amsterdam, Med Ctr, Amsterdam, Netherlands..
    Kiladjian, J. J.
    Hosp St Louis, Paris, France..
    Boekhorst, P.
    Erasmus MC, Rotterdam, Netherlands..
    Schouten, H.
    Maastricht Univ, Med Ctr, Maastricht, Netherlands..
    Sackmann, F.
    Fundaleu, Buenos Aires, DF, Argentina..
    Fuentes, A.
    Univ Hosp La Paz, Madrid, Spain..
    Hernandez-Maraver, D.
    Univ Hosp La Paz, Madrid, Spain..
    Pahl, H.
    Univ Hosp Freiburg, Freiburg, Germany..
    Stegelmann, F.
    Univ Hosp Ulm, Ulm, Germany..
    Doehner, K.
    Univ Hosp Ulm, Ulm, Germany..
    Bonatz, K.
    Med Klin, Mannheim, Germany..
    Reiter, A.
    Med Klin, Mannheim, Germany..
    Boyer, F.
    CHU Angers, Angers, France..
    Etienne, G.
    CHU Angers, Angers, France..
    Ianotto, J. C.
    Univ Hosp, Brest, France..
    Ranta, D.
    Univ Hosp, Nancy, France..
    Roy, L.
    Ctr Hosp Univ, Poitiers, France..
    Cahn, J. Y.
    CHU Grenoble, Grenoble, France..
    Harrison, C.
    Guys & St Thomas NHS Fdn Trust, London, England..
    Radia, D.
    Guys & St Thomas NHS Fdn Trust, London, England..
    Muxi, P.
    Hosp Britanico, Montevideo, Uruguay..
    Maldonado, N.
    Univ Puerto Rico, Sch Med, San Juan, PR 00936 USA..
    Besses, C.
    Hosp Mar, Barcelona, Spain..
    Cervantes, F.
    Univ Barcelona, Barcelona, Spain..
    Johansson, P.
    NU Hosp Org, Uddevalla, Sweden..
    Barosi, G.
    IRCCS Policlin S Matteo Fdn, Pavia, Italy..
    Passamonti, F.
    Fdn IRCCS Policlin San Matteo, Pavia, Italy..
    Andreasson, B.
    NU Hosp Org, Uddevalla, Sweden..
    Samuelsson, J.
    Stockholm South Hosp, Stockholm, Sweden..
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Sun, X.
    Chinese Acad Med Sci, Tianjin, Peoples R China.;Peking Union Med Coll, Tianjin, Peoples R China..
    Xu, J.
    Chinese Acad Med Sci, Tianjin, Peoples R China.;Peking Union Med Coll, Tianjin, Peoples R China..
    Zhang, P.
    Chinese Acad Med Sci, Tianjin, Peoples R China.;Peking Union Med Coll, Tianjin, Peoples R China..
    Xu, Z.
    Chinese Acad Med Sci, Tianjin, Peoples R China.;Peking Union Med Coll, Tianjin, Peoples R China..
    Barbui, T.
    Osped Riuniti Bergamo, Bergamo, Italy.;Osped Riuniti Bergamo, Bergamo, Italy..
    Senyak, Z.
    MPN Forum, Asheville, NC USA..
    Grieshammer, M.
    Johannes Wesling Klinikum, Minden, Germany..
    Rambaldi, A.
    Osped Riuniti Bergamo, Bergamo, Italy..
    Ferrari, M.
    Osped Riuniti Bergamo, Bergamo, Italy..
    Lehmann, T.
    Univ Basel Hosp, Basel, Switzerland..
    Scherber, R.
    Mayo Clin, Phoenix, AZ USA..
    Mesa, R.
    Mayo Clin, Phoenix, AZ USA..
    Development Of An Mf Patient Reported Outcome (Pro) Tool For Fda Qualification: Comprehensive Literature Search And Physician Cognitive Debriefing Results2016In: Haematologica, ISSN 0390-6078, E-ISSN 1592-8721, Vol. 101, p. 564-564Article in journal (Other academic)
  • 27.
    Geyer, H.
    et al.
    Mayo Clin, Scottsdale, AZ USA..
    Kosiorek, H.
    Mayo Clin, Scottsdale, AZ USA..
    Scherber, R.
    Mayo Clin, Scottsdale, AZ USA..
    Dueck, A.
    Mayo Clin, Scottsdale, AZ USA..
    Jean-Jacques, K.
    Hosp St Louis, Paris, France..
    Xiao, Z.
    Chinese Acad Med Sci, Tianjin, Peoples R China.;Peking Union Med Coll, Tianjin, Peoples R China..
    Zweegman, S.
    Vrije Univ Amsterdam, Med Ctr, Amsterdam, Netherlands..
    Sackman, F.
    Fundaleu, Buenos Aires, DF, Argentina..
    Hernandez-Maraver, D.
    Univ Hosp La Paz, Madrid, Spain..
    Dohner, K.
    Univ Hosp Ulm, Ulm, Germany..
    Harrison, C.
    Guys & St Tomas NHS Fdn Trust, London, England..
    Radia, D.
    Guys & St Tomas NHS Fdn Trust, London, England..
    Muxi, P.
    Hosp Britanico, Montevideo, Uruguay..
    Besses, C.
    Hosp Del Mar, Barcelona, Spain..
    Cervantes, F.
    Univ Barcelona, Barcelona, Spain..
    Johansson, P.
    NU Hosp Org, Uddevalla, Sweden..
    Andreasson, B.
    NU Hosp Org, Uddevalla, Sweden..
    Rambaldi, A.
    Azienda Ospedal, Bergamo, Italy..
    Barbui, T.
    Azienda Ospedal, Bergamo, Italy..
    Vannucchi, A.
    Osped Circolo Varese, Varese, Italy..
    Passamonti, F.
    Fdn IRCCS Policlin San Matteo, Pavia, Italy..
    Samuelsson, J.
    Stockholm South Hosp, Stockholm, Sweden..
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Bonatz, K.
    Med Klin, Mannheim, Germany..
    Reiter, A.
    Med Klin, Mannheim, Germany..
    Boyer, F.
    Ctr Hosp Univ, Angers, France..
    Etienne, G.
    Inst Bergonie, Bordeaux, France..
    Ianotto, J. -C
    Ranta, D.
    Univ Hosp, Nancy, France..
    Roy, L.
    Ctr Hosp Univ, Poitiers, France..
    Cahn, J. -Y
    Maldonado, N.
    Univ Puerto Rico, Sch Med, San Juan, PR 00936 USA..
    Barosi, G.
    IRCCS Policlin S Matteo Fdn, Pavia, Italy..
    Ferrari, M.
    Osped Riuniti Bergamo, I-24100 Bergamo, Italy..
    Cannon, K.
    Mayo Clin, Scottsdale, AZ USA..
    te Boekhorst, P. A. W.
    Erasmus MC, Rotterdam, Netherlands..
    Klauke, K.
    Leiden Univ, Med Ctr, Leiden, Netherlands..
    Schouten, H.
    Maastricht Univ, Med Ctr, Maastricht, Netherlands..
    Pahl, H.
    Univ Hosp Freiburg, Freiburg, Germany..
    Griesshammer, M.
    Johannes Wesling Klinikum, Minden, Germany..
    Stegelmann, F.
    Univ Hosp Ulm, Ulm, Germany..
    Lehmann, T.
    Univ Basel Hosp, CH-4031 Basel, Switzerland..
    Xu, Z.
    Chinese Acad Med Sci, Inst Hematol, Tianjin, Peoples R China.;Blood Dis Hosp, Tianjin, Peoples R China..
    Zhang, Y.
    Chinese Acad Med Sci, Inst Hematol, Tianjin, Peoples R China.;Blood Dis Hosp, Tianjin, Peoples R China..
    Sun, X.
    Chinese Acad Med Sci, Inst Hematol, Tianjin, Peoples R China.;Blood Dis Hosp, Tianjin, Peoples R China..
    Xu, J.
    Chinese Acad Med Sci, Inst Hematol, Tianjin, Peoples R China.;Blood Dis Hosp, Tianjin, Peoples R China..
    Zhang, P.
    Chinese Acad Med Sci, Inst Hematol, Tianjin, Peoples R China.;Blood Dis Hosp, Tianjin, Peoples R China..
    Mesa, R.
    Mayo Clin, Scottsdale, AZ USA..
    PRIMARY MYELOFIBROSIS, POST-ET AND POST-PV MYELOFIBROSIS HAVE DISTINCT CLINICAL PROFILES AND SYMPTOMATIC BURDENS: AN ANALYSIS BY THE MPN QUALITY OF LIFE INTERNATIONAL STUDY GROUP (MPN-QOL ISG)2015In: Haematologica, ISSN 0390-6078, E-ISSN 1592-8721, Vol. 100, p. 261-262Article in journal (Other academic)
  • 28.
    Geyer, H.
    et al.
    Mayo Clin, Phoenix, AZ USA..
    Scherber, R.
    OHSU, Portland, OR USA..
    Kosiorek, H.
    Mayo Clin, Phoenix, AZ USA..
    Dueck, A.
    Mayo Clin, Phoenix, AZ USA..
    Kiladjian, J. J.
    Hosp St Louis, Paris, France..
    Slot, S.
    Vrije Univ Amsterdam, Med Ctr, Amsterdam, Netherlands..
    Zweegman, S.
    Vrije Univ Amsterdam, Med Ctr, Amsterdam, Netherlands..
    Boekhorst, P.
    Erasmus Med Cener, Rotterdam, Netherlands..
    Schouten, H.
    Maastricht Univ, Med Ctr, Maastricht, Netherlands..
    Sackmann, F.
    Fundaleu, Buenos Aires, DF, Argentina..
    Fuentes, A.
    Univ Hosp La Paz, Madrid, Spain..
    Hernandez-Maraver, D.
    Univ Hosp La Paz, Madrid, Spain..
    Pahl, H.
    Univ Hosp Freiburg, Freiburg, Germany..
    Stegelmann, F.
    Univ Hosp Ulm, Ulm, Germany..
    Doehner, K.
    Univ Hosp Ulm, Ulm, Germany..
    Bonatz, K.
    Med Klin, Mannheim, Germany..
    Reiter, A.
    Med Klin, Mannheim, Germany..
    Boyer, F.
    Ctr Hosp Univ, Angers, France..
    Etienne, G.
    Ctr Hosp Univ, Angers, France..
    Ianotto, J. C.
    Hosp Univ, Brest, France..
    Ranta, D.
    Hosp Univ, Nancy, France..
    Roy, L.
    Ctr Hosp Univ, Poitiers, France..
    Cahn, J. Y.
    Ctr Hosp Univ, Grenoble, France..
    Harrison, C.
    Guys & St Thomas NHS Fdn Trust, London, England..
    Radia, D.
    Guys & St Thomas NHS Fdn Trust, London, England..
    Muxi, P.
    Hosp Britanico, Montevideo, Uruguay..
    Maldonado, N.
    Univ Puerto Rico, Sch Med, San Juan, PR 00936 USA..
    Besses, C.
    Hosp del Mar, Barcelona, Spain..
    Cervantes, F.
    Univ Barcelona, Barcelona, Spain..
    Johansson, P.
    NU Hosp Org, Uddevalla, Sweden..
    Barosi, G.
    IRCCS Policlin S Matteo Fdn, Pavia, Italy..
    Vannucchi, A.
    Osped Circolo Varese, Varese, Italy..
    Passamonti, F.
    Fdn IRCCS Policlin San Matteo, Pavia, Italy..
    Andreasson, B.
    NU Hosp Org, Uddavalla, Sweden..
    Samuelsson, J.
    Stockholm South Hosp, Stockholm, Sweden..
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Sun, X.
    Chinese Acad Med Sci, Tianjin, Peoples R China.;Peking Union Med Coll, Tianjin, Peoples R China..
    Xu, J.
    Chinese Acad Med Sci, Tianjin, Peoples R China.;Peking Union Med Coll, Tianjin, Peoples R China..
    Zhang, P.
    Chinese Acad Med Sci, Tianjin, Peoples R China.;Peking Union Med Coll, Tianjin, Peoples R China..
    Xu, Z.
    Chinese Acad Med Sci, Tianjin, Peoples R China.;Peking Union Med Coll, Tianjin, Peoples R China..
    Barbui, T.
    Osped Riuniti Bergamo, Bergamo, Italy.;Osped Riuniti Bergamo, Bergamo, Italy..
    Senyak, Z.
    MPN Forum, Asheville, NC USA..
    Grieshammer, M.
    Johannes Wesling Klinikum, Minden, Germany..
    Rambaldi, A.
    Osped Riuniti Bergamo, Bergamo, Italy..
    Ferrari, M.
    Osped Riuniti Bergamo, Bergamo, Italy..
    Lehmann, T.
    Univ Basel Hosp, Basel, Switzerland..
    Mesa, R.
    Mayo Clin, Phoenix, AZ USA..
    Impact Of Splenomegaly On Mpn Symptoms And Association With Clinical Features: An Analysis By The Mpn Quality Of Life International Study Group2016In: Haematologica, ISSN 0390-6078, E-ISSN 1592-8721, Vol. 101, p. 555-555Article in journal (Other academic)
  • 29.
    Geyer, Holly L.
    et al.
    Mayo Clin, Div Hosp Internal Med, 13400 E Shea Blvd, Scottsdale, AZ 85259 USA..
    Andreasson, Bjorn
    NU Hosp Org, Internal Med, Uddevalla, Sweden..
    Kosiorek, Heidi E.
    Mayo Clin, Sect Biostat, 13400 E Shea Blvd, Scottsdale, AZ 85259 USA..
    Dueck, Amylou C.
    Mayo Clin, Sect Biostat, 13400 E Shea Blvd, Scottsdale, AZ 85259 USA..
    Scherber, Robyn M.
    Oregon Hlth & Sci Univ, Dept Hematol & Oncol, Portland, OR 97201 USA..
    Martin, Kari A.
    Mayo Clin, Dept Psychiat, 13400 E Shea Blvd, Scottsdale, AZ 85259 USA..
    Butler, Kristina A.
    Mayo Clin, Dept Gynecol, 13400 E Shea Blvd, Scottsdale, AZ 85259 USA..
    Harrison, Claire N.
    Guys & St Thomas NHS Fdn Trust, Dept Haematol, London, England..
    Radia, Deepti H.
    Guys & St Thomas NHS Fdn Trust, Dept Haematol, London, England..
    Cervantes, Francisco
    Univ Barcelona, Dept Hematol, Hosp Clin, IDIBAPS, Barcelona, Spain..
    Kiladjian, Jean-Jacques
    Hosp St Louis, Clin Invest Ctr, Paris, France..
    Reiter, Andreas
    Univ Mannheim, Med Clin, D-68131 Mannheim, Germany..
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Passamonti, Francesco
    Univ Pavia, Dept Hematol, IRCCS Fdn San Matteo Polyclin, Via Palestro 3, I-27100 Pavia, Italy..
    Senyak, Zhenya
    MPN Forum, Asheville, NC USA..
    Vannucchi, Alessandro M.
    Circolo Hosp, Div Hematol, Varese, Italy..
    Paoli, Chiara
    Univ Florence, Dept Med, Florence, Italy..
    Xiao, Zhijian
    Chinese Acad Med Sci, Inst Hematol, MDS & MPN Ctr, Tianjin, Peoples R China.;Chinese Acad Med Sci, Blood Dis Hosp, Tianjin, Peoples R China.;Peking Union Med Coll, Tianjin, Peoples R China..
    Samuelsson, Jan
    Stockholm South Hosp, Dept Internal Med, Stockholm, Sweden..
    Mesa, Ruben A.
    Mayo Clin, Div Hematol Oncol, 13400 E Shea Blvd, Scottsdale, AZ 85259 USA..
    The role of sexuality symptoms in myeloproliferative neoplasm symptom burden and quality of life: An analysis by the MPN QOL International Study Group2016In: Cancer, ISSN 0008-543X, E-ISSN 1097-0142, Vol. 122, no 12, p. 1888-1896Article in journal (Refereed)
    Abstract [en]

    BACKGROUNDPatients with myeloproliferative neoplasms (MPNs) including polycythemia vera, essential thrombocythemia, and myelofibrosis, are faced with oppressive symptom profiles that compromise daily functioning and quality of life. Among these symptoms, sexuality-related symptoms have emerged as particularly prominent and largely unaddressed. In the current study, the authors evaluated how sexuality symptoms from MPN relate to other patient characteristics, disease features, treatments, and symptoms. METHODSA total of 1971 patients with MPN (827 with essential thrombocythemia, 682 with polycythemia vera, 456 with myelofibrosis, and 6 classified as other) were prospectively evaluated and patient responses to the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) and the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC-QLQ C30) were collected, along with information regarding individual disease characteristics and laboratory data. Sexuality scores were compared with an age-matched, healthy control population. RESULTSOverall, patients with MPN were found to have greater sexual dysfunction compared with the healthy population (MPN-SAF score of 3.6 vs 2.0; P<.001), with 64% of patients with MPN describing some degree of sexual dysfunction and 43% experiencing severe symptoms. The presence of sexual symptoms correlated closely with all domains of patient functionality (physical, social, cognitive, emotional, and role functioning) and were associated with a reduced quality of life. Sexual problems also were found to be associated with other MPN symptoms, particularly depression and nocturnal and microvascular-related symptoms. Sexual dysfunction was more severe in patients aged >65 years and in those with cytopenias and transfusion requirements, and those receiving certain therapies such as immunomodulators or steroids. ConclusionsThe results of the current study identify the topic of sexuality as a prominent issue for the MPN population, and this area would appear to benefit from additional investigation and management. Cancer 2016;122:1888-96. (c) 2016 American Cancer Society. Sexuality problems impact all domains of functionality, depression, microvascular symptoms, and overall quality of life among patients with myeloproliferative neoplasms. These problems correlate with patient age, the presence of cytopenias, transfusion requirements, and common therapies for myeloproliferative neoplasms. See also pages 1804-6.

  • 30.
    Geyer, Holly L.
    et al.
    Mayo Clin, Dept Internal Med, Scottsdale, AZ 85259 USA..
    Kosiorek, Heidi
    Mayo Clin, Sect Biostat, Scottsdale, AZ USA..
    Dueck, Amylou C.
    Mayo Clin, Sect Biostat, Scottsdale, AZ USA..
    Scherber, Robyn
    Oregon Hlth & Sci Univ, Portland, OR USA..
    Slot, Stefanie
    Vrije Univ Amsterdam Med Ctr, Dept Hematol, Amsterdam, Netherlands..
    Zweegman, Sonja
    Vrije Univ Amsterdam Med Ctr, Dept Hematol, Amsterdam, Netherlands..
    te Boekhorst, Peter A. W.
    Erasmus MC, Dept Hematol, Rotterdam, Netherlands..
    Senyak, Zhenya
    MPN Forum, Asheville, NC USA..
    Schouten, Harry C.
    MUMC, Dept Hematol, Maastricht, Netherlands..
    Sackmann, Federico
    Fundaleu, Buenos Aires, DF, Argentina..
    Kerguelen Fuentes, Ana
    Univ Hosp La Paz, Dept Haematol, Madrid, Spain..
    Hernandez-Maraver, Dolores
    Univ Hosp La Paz, Dept Haematol, Madrid, Spain..
    Pahl, Heike L.
    Univ Hosp Freiburg, Dept Mol Hematol, Freiburg, Germany..
    Griesshammer, Martin
    Johannes Wesling Klinikum, Minden, Germany..
    Stegelmann, Frank
    Univ Hosp Ulm, Dept Internal Med 3, Ulm, Germany..
    Doehner, Konstanze
    Univ Hosp Ulm, Dept Internal Med 3, Ulm, Germany..
    Lehmann, Thomas
    Univ Hosp, Dept Hematol, Basel, Switzerland..
    Bonatz, Karin
    Univ Med, Med Klin, Mannheim, Germany..
    Reiter, Andreas
    Univ Med, Med Klin, Mannheim, Germany..
    Boyer, Francoise
    Ctr Hosp Univ, Angers, France..
    Etienne, Gabriel
    Inst Bergonie, Bordeaux, France..
    Ianotto, Jean-Christophe
    Ctr Hosp Univ, Brest, France..
    Ranta, Dana
    Hosp Univ, Nancy, France..
    Roy, Lydia
    Ctr Hosp Univ, Poitiers, France..
    Cahn, Jean-Yves
    Ctr Hosp Univ, Grenoble, France..
    Harrison, Claire N.
    Guys & St Thomas NHS Fdn Trust, Dept Haematol, London, England..
    Radia, Deepti
    Guys & St Thomas NHS Fdn Trust, Dept Haematol, London, England..
    Muxi, Pablo
    Hosp Britan, Unidadde Hematol, Montevideo, Uruguay..
    Maldonado, Norman
    Univ Puerto Rico, Sch Med, San Juan, PR 00936 USA..
    Besses, Carlos
    Hosp del Mar, Dept Hematol, Barcelona, Spain..
    Cervantes, Francisco
    Univ Barcelona, Hosp Clin, Dept Hematol, IDIBAPS, Barcelona, Spain..
    Johansson, Peter L.
    NU Hosp Org, Internal Med, Uddevalla, Sweden..
    Barbui, Tiziano
    Hosp Papa Giovanni XXIII, Res Fdn FROM, Bergamo, Italy..
    Barosi, Giovanni
    IRCCS Policlin S Matteo Fdn, Lab Clin Epidemiol, Pavia, Italy..
    Vannucchi, Alessandro M.
    Univ Florence, Ctr Res & Innovat Myeloproliferat Neoplasms, Florence, Italy..
    Paoli, Chiara
    Univ Florence, Ctr Res & Innovat Myeloproliferat Neoplasms, Florence, Italy..
    Passamonti, Francesco
    Univ Insubria, Dipartimento Med Clin & Sperimentale, Ematol, Varese, Italy..
    Andreasson, Bjorn
    NU Hosp Org, Internal Med, Uddevalla, Sweden..
    Ferrari, Maria L.
    Osped Riuniti Bergamo, Biol Sci, Bergamo, Italy..
    Rambaldi, Alessandro
    Hosp Papa Giovanni XXIII, Res Fdn FROM, Bergamo, Italy..
    Samuelsson, Jan
    Stockholm South Hosp, Dept Internal Med, Stockholm, Sweden..
    Cannon, Keith
    Mayo Clin, Dept Internal Med, Scottsdale, AZ 85259 USA..
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Xiao, Zhijian
    Chinese Acad Med Sci, Inst Hematol, MDS & MPN Ctr, Tianjin, Peoples R China.;Chinese Acad Med Sci, Blood Dis Hosp, Tianjin, Peoples R China.;Peking Union Med Coll, Tianjin, Peoples R China..
    Xu, Zefeng
    Chinese Acad Med Sci, Inst Hematol, MDS & MPN Ctr, Tianjin, Peoples R China.;Chinese Acad Med Sci, Blood Dis Hosp, Tianjin, Peoples R China.;Peking Union Med Coll, Tianjin, Peoples R China..
    Zhang, Yue
    Chinese Acad Med Sci, Inst Hematol, MDS & MPN Ctr, Tianjin, Peoples R China.;Chinese Acad Med Sci, Blood Dis Hosp, Tianjin, Peoples R China.;Peking Union Med Coll, Tianjin, Peoples R China..
    Sun, Xiujuan
    Chinese Acad Med Sci, Inst Hematol, MDS & MPN Ctr, Tianjin, Peoples R China.;Chinese Acad Med Sci, Blood Dis Hosp, Tianjin, Peoples R China.;Peking Union Med Coll, Tianjin, Peoples R China..
    Xu, Junqing
    Chinese Acad Med Sci, Inst Hematol, MDS & MPN Ctr, Tianjin, Peoples R China.;Chinese Acad Med Sci, Blood Dis Hosp, Tianjin, Peoples R China.;Peking Union Med Coll, Tianjin, Peoples R China..
    Kiladjian, Jean-Jacques
    Hosp St Louis, Clin Invest Ctr, Paris, France..
    Zhang, Peihong
    Chinese Acad Med Sci, Blood Dis Hosp, Tianjin, Peoples R China.;Peking Union Med Coll, Tianjin, Peoples R China.;Chinese Acad Med Sci, Inst Hematol, Dept Pathol, Tianjin, Peoples R China..
    Gale, Robert Peter
    Imperial Coll, London, England..
    Mesa, Ruben A.
    Mayo Clin, Dept Hematol & Oncol, Scottsdale, AZ 85259 USA..
    Associations between gender, disease features and symptom burden in patients with myeloproliferative neoplasms: an analysis by the MPN QOL International Working Group2016In: Haematologica, ISSN 0390-6078, E-ISSN 1592-8721, Vol. 102, no 1, p. 85-93Article in journal (Refereed)
    Abstract [en]

    The myeloproliferative neoplasms, including polycythemia vera, essential thrombocythemia and myelofibrosis, are distinguished by their debilitating symptom profiles, life-threatening complications and profound impact on quality of life. The role gender plays in the symptomatology of myeloproliferative neoplasms remains under-investigated. In this study we evaluated how gender relates to patients' characteristics, disease complications and overall symptom expression. A total of 2,006 patients (polycythemia vera=711, essential thrombocythemia=830, myelofibrosis=460, unknown=5) were prospectively evaluated, with patients completing the Myeloproliferative Neoplasm-Symptom Assessment Form and Brief Fatigue Inventory Patient Reported Outcome tools. Information on the individual patients' characteristics, disease complications and laboratory data was collected. Consistent with known literature, most female patients were more likely to have essential thrombocythemia (48.6% versus 33.0%; P<0.001) and most male patients were more likely to have polycythemia vera (41.8% versus 30.3%; P<0.001). The rate of thrombocytopenia was higher among males than females (13.9% versus 8.2%; P<0.001) and males also had greater red-blood cell transfusion requirements (7.3% versus 4.9%; P=0.02) with shorter mean disease duration (6.4 versus 7.2 years, P=0.03). Despite there being no statistical differences in risk scores, receipt of most therapies or prior complications (hemorrhage, thrombosis), females had more severe and more frequent symptoms for most individual symptoms, along with overall total symptom score (22.8 versus 20.3; P<0.001). Females had particularly high scores for abdominal-related symptoms (abdominal pain/discomfort) and microvascular symptoms (headache, fatigue, insomnia, concentration difficulties, dizziness; all P<0.01). Despite complaining of more severe symptom burden, females had similar quality of life scores to those of males. The results of this study suggest that gender contributes to the heterogeneity of myeloproliferative neoplasms by influencing phenotypic profiles and symptom expression.

  • 31. Geyer, Holly L.
    et al.
    Scherber, Robyn M.
    Dueck, Amylou C.
    Kiladjian, Jean-Jacques
    Xiao, Zhijian
    Slot, Stefanie
    Zweegman, Sonja
    Sackmann, Federico
    Kerguelen Fuentes, Ana
    Hernandez-Maraver, Dolores
    Doehner, Konstanze
    Harrison, Claire N.
    Radia, Deepti
    Muxi, Pablo
    Besses, Carlos
    Cervantes, Francisco
    Johansson, Peter L.
    Andreasson, Bjorn
    Rambaldi, Alessandro
    Barbui, Tiziano
    Vannucchi, Alessandro M.
    Passamonti, Francesco
    Samuelsson, Jan
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Mesa, Ruben A.
    Distinct clustering of symptomatic burden among myeloproliferative neoplasm patients: retrospective assessment in 1470 patients2014In: Blood, ISSN 0006-4971, E-ISSN 1528-0020, Vol. 123, no 24, p. 3803-3810Article in journal (Refereed)
    Abstract [en]

    Symptom burden in myeloproliferative neoplasms (MPNs) is heterogeneous even among patients within the same MPN diagnosis. Using cluster analysis from prospectively gathered symptom burden data in 1470 international patients with essential thrombocythemia (ET), polycythemia vera (PV), or myelofibrosis (MF), we assessed for the presence of clusters and relationship to disease features and prognosis. In MF (4 clusters identified), clusters significantly differed by Dynamic International Prognostic Scoring System (DIPSS) risk (P < .001), leukopenia (P = .009), thrombocytopenia (P < .001), and spleen size (P = .02). Although an association existed between clusters and DIPSS risk, high symptom burden was noted in some low and intermediate-1-risk MF patients. In PV (5 clusters identified), total symptom score increased across clusters (P < .001), but clusters did not significantly differ by PV risk or the risk assessment variable of age. Among ET patients (5 clusters identified), clusters differed by gender (P = .04), anemia (P = .01), and prior hemorrhage (P = .047). Total symptom score increased across clusters (P < .001), but clusters did not significantly differ by International Prognostic Score for ET risk including the risk assessment variables. Significant symptom heterogeneity exists within each MPN subtype, sometimes independent of disease features or prognosis.

  • 32. Geyer, Holly Lynn
    et al.
    Dueck, Amylou C.
    Emanuel, Robyn M.
    Kiladjian, Jean-Jacques
    Slot, Stefanie
    Zweegman, Sonja
    Boekhorst, Peter
    Commandeur, Suzan
    Schouten, Harry C.
    Sackmann, Federico
    Kerguelen Fuentes, Ana
    Hernandez-Maraver, Dolores
    Pahl, Heike
    Griesshammer, Martin
    Stegelmann, Frank
    Doehner, Konstanze
    Boyer, Francoise
    Etienne, Gabriel
    Lanotto, Jean-Christophe
    Ranta, Dana
    Roy, Lydia
    Cahn, Jean-Yves
    Harrison, Claire N.
    Radia, Deepti H.
    Muxi, Pablo J.
    Maldonado, Norman I.
    Besses, Carlos
    Cervantes, Francisco
    Johansson, Peter
    Barosi, Giovanni
    Vannucchi, Alessandro M.
    Passamonti, Francesco
    Andreasson, Bjorn
    Ferarri, Maria L.
    Rambaldi, Alessandro
    Samuelsson, Jan
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Xiao, Zhijian
    Zhang, Yue
    Sun, Xiujuan
    Xu, Junqing
    Gale, Robert Peter
    Tefferi, Ayalew
    Zhang, Peihong
    Lehmann, Thomas
    Reiter, Andreas
    Bonatz, Karin
    Mesa, Ruben A.
    The Myelofibrosis Symptom Burden (MF-SB): An International Phenotypic Cluster Analysis of 329 Patients2012In: Blood, ISSN 0006-4971, E-ISSN 1528-0020, Vol. 120, no 21, p. 1731-Article in journal (Refereed)
  • 33. Geyer, Holly Lynn
    et al.
    Scherber, Robyn M.
    Dueck, Amylou Constance
    Kiladjian, Jean-Jacques
    Xiao, Zhijian
    Slot, Stephanie
    Zweegman, Sonja
    Kerguelen Fuentes, Ana
    Hernandez-Maraver, Dolores
    Dohner, Konstanze
    Harrison, Claire N.
    Radia, Deepti H.
    Muxi, Pablo J.
    Sackman, Federico
    Besses, Carlos
    Cervantes, Francisco
    Johansson, Peter L.
    Andreasson, Bjorn
    Rambaldi, Alessandro
    Barbui, Tiziano
    Vannucchi, Alessandro M.
    Passamonti, Francesco
    Samuelsson, Jan
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Bonatz, Karin
    Reiter, Andreas
    Boyer, Francoise
    Etienne, Gabriel
    Ianotto, Jean-Christophe
    Ranta, Dana
    Roy, Lydia
    Cahn, Jean-Yves
    Maldonado, Norman I.
    Barosi, Giovanni
    Ferrari, Maria
    Cannon, Keith
    te Boekhorst, Peter
    Schouten, Harry C.
    Pahl, Heike L.
    Griesshammer, Martin
    Stegelmann, Frank
    Lehmann, Thomas
    Xu, Zefeng
    Zhang, Yue
    Sun, Xiujuan
    Xu, Junqing
    Zhang, Peihong
    Mesa, Ruben
    Symptom Severity and Clinical Variables of Polycythemia Vera Patients with Splenomegaly, Phlebotomy Requirements and/or Hydroxyurea Use: a Retrospective Evaluation of 1334 Patients2014In: Blood, ISSN 0006-4971, E-ISSN 1528-0020, Vol. 124, no 21Article in journal (Other academic)
  • 34. Geyer, Holly
    et al.
    Scherber, Robyn
    Kosiorek, Heidi
    Dueck, Amylou C
    Kiladjian, Jean-Jacques
    Xiao, Zhijian
    Slot, Stefanie
    Zweegman, Sonja
    Sackmann, Federico
    Fuentes, Ana Kerguelen
    Hernández-Maraver, Dolores
    Döhner, Konstanze
    Harrison, Claire N
    Radia, Deepti
    Muxi, Pablo
    Besses, Carlos
    Cervantes, Francisco
    Johansson, Peter L
    Andreasson, Bjorn
    Rambaldi, Alessandro
    Barbui, Tiziano
    Bonatz, Karin
    Reiter, Andreas
    Boyer, Francoise
    Etienne, Gabriel
    Ianotto, Jean-Christophe
    Ranta, Dana
    Roy, Lydia
    Cahn, Jean-Yves
    Maldonado, Norman
    Barosi, Giovanni
    Ferrari, Maria L
    Gale, Robert Peter
    Birgegard, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Xu, Zefeng
    Zhang, Yue
    Sun, Xiujuan
    Xu, Junqing
    Zhang, Peihong
    Te Boekhorst, Peter A W
    Commandeur, Suzan
    Schouten, Harry
    Pahl, Heike L
    Griesshammer, Martin
    Stegelmann, Frank
    Lehmann, Thomas
    Senyak, Zhenya
    Vannucchi, Alessandro M
    Passamonti, Francesco
    Samuelsson, Jan
    Mesa, Ruben A
    Symptomatic Profiles of Patients With Polycythemia Vera: Implications of Inadequately Controlled Disease2016In: Journal of Clinical Oncology, ISSN 0732-183X, E-ISSN 1527-7755, Vol. 34, no 2, p. 151-+Article in journal (Refereed)
    Abstract [en]

    PURPOSE: Polycythemia vera (PV) is a myeloproliferative neoplasm (MPN) associated with disabling symptoms and a heightened risk of life-threatening complications. Recent studies have demonstrated the effectiveness of JAK inhibitor therapy in patients with PV patients who have a history of prior hydroxyurea (HU) use (including resistance or intolerance), phlebotomy requirements, and palpable splenomegaly. We aimed to determine how these features contribute alone and in aggregate to the PV symptom burden.

    PATIENTS AND METHODS: Through prospective evaluation of 1,334 patients with PV who had characterized symptom burden, we assessed patient demographics, laboratory data, and the presence of splenomegaly by disease feature (ie, known HU use, known phlebotomy requirements, splenomegaly).

    RESULTS: The presence of each feature in itself is associated with a moderately high symptom burden (MPN symptom assessment form [SAF] total symptom score [TSS] range, 27.7 to 29.2) that persists independent of PV risk category. In addition, symptoms incrementally increase in severity with the addition of other features. Patients with PV who had all three features (PV-HUPS) faced the highest total score (MPN-SAF TSS, 32.5) but had similar individual symptom scores to patients with known HU use (PV-HU), known phlebotomy (PV-P), and splenomegaly (PV-S).

    CONCLUSION: The results of this study suggest that patients with PV who have any one of the features in question (known HU use, known phlebotomy, or splenomegaly) have significant PV-associated symptoms. Furthermore, it demonstrates that many PV symptoms remain severe independent of the number of features present.

  • 35. Griesshammer, M.
    et al.
    Gugliotta, L.
    Harrison, C.
    Besses, C. R.
    Kiladjian, J-J
    Coll, R.
    Smith, J.
    Birgegard, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    The exels study: Real-world cytoreductive treatment patterns for essential thrombocythaemia in Europe (analysis of 3643 patients)2013In: Onkologie (Basel), ISSN 0378-584X, E-ISSN 1423-0240, Vol. 36, no Suppl. 7, p. 111-111Article in journal (Other academic)
  • 36.
    Griesshammer, M.
    et al.
    Johannes Wesling Klinikum, Minden, Germany..
    Stegelmann, F.
    Univ Hosp Ulm, Dept Internal Med 3, Ulm, Germany..
    Mohr, A.
    Matzdorff, A.
    Caritasklin St Theresia, Saarbrucken, Germany..
    Besses, C.
    Hosp del Mar, Dept Haematol, IMIM, Barcelona, Spain..
    Gugliotta, L.
    St Orsola Malpighi Hosp, Dept Haematol, Bologna, Italy..
    Harrison, C.
    Guys & St Thomas NHS Fdn Trust, Dept Haematol, London, England..
    Kiladjian, J. J.
    Hop St Louis, AP HP, Ctr Invest Clin, Paris, France..
    Hamdani, M.
    Shire Pharmaceut, Global Biometr, Wayne, NJ USA..
    Achenbach, H.
    Shire GmbH, Res & Dev, Zug, Switzerland..
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Treatment of essential thrombocythaemia in Europe: an observational study of 3649 high-risk patients in EXELS2015In: Oncology Research and Treatment, ISSN 2296-5270, Vol. 38, p. 216-216Article in journal (Other academic)
  • 37. Gugliotta, L.
    et al.
    Specchia, G.
    Gaidano, G.
    Scalzulli, P. R.
    Tieghi, A.
    Kiladjian, J-J
    Besses, C.
    Griesshammer, M.
    Harrison, C.
    Hamdani, M.
    Achenbach, H.
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Treatment Of Essential Thrombocythaemia In Europe: An Observational Study Of 3649 High-Risk Patients In Exels2015In: Haematologica, ISSN 0390-6078, E-ISSN 1592-8721, Vol. 100, p. 43-43Article in journal (Other academic)
  • 38.
    Gugliotta, L.
    et al.
    St Orsola Malpighi Hosp, Dept Haematol L & A Seragnoli, Bologna, Italy..
    Tortorella, G.
    Arcispedale Santa Maria Nuova IRCCS, Reggio Emilia, Italy..
    Besses, C.
    Hosp del Mar IMIM, Dept Haematol, Barcelona, Spain..
    Griesshammer, M.
    Johannes Wesling Med Ctr, Hematol & Oncol, Minden, Germany..
    Harrison, C.
    Guys & St Thomas NHS Fdn Trust, Dept Haematol, London, England..
    Kiladjian, J. J.
    Hop St Louis, APHP, Ctr Invest Clin, Paris, France..
    Wu, J.
    Shire Pharmaceut, Global Biometr, Lexington, MA USA..
    Achenbach, H.
    Shire GmbH, Res & Dev, Zug, Switzerland..
    Marelli, C.
    Shire GmbH, Res & Dev, Zug, Switzerland..
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Palpitations And Arrhythmia In 3649 High-Risk Patients With Essential Thrombocythemia: Results From The Prospective Long-Term Observational Exels Study2016In: Haematologica, ISSN 0390-6078, E-ISSN 1592-8721, Vol. 101, p. 557-557Article in journal (Other academic)
  • 39. Gugliotta, Luigi
    et al.
    Besses, Carlos
    Griesshammer, Martin
    Harrison, Claire
    Kiladjian, Jean-Jacques
    Coll, Ruth
    Smith, Jonathan
    Abhyankar, Brihad
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Combination therapy of hydroxycarbamide with anagrelide in patients with essential thrombocythemia in the evaluation of Xagrid (R) efficacy and long-term safety study2014In: Haematologica, ISSN 0390-6078, E-ISSN 1592-8721, Vol. 99, no 4, p. 679-687Article in journal (Refereed)
    Abstract [en]

    Available information is limited regarding the use of cytoreductive combination therapy in high-risk patients with essential thrombocythemia. This analysis aims to evaluate the clinical relevance and patterns of cytoreductive combination treatment in European high-risk patients with essential thrombocythemia in the Evaluation of Xagrid (R) Efficacy and Long-term Safety study. Of 3643 patients, 347 (9.5%) received combination therapy. Data were recorded at each 6-month update. Of 347 patients who received combination therapy, 304 (87.6%) received hydroxycarbamide + anagrelide. Monotherapies received before this combination were hydroxycarbamide (n=167, 54.9%) and anagrelide (n=123, 40.5%). Median weekly doses of hydroxycarbamide and anagrelide were: 7000 and 10.5 mg when used as prior monotherapy; 3500 and 7.0 mg when used as add-on treatment. Overall, median platelet counts were 581x10(9)/L and 411x10(9)/L before and after starting hydroxycarbamide + anagrelide, respectively. In patients with paired data (n=153), the number of patients with platelet counts less than 400x10(9)/L increased from 33 (21.6%) to 74 (48.4%; P<0.0001), and with platelet counts less than 600x10(9)/L, from 82 (53.6%) to 132 (86.3%; P<0.0001). Hydroxycarbamide + anagrelide was discontinued in 158 patients: 76 (48.1%) stopped hydroxycarbamide, 59 (37.3%) stopped anagrelide, 19 (12.0%) stopped both and 4 (2.5%) had another therapy added. The most frequent reasons for discontinuation were intolerance/side-effects, lack of efficacy, and therapeutic strategy. Combination therapy, usually hydroxycarbamide + anagrelide, is used in approximately 10% of all high-risk patients with essential thrombocythemia and may be a useful approach in treating patients for whom monotherapy is unsatisfactory.

  • 40. Gugliotta, Luigi
    et al.
    Besses, Carlos
    Griesshammer, Martin
    Harrison, Claire N.
    Kiladjian, Jean-Jacques
    Coll, Ruth
    Smith, Jonathan
    Abhyankar, Brihad
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Combination of Cytoreductive Therapies in Patients with Essential Thrombocythemia: A Preliminary Report From the EXELS Study2012In: Blood, ISSN 0006-4971, E-ISSN 1528-0020, Vol. 120, no 21, p. 2842-Article in journal (Refereed)
  • 41. Harrison, C.
    et al.
    Besses, C.
    Kiladjian, J. J.
    Griesshammer, M.
    Gugliotta, L.
    Coll, R.
    Smith, J.
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Cytoreductive treatment patterns for essential thrombocythaemia in Europe: analysis of 3643 patients in the EXELS study2013In: British Journal of Haematology, ISSN 0007-1048, E-ISSN 1365-2141, Vol. 161, no SI, p. 17-17Article in journal (Other academic)
  • 42. Harrison, C.
    et al.
    Tiplady, C.
    Fernandes, S.
    Knechtli, C.
    Sadullah, S.
    Besses, C.
    Greisshammer, M.
    Gugliotta, L.
    Kiladjian, J. J.
    Hamdani, M.
    Achenbach, H.
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Treatment of essential thrombocythaemia in Europe: an observational study of 3649 high-risk patients in EXELS2015In: British Journal of Haematology, ISSN 0007-1048, E-ISSN 1365-2141, Vol. 169, no S1, p. 7-7Article in journal (Other academic)
  • 43. Hedenus, Michael
    et al.
    Karlsson, Torbjörn
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Ludwig, Heinz
    Rzychon, Beate
    Felder, Marcel
    Roubert, Bernard
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Intravenous iron alone resolves anemia in patients with functional iron deficiency and lymphoid malignancies undergoing chemotherapy2014In: Medical Oncology, ISSN 1357-0560, E-ISSN 1559-131X, Vol. 31, no 12, p. 302-Article in journal (Refereed)
    Abstract [en]

    This randomized trial evaluated ferric carboxymaltose without erythropoiesis-stimulating agents (ESA) for correction of anemia in cancer patients with functional iron deficiency. Patients on treatment for indolent lymphoid malignancies, who had anemia [hemoglobin (Hb) 8.5-10.5 g/dL] and functional iron deficiency [transferrin saturation (TSAT) <= 20 %, ferritin >30 ng/mL (women) or >40 ng/mL (men)], were randomized to ferric carboxymaltose (1,000 mg iron) or control. Primary end point was the mean change in Hb from baseline to weeks 4, 6 and 8 without transfusions or ESA. Difficulties with patient recruitment led to premature termination of the study. Seventeen patients (8 ferric carboxymaltose and 9 control) were included in the analysis. In the ferric carboxymaltose arm, mean Hb increase was significantly higher versus control at week 8 (p = 0.021). All ferric carboxymaltose- treated patients achieved an Hb increase >1 g/dL (control 6/9; p = 0.087), and mean TSAT was >20 % from week 2 onwards. No treatment-related adverse events were reported. In conclusion, ferric carboxymaltose without ESA effectively increased Hb and iron status in this small patient population.

  • 44. Johansson, Peter
    et al.
    Mesa, Ruben
    Scherber, Robyn
    Abelsson, Johanna
    Samuelsson, Jan
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Andréasson, Björn
    Association between quality of life and clinical parameters in patients with myeloproliferative neoplasms2012In: Leukemia and Lymphoma, ISSN 1042-8194, E-ISSN 1029-2403, Vol. 53, no 3, p. 441-444Article in journal (Refereed)
    Abstract [en]

    The Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) is a validated quality of life (QoL) instrument. In our Swedish cohort of 114 patients the symptomatic burden was found to be severe, with fatigue reported in 88% of the patients and reduced QoL in the majority of patients. Patients with primary myelofibrosis had the highest scores, low QoL, for most MPN-SAF items, compared to patients with polycythemia vera and essential thrombocythemia. Higher age showed significant associations with the BFI (Brief Fatigue Inventory) score, early satiety, concentration problems, dizziness, insomnia, cough and weight loss. Blood values, disease duration and myelosuppressive treatment did not significantly associate with any of the MPN-SAF items, with the exception of higher hemoglobin, which correlated with sad mood. Male patients with MPN scored significantly higher as regards sexual problems and weight loss compared to female patients. Overall, the MPN-SAF was found to be a valid instrument for assessing symptomatic burden among this population.

  • 45. Kiladjian, Jean-Jacques
    et al.
    Besses, Carlos
    Griesshammer, Martin
    Gugliotta, Luigi
    Harrison, Claire
    Coll, Ruth
    Smith, Jonathan
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Efficacy and Safety of Cytoreductive Therapies in Patients with Essential Thrombocythaemia Aged > 80 Years: An Interim Analysis of the EXELS Study2013In: Clinical drug investigation, ISSN 1173-2563, E-ISSN 1179-1918, Vol. 33, no 1, p. 55-63Article in journal (Refereed)
    Abstract [en]

    Background The median age of patients diagnosed with essential thrombocythaemia (ET) is 65-70 years but the management of very elderly patients (aged >80 years) with ET has not been well characterized. Objective This study aimed to document the treatment patterns of very elderly patients with ET in a multinational, real-world setting. Study Design EXELS (Evaluation of Xagrid Efficacy and Long-term Safety) is a phase IV observational study, designed to monitor the efficacy and safety of cytoreductive therapies in clinical practice. In total, 3,598 high-risk patients with ET were recruited from May 2005 to April 2009, in 13 European countries. Data were collected at registration and every 6 months thereafter for 5 years. This analysis was performed on a data-cut taken approximately 2 years after the last patient was registered. Patients In total, 395 patients aged >80 years at registration into EXELS were included in the analysis; of these, 42.2 % had experienced a previous thrombohaemorrhagic event. Results At registration, the most frequently prescribed cytoreductive therapy for patients aged >80 years was hydroxycarbamide (HC), which accounted for 82.8 % of patients whereas anagrelide use was less frequent (8.6 %). Very elderly patients were more likely to be switched from anagrelide than from HC (47.1 vs. 17.4 %; 95 % confidence interval for difference in proportion 12.4-46.9; Chi-squared test p < 0.001). Median platelet count during treatment was similar to 430 x 10(9)/L. In patients aged >80 years, the main reason for switch was intolerance/side effects (34.1 %); 0/16 patients reported treatment with anagrelide was non-efficacious compared with 8/57 (14 %) patients receiving HC, and 7/16 (43.8 %) anagrelide patients switched because of intolerance versus 18/57 (31.6 %) patients receiving HC. At least one predefined clinical event (PDE) was experienced by 27.3 % of patients aged >80 years. The most common PDEs reported in the very elderly age group were death (non-PDE related; 11.1 %), other cardiovascular symptoms (5.8 %), haematological transformation (3.8 %), congestive heart failure (3.3 %), myocardial infarction and angina (2.8 %), and thromboembolic events (6.3 %). Conclusion Well-tolerated and effective cytoreductive therapy has been achieved in patients aged >80 years by following individual treatment modalities that appear in agreement with the recent European LeukemiaNet (ELN) guidelines. Clinical Trial Registration Registered as ClinicalTrials.gov identifier NCT00567502; Protocol No: SPD422-401.

  • 46.
    Lönnberg, Maria
    et al.
    Uppsala University, Disciplinary Domain of Science and Technology, Chemistry, Department of Chemistry - BMC, Analytical Chemistry.
    Andrén, Maria
    MAIIA Diagnostics, Uppsala Sweden.
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Drevin, Malin
    MAIIA Diagnostics, Uppsala Sweden.
    Garle, Mats
    Doping Control Laboratory, Karolinska University Hospital, Stockholm Sweden.
    Carlsson, Jan
    Uppsala University, Disciplinary Domain of Science and Technology, Chemistry, Department of Chemistry - Ångström, Physical Chemistry.
    Rapid detection of erythropoiesis-stimulating agents in urine and serum2012In: Analytical Biochemistry, ISSN 0003-2697, E-ISSN 1096-0309, Vol. 420, no 2, p. 101-114Article in journal (Refereed)
    Abstract [en]

    A rapid and easy-to-use test kit, EPO WGA MAIIA, which can be used for distinguishing various endogenous human erythropoietins (hEPO) and several recombinant hEPOs and EPO analogues, has been evaluated. The test is based on chromatographic separation of the glycosylated isoforms of EPO using wheat germ agglutinin (WGA), and a sensitive immunoassay utilizing anti-EPO carbon black nanostrings and image scanning for quantification. All the reactions take place along the porous layer of a lateral flow micro-column containing WGA and anti-EPO zones. The presence of molecules resembling hEPO, like Mircera, was detected by the aberrant affinity interaction with the antibody zone on the strip. It was possible to distinguish nine recombinant hEPO expressed in hamster and human cell-lines, and also Aranesp and Mircera, from endogenous urine hEPO. The required amount of EPO in the samples, a few pg, is very low compared to other methods for EPO isoform identification. This EPO isoform determination method opens the possibility to monitor recombinant EPO therapy for clinical research and seems to be a valuable candidate to the arsenal of EPO doping control tests.

  • 47.
    Lönnberg, Maria
    et al.
    Uppsala University, Disciplinary Domain of Science and Technology, Chemistry, Department of Chemistry - BMC, Analytical Chemistry.
    Garle, Mats
    Doping Control Laboratory, Karolinska University Hospital, Stockholm, Sweden.
    Lönnberg, Lina
    Department of Ecology, Swedish University of Agricultural Sciences, Uppsala, Sweden.
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Patients with anaemia can shift from kidney to liver production of erythropoietin as shown by glycoform analysis2013In: Journal of Pharmaceutical and Biomedical Analysis, ISSN 0731-7085, E-ISSN 1873-264X, Vol. 81-82, p. 187-192Article in journal (Refereed)
    Abstract [en]

    The primary production site of erythropoietin (EPO) is shifted from the liver to the kidney shortly after birth. Under conditions of lost or reduced kidney production, it is valuable to measure the production capacity of the liver. However, there is a lack of urine or serum based methods that can distinguish endogenous EPO produced in different cell types. Here is presented a method based on chromatographic interaction with the lectin wheat germ agglutinin (WGA) that can distinguish presumably liver-produced EPO, found in anaemic patients receiving epoetin and darbepoetin, from kidney-produced EPO found in healthy individuals.

    All the tested samples from haemodialysis patients with end-stage renal disease showed a presence of liver EPO. In some samples, the liver-produced EPO made up 90–100% of total EPO at a concentration of 8–10 ng/L in urine, which indicates that the liver has a quite high production capacity, although not adequate for the degree of anaemia.

    This glycoform analysis has made it possible to affirm that some anaemic patients can increase their liver-production of EPO. The use of such a method can give better insight into the regulation of non-renal endogenous EPO production, a potential source of EPO intended to replace administration of exogenous EPO.

  • 48.
    Marchetti, M.
    et al.
    Osped Cardinal Massaia, Asti, Italy..
    Barosi, G.
    IRCCS Policlin San Matteo Fdn, Pavia, Italy..
    Cervantes, F.
    Hosp Clin Barcelona, IDIBAPS, Barcelona, Spain..
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology. Univ Uppsala Hosp, Uppsala, Sweden..
    Griesshammer, M.
    Johannes Wesling Klinikum Minden, Minden, Germany..
    Harrison, C.
    Guys & St Thomas NHS Fdn, London, England..
    Hehlmann, R.
    Heidelberg Univ, Med Fak Mannheim, Mannheim, Germany..
    Kiladjian, J-J
    Ctr Invest Clin CIC 1427, Paris, France..
    Kroeger, N.
    Univ Hosp Hamburg Eppendorf, Hamburg, Germany..
    McMullin, M. F.
    Queens Univ Belfast, Belfast, Antrim, North Ireland..
    Passamonti, F.
    Univ Florence, Dept Expt & Clin Med, Florence, Italy..
    Vannucchi, A.
    Osped Circolo & Fdn Macchi, Varese, Italy..
    Barbui, T.
    Osped Papa Giovanni XXIII, Res Fdn, Bergamo, Italy..
    Which patients with myelofibrosis should receive ruxolitinib therapy?: ELN-SIE evidence-based recommendations2017In: Leukemia, ISSN 0887-6924, E-ISSN 1476-5551, Vol. 31, no 4, p. 882-888Article in journal (Refereed)
    Abstract [en]

    Ruxolitinib is an oral Janus-activated kinase 1 (JAK1)/JAK2 inhibitor approved for the treatment of patients with myelofibrosis based on the results of two randomized clinical trials. However, discordant indications were provided by regulatory agencies and scientific societies for selecting the most appropriate candidates to this drug. The European LeukemiaNet and the Italian Society of Hematology shared the aim of building evidence-based recommendations for the use of ruxolitinib according to the GRADE methodology. Eighteen patient-intervention-comparator-outcome profiles were listed, each of them comparing ruxolitinib to other therapies with the aim of improving one of the three clinical outcomes: (a) splenomegaly, (b) disease-related symptoms, and (c) survival. Ruxolitinib was strongly recommended for improving symptomatic or severe (415 cm below the costal margin) splenomegaly in patients with an International Prognostic Scoring System (IPSS)/dynamic IPSS risk intermediate 2 or high. Ruxolitinib was also strongly recommended for improving systemic symptoms in patients with an MPN10 score 444, refractory severe itching, unintended weight loss not attributable to other causes or unexplained fever. Because of weak evidence, the panel does not recommend ruxolitinib therapy for improving survival. Also, the recommendations given above do not necessarily apply to patients who are candidates for allogeneic stem cell transplant.

  • 49.
    Scherber, R.
    et al.
    OHSU, Hematol & Oncol, Portland, OR USA.;Mayo Clin, Hematol & Oncol, Scottsdale, AZ USA..
    Dueck, A.
    Mayo Clin, Phoenix, AZ USA..
    Geyer, H.
    Mayo Clin, Phoenix, AZ USA.;Mayo Clin, Internal Med, Scottsdale, AZ USA..
    Kosiorek, H.
    Mayo Clin, Phoenix, AZ USA..
    Kiladjian, J. J.
    Hosp St Louis, Paris, France..
    Slot, S.
    Vrije Univ Amsterdam, Med Ctr, Amsterdam, Netherlands..
    Zweegman, S.
    Vrije Univ Amsterdam, Med Ctr, Amsterdam, Netherlands..
    Boekhorst, P.
    Erasmus Med Cener, Rotterdam, Netherlands..
    Schouten, H.
    Maastricht Univ, Med Ctr, Maastricht, Netherlands..
    Sackmann, F.
    Fundaleu, Buenos Aires, DF, Argentina..
    Fuentes, A.
    Univ Hosp La Paz, Madrid, Spain..
    Hernandez-Maraver, D.
    Univ Hosp La Paz, Madrid, Spain..
    Pahl, H.
    Univ Hosp Freiburg, Freiburg, Germany..
    Stegelmann, F.
    Univ Hosp Ulm, Ulm, Germany..
    Doehner, K.
    Univ Hosp Ulm, Ulm, Germany..
    Bonatz, K.
    Med Klin, Mannheim, Germany..
    Reiter, A.
    Med Klin, Mannheim, Germany..
    Boyer, F.
    Ctr Hosp Univ, Angers, France..
    Etienne, G.
    Ctr Hosp Univ, Angers, France..
    Ianotto, J. C.
    Univ Hosp, Brest, France..
    Ranta, D.
    Univ Hosp, Nancy, France..
    Roy, L.
    Ctr Hosp Univ, Poitiers, France..
    Cahn, J. Y.
    Ctr Hosp Univ, Grenoble, France..
    Harrison, C.
    Guys & St Thomas NHS Fdn Trust, London, England..
    Radia, D.
    Guys & St Thomas NHS Fdn Trust, London, England..
    Muxi, P.
    Hosp Britanico, Montevideo, Uruguay..
    Maldonado, N.
    Univ Puerto Rico, Sch Med, San Juan, PR 00936 USA..
    Besses, C.
    Hosp Mar, Barcelona, Spain..
    Cervantes, F.
    Univ Barcelona, Barcelona, Spain..
    Johansson, P.
    NU Hosp Org, Uddevalla, Sweden..
    Barosi, G.
    IRCCS Policlin S Matteo Fdn, Pavia, Italy..
    Vannucchi, A.
    Osped Circolo Varese, Varese, Italy..
    Passamonti, F.
    Fdn IRCCS Policlin San Matteo, Pavia, Italy..
    Andreasson, B.
    NU Hosp Org, Uddevalla, Sweden..
    Samuelsson, J.
    Stockholm South Hosp, Stockholm, Sweden..
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Sun, X.
    Chinese Acad Med Sci, Tianjin, Peoples R China.;Peking Union Med Coll, Tianjin, Peoples R China..
    Xu, J.
    Chinese Acad Med Sci, Tianjin, Peoples R China.;Peking Union Med Coll, Tianjin, Peoples R China..
    Zhang, P.
    Chinese Acad Med Sci, Tianjin, Peoples R China.;Peking Union Med Coll, Tianjin, Peoples R China..
    Xu, Z.
    Chinese Acad Med Sci, Tianjin, Peoples R China.;Peking Union Med Coll, Tianjin, Peoples R China..
    Barbui, T.
    Osped Riuniti Bergamo, Bergamo, Italy.;Osped Riuniti Bergamo, Bergamo, Italy..
    Senyak, Z.
    MPN Forum, Asheville, NC USA..
    Grieshammer, M.
    Johannes Wesling Klinikum, Minden, Germany..
    Rambaldi, A.
    Osped Riuniti Bergamo, Bergamo, Italy..
    Ferrari, M.
    Osped Riuniti Bergamo, Bergamo, Italy..
    Lehmann, T.
    Univ Basel Hosp, Basel, Switzerland..
    Mesa, R.
    Mayo Clin, Phoenix, AZ USA..
    SYMPTOMS, RISK CLASSIFICATION, AND SPLEEN SIZE IN JAK2 INHIBITOR-NAIVE MYELOFIBROSIS: IMPLICATIONS FOR JAK2 INHIBITOR TREATMENT2016In: Haematologica, ISSN 0390-6078, E-ISSN 1592-8721, Vol. 101, p. 557-558Article in journal (Other academic)
  • 50. Scotch, Allison H
    et al.
    Kosiorek, Heidi
    Scherber, Robyn
    Dueck, Amylou C
    Slot, Stefanie
    Zweegman, Sonja
    Boekhorst, Peter A W Te
    Commandeur, Suzan
    Schouten, Harry
    Sackmann, Federico
    Fuentes, Ana Kerguelen
    Hernández-Maraver, Dolores
    Pahl, Heike L
    Griesshammer, Martin
    Stegelmann, Frank
    Döhner, Konstanze
    Lehmann, Thomas
    Bonatz, Karin
    Reiter, Andreas
    Boyer, Francoise
    Etienne, Gabriel
    Ianotto, Jean-Christophe
    Ranta, Dana
    Roy, Lydia
    Cahn, Jean-Yves
    Harrison, Claire N
    Radia, Deepti
    Muxi, Pablo
    Maldonado, Norman
    Besses, Carlos
    Cervantes, Francisco
    Johansson, Peter L
    Barbui, Tiziano
    Barosi, Giovanni
    Vannucchi, Alessandro M
    Paoli, Chiara
    Passamonti, Francesco
    Andreasson, Bjorn
    Ferrari, Maria L
    Rambaldi, Alessandro
    Samuelsson, Jan
    Birgegård, Gunnar
    Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences, Haematology.
    Xiao, Zhijian
    Xu, Zefeng
    Zhang, Yue
    Sun, Xiujuan
    Xu, Junqing
    Kiladjian, Jean-Jacques
    Zhang, Peihong
    Gale, Robert Peter
    Mesa, Ruben A
    Geyer, Holly L
    Symptom burden profile in myelofibrosis patients with thrombocytopenia: Lessons and unmet needs2017In: Leukemia research: a Forum for Studies on Leukemia and Normal Hemopoiesis, ISSN 0145-2126, E-ISSN 1873-5835, Vol. 63, p. 34-40Article in journal (Refereed)
    Abstract [en]

    Myelofibrosis is a myeloproliferative neoplasm associated with progressive cytopenias and high symptom burden. MF patients with thrombocytopenia have poor prognosis but the presence of thrombocytopenia frequently precludes the use of JAK2 inhibitors. In this study, we assessed quality of life and symptom burden in 418 MF patients with (n = 89) and without (n = 329) thrombocytopenia using prospective data from the MPN-QOL study group database, including the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) and Total Symptom Score (MPN10). Thrombocytopenia, defined as platelet count < 100 x10(9)/L (moderate 51-100 x 10(9)/L; severe <= 50 x10(9)/L), was associated with anemia (76% vs. 45%, p < 0.001), leukopenia (29% vs. 11%, p < 0.001), and need for red blood cell transfusion (35% vs. 19%, p = 0.002). Thrombocytopenic patients had more fatigue, early satiety, inactivity, dizziness, sad mood, cough, night sweats, itching, fever, and weight loss; total symptom scores were also higher (33 vs. 24, p < 0.001). Patients with severe thrombocytopenia were more likely to have anemia (86% vs. 67%, p = 0.04), leukopenia (40% vs. 20%, p = 0.04), and transfusion requirements (51% vs. 20%, p = 0.002) but few differences in symptoms when compared to patients with moderate thrombocytopenia. These results suggest that MF patients with thrombocytopenia experience greater symptomatic burden than MF patients without thrombocytopenia and may benefit from additional therapies.

12 1 - 50 of 57
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