Myelofibrosis patients in Belgium: disease characteristics

Abstract Objective: To date, only a small number of epidemiological studies on myelofibrosis have been performed. The current study aimed to characterize the myelofibrosis patient population in Belgium according to pre-defined disease parameters (diagnosis, risk categories, hemoglobin <10 g/dl, spleen size, constitutional symptoms, platelet count, myeloblast count), with a view to obtaining a deeper understanding of the proportion of patients that may benefit from the novel myelofibrosis therapeutic strategies. Methods: A survey was used to collect data on prevalence and disease parameters on all myelofibrosis patients seen at each of 18 participating hematologic centers in 2011. Aggregated data from all centers were used for analysis. Analyses were descriptive and quantitative. Results: A total of 250 patients with myelofibrosis were captured; of these, 136 (54%) were male and 153 (61%) were over 65 years old. One hundred sixty-five (66%) of myelofibrosis patients had primary myelofibrosis and 85 (34%) had secondary myelofibrosis. One hundred ninety-three myelofibrosis patients (77%) had a palpable spleen. About a third of patients (34%) suffered from constitutional symptoms. Two hundred twenty-two (89%) myelofibrosis patients had platelet count ≧50 000/μl and 201 (80%) had platelet count ≧100 000/μl. Of 250 patients, 85 (34%) had a myeloblast count ≧1%. Six (2%) patients had undergone a splenectomy. Thirteen (5·2%) patients had undergone radiotherapy for splenomegaly. Conclusions: The results of this survey provide insight into the characteristics of the Belgian myelofibrosis population. They also suggest that a large proportion of these patients could stand to benefit from the therapies currently under development.

[1]  F. Cervantes How I treat myelofibrosis. , 2014, Blood.

[2]  T. Barbui,et al.  Three-year efficacy, safety, and survival findings from COMFORT-II, a phase 3 study comparing ruxolitinib with best available therapy for myelofibrosis. , 2013, Blood.

[3]  J. Dipersio,et al.  Efficacy, safety, and survival with ruxolitinib in patients with myelofibrosis: results of a median 3-year follow-up of COMFORT-I , 2013, Haematologica.

[4]  L. Knoops,et al.  Disease characteristics in Belgian myelofibrosis patients and management guidelines anno 2013 , 2013 .

[5]  M. Cazzola,et al.  Phase 3 Study Of Pomalidomide In Myeloproliferative Neoplasm (MPN)-Associated Myelofibrosis With RBC-Transfusion-Dependence , 2013 .

[6]  H. Kantarjian,et al.  Interim analysis of safety and efficacy of ruxolitinib in patients with myelofibrosis and low platelet counts , 2013, Journal of Hematology & Oncology.

[7]  C. Harrison,et al.  Treatment and management of myelofibrosis in the era of JAK inhibitors , 2013, Biologics : targets & therapy.

[8]  M. Sáez,et al.  Population-based incidence of myeloid malignancies: fifteen years of epidemiological data in the province of Girona, Spain , 2013, Haematologica.

[9]  R. Hoffman,et al.  A comprehensive review and analysis of the effect of ruxolitinib therapy on the survival of patients with myelofibrosis. , 2013, Blood.

[10]  S. Verstovsek,et al.  Investigational Janus kinase inhibitors , 2013, Expert opinion on investigational drugs.

[11]  R. Laborde,et al.  Safety and efficacy of CYT387, a JAK1 and JAK2 inhibitor, in myelofibrosis , 2013, Leukemia.

[12]  S. Verstovsek,et al.  What is next beyond janus kinase 2 inhibitors for primary myelofibrosis? , 2013, Current opinion in hematology.

[13]  A. Tefferi,et al.  Primary myelofibrosis: 2013 update on diagnosis, risk-stratification, and management , 2013 .

[14]  B. Firwana,et al.  Comprehensive review of JAK inhibitors in myeloproliferative neoplasms , 2013, Therapeutic advances in hematology.

[15]  S. Verstovsek,et al.  Assessing efficacy in myelofibrosis treatment: a focus on JAK inhibition , 2012, Expert review of hematology.

[16]  C. Stiller,et al.  Incidence, survival and prevalence of myeloid malignancies in Europe. , 2012, European journal of cancer.

[17]  M. Cazzola,et al.  Long-term outcomes of 107 patients with myelofibrosis receiving JAK1/JAK2 inhibitor ruxolitinib: survival advantage in comparison to matched historical controls. , 2012, Blood.

[18]  A. Mead,et al.  Guideline for the diagnosis and management of myelofibrosis , 2012, British journal of haematology.

[19]  R. Hoffman,et al.  Ruxolitinib: The First FDA Approved Therapy for the Treatment of Myelofibrosis , 2012, Clinical Cancer Research.

[20]  Jason Gotlib,et al.  A double-blind, placebo-controlled trial of ruxolitinib for myelofibrosis. , 2012, The New England journal of medicine.

[21]  Francisco Cervantes,et al.  JAK inhibition with ruxolitinib versus best available therapy for myelofibrosis. , 2012, The New England journal of medicine.

[22]  G. Barosi,et al.  Management of Myeloproliferative Neoplasms: From Academic Guidelines to Clinical Practice , 2012, Current Hematologic Malignancy Reports.

[23]  L. To,et al.  Results of a Phase 2 Study of Pacritinib (SB1518), a Novel Oral JAK2 Inhibitor, In Patients with Primary, Post-Polycythemia Vera, and Post-Essential Thrombocythemia Myelofibrosis , 2011 .

[24]  Catriona Jamieson,et al.  Safety and efficacy of TG101348, a selective JAK2 inhibitor, in myelofibrosis. , 2011, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[25]  M. Griesshammer,et al.  Philadelphia-negative classical myeloproliferative neoplasms: critical concepts and management recommendations from European LeukemiaNet. , 2011, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[26]  F. Passamonti,et al.  DIPSS plus: a refined Dynamic International Prognostic Scoring System for primary myelofibrosis that incorporates prognostic information from karyotype, platelet count, and transfusion status. , 2011, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[27]  T. Petrella,et al.  Twenty-five years of epidemiological recording on myeloid malignancies: data from the specialized registry of hematologic malignancies of Côte d’Or (Burgundy, France) , 2011, Haematologica.

[28]  M. Cazzola,et al.  A dynamic prognostic model to predict survival in primary myelofibrosis: a study by the IWG-MRT (International Working Group for Myeloproliferative Neoplasms Research and Treatment). , 2010, Blood.

[29]  N. Kröger,et al.  Outcome of transplantation for myelofibrosis. , 2010, Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation.

[30]  H. Einsele,et al.  Allogeneic stem cell transplantation after reduced-intensity conditioning in patients with myelofibrosis: a prospective, multicenter study of the Chronic Leukemia Working Party of the European Group for Blood and Marrow Transplantation. , 2009, Blood.

[31]  H. Deeg,et al.  Pomalidomide is active in the treatment of anemia associated with myelofibrosis. , 2009, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[32]  C. Bloomfield,et al.  The 2008 revision of the World Health Organization (WHO) classification of myeloid neoplasms and acute leukemia: rationale and important changes. , 2009, Blood.

[33]  R. Mesa How I treat symptomatic splenomegaly in patients with myelofibrosis. , 2009, Blood.

[34]  O. Abdel-Wahab,et al.  Primary myelofibrosis: update on definition, pathogenesis, and treatment. , 2009, Annual review of medicine.

[35]  R. Mesa,et al.  New prognostic scoring system for primary myelofibrosis based on a study of the International Working Group for Myelofibrosis Research and Treatment. , 2008, Blood.

[36]  D. Rondelli Allogeneic hematopoietic stem cell transplantation for myelofibrosis , 2008, Haematologica.

[37]  G. Barosi,et al.  Life expectancy and prognostic factors in the classic BCR/ABL-negative myeloproliferative disorders , 2008, Leukemia.

[38]  J. Sloan,et al.  The Burden of Fatigue and Quality of Life in Myeloproliferative Disorders (MPDs): An International Internet Based Survey of 1179 MPD Patients. , 2006 .

[39]  R. Mesa,et al.  Palliative goals, patient selection, and perioperative platelet management: Outcomes and lessons from 3 decades of splenectomy for myelofibrosiswith myeloid metaplasia at the Mayo Clinic , 2006, Cancer.

[40]  Mario Cazzola,et al.  A gain-of-function mutation of JAK2 in myeloproliferative disorders. , 2005, The New England journal of medicine.

[41]  Stefan N. Constantinescu,et al.  A unique clonal JAK2 mutation leading to constitutive signalling causes polycythaemia vera , 2005, Nature.

[42]  Sandra A. Moore,et al.  Activating mutation in the tyrosine kinase JAK2 in polycythemia vera, essential thrombocythemia, and myeloid metaplasia with myelofibrosis. , 2005, Cancer cell.

[43]  P. Campbell,et al.  Acquired mutation of the tyrosine kinase JAK2 in human myeloproliferative disorders , 2005, The Lancet.

[44]  A. Elmaagacli,et al.  Outcome of allogeneic stem cell transplantation in patients with myelofibrosis , 2004, Bone Marrow Transplantation.

[45]  P. Johansson,et al.  Trends in the incidence of chronic Philadelphia chromosome negative (Ph‐) myeloproliferative disorders in the city of Göteborg, Sweden, during 1983–99 , 2004, Journal of internal medicine.

[46]  A. Tefferi Myelofibrosis with myeloid metaplasia. , 2000, The New England journal of medicine.

[47]  J. Sloan,et al.  The burden of fatigue and quality of life in myeloproliferative disorders (MPDs) , 2007, Cancer.

[48]  H. Møller,et al.  The incidence and outcome of myeloid malignancies in 2,112 adult patients in southeast England. , 2006, Haematologica.

[49]  H. Møller,et al.  The incidence and outcome of myeloid malignancies in 2,112 adult patients in South East-England. , 2006 .