A randomized study of pomalidomide vs placebo in persons with myeloproliferative neoplasm-associated myelofibrosis and RBC-transfusion dependence

Leukemia

Volumn 31, Issue 4, 2017, Pages 896-902

  Tefferi, A ^a   Al Ali, H K ^b   Barosi, G ^c   Devos, T ^d   Gisslinger, H ^e   Jiang, Q ^f   Kiladjian, J J ^g   Mesa, R ^h   Passamonti, F ⁱ   Ribrag, V ^j   Schiller, G ^k   Vannucchi, A M ^l,m   Zhou, D ⁿ   Reiser, D ^o   Zhong, J ^o   Gale, R P ^o,p  

^a MAYO CLINIC   (United States)

^b UNIVERSITY HOSPITAL LEIPZIG   (Germany)

^c FONDAZIONE IRCCS POLICLINICO SAN MATTEO   (Italy)

^d UNIVERSITY HOSPITALS LEUVEN   (Belgium)

^e MEDICAL UNIVERSITY OF VIENNA   (Austria)

^f PEKING UNIVERSITY PEOPLE S HOSPITAL   (China)

^g SAINT LOUIS HOSPITAL   (France)

^h MAYO CLINIC   (United States)

ⁱ UNIVERSITY OF INSUBRIA   (Italy)

^j UNIVERSITÉ PARIS SACLAY   (France)

^k UNIVERSITY OF CALIFORNIA   (United States)

^l CAREGGI UNIVERSITY HOSPITAL   (Italy)

^m UNIVERSITY OF FLORENCE   (Italy)

ⁿ PEKING UNION MEDICAL COLLEGE HOSPITAL   (China)

^o CELGENE CORPORATION   (United States)

^p IMPERIAL COLLEGE LONDON   (United Kingdom)

more..

Author keywords

[No Author keywords available]

Indexed keywords

PLACEBO; POMALIDOMIDE; BIOLOGICAL MARKER; IMMUNOLOGIC FACTOR; THALIDOMIDE;

ADULT; AGE; AGED; ANEMIA; ARTICLE; CLINICAL OUTCOME; CONTROLLED STUDY; DIARRHEA; DOUBLE BLIND PROCEDURE; DRUG EFFICACY; DRUG SAFETY; EDEMA; ERYTHROCYTE TRANSFUSION; FATIGUE; FEMALE; FEVER; HUMAN; LEUKOCYTE COUNT; MAJOR CLINICAL STUDY; MALE; MULTICENTER STUDY; MYELOFIBROSIS; MYELOPROLIFERATIVE NEOPLASM; NEUTROPENIA; PHASE 3 CLINICAL TRIAL; PNEUMONIA; PROSPECTIVE STUDY; RANDOMIZED CONTROLLED TRIAL; THROMBOCYTE COUNT; THROMBOCYTOPENIA; TREATMENT RESPONSE; ANALOGS AND DERIVATIVES; CLINICAL TRIAL; COMPLICATION; MIDDLE AGED; MYELOPROLIFERATIVE DISORDERS; PHENOTYPE; PRIMARY MYELOFIBROSIS; PROCEDURES; TREATMENT OUTCOME; VERY ELDERLY; WORKFLOW;

ADULT; AGED; AGED, 80 AND OVER; BIOMARKERS; ERYTHROCYTE TRANSFUSION; FEMALE; HUMANS; IMMUNOLOGIC FACTORS; MALE; MIDDLE AGED; MYELOPROLIFERATIVE DISORDERS; PHENOTYPE; PRIMARY MYELOFIBROSIS; THALIDOMIDE; TREATMENT OUTCOME; WORKFLOW;

EID: 84995755297     PISSN: 08876924     EISSN: 14765551     Source Type: Journal    
DOI: 10.1038/leu.2016.300     Document Type: Article

References (32)

1. Mesa RA, Green A, Barosi G, Verstovsek S, Vardiman J, Gale RP. MPN-associated myelofibrosis (MPN-MF). Leuk Res 2011; 35: 12-13
2. Cervantes F, Dupriez B, Pereira A, Passamonti F, Reilly JT, Morra E et al. New prognostic scoring system for primary myelofibrosis based on a study of the International Working Group for Myelofibrosis Research and Treatment. Blood 2009; 113: 2895-2901
3. Passamonti F, Cervantes F, Vannucchi AM, Morra E, Rumi E, Pereira A 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). Blood 2010; 115: 1703-1708
4. Elena C, Passamonti F, Rumi E, Malcovati L, Arcaini L, Boveri E et al. Red blood cell transfusion-dependency implies a poor survival in primary myelofibrosis irrespective of IPSS and DIPSS. Haematologica 2011; 96: 167-170
5. Tefferi A, Mesa RA, Pardanani A, Hussein K, Schwager S, Hanson CA et al. Red blood cell transfusion need at diagnosis adversely affects survival in primary myelofibrosisincreased serum ferritin or transfusion load does not. Am J Hematol 2009; 84: 265-267
6. Gangat N, Caramazza D, Vaidya R, George G, Begna K, Schwager S 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. J Clin Oncol 2011; 29: 392-397
7. Elkinson S, McCormack PL. Pomalidomide: first global approval. Drugs 2013; 73: 595-604
8. Cervantes F, Alvarez-Larrán A, Hernández-Boluda JC, Sureda A, Torrebadell M, Montserrat E. Erythropoietin treatment of the anaemia of myelofibrosis with myeloid metaplasia: results in 20 patients and review of the literature. Br J Haematol 2004; 127: 399-403
9. Tsiara SN, Chaidos A, Bourantas LK, Kapsali HD, Bourantas KL. Recombinant human erythropoietin for the treatment of anaemia in patients with chronic idiopathic myelofibrosis. Acta Haematol 2007; 117: 156-161
10. Cervantes F, Alvarez-Larrán A, Hernández-Boluda JC, Sureda A, Granell M, Vallansot R et al. Darbepoetin-alpha for the anaemia of myelofibrosis with myeloid metaplasia. Br J Haematol 2006; 134: 184-186
11. Thomas DA, Giles FJ, Albitar M, Cortes JE, Verstovsek S, Faderl S et al. Thalidomide therapy for myelofibrosis with myeloid metaplasia. Cancer 2006; 106: 1974-1984
12. Abgrall JF, Guibaud I, Bastie JN, Flesch M, Rossi JF, Lacotte-Thierry L et al. Groupe Ouest-Est Leucémies et Maladies du Sang (GOELAMS). Thalidomide versus placebo in myeloid metaplasia with myelofibrosis: a prospective, randomized, double-blind, multicenter study. Haematologica 2006; 91: 1027-1032
13. Marchetti M, Barosi G, Balestri F, Viarengo G, Gentili S, Barulli S et al. Low-dose thalidomide ameliorates cytopenias and splenomegaly in myelofibrosis with myeloid metaplasia: a phase II trial. J Clin Oncol 2004; 22: 424-431
14. Mesa RA, Steensma DP, Pardanani A, Li CY, Elliott M, Kaufmann SH et al. A phase 2 trial of combination low-dose thalidomide and prednisone for the treatment of myelofibrosis with myeloid metaplasia. Blood 2003; 101: 2534-2541
15. Tefferi A, Barosi G, Mesa RA, Cervantes F, Deeg HJ, Reilly JT et al. International Working Group (IWG) consensus criteria for treatment response in myelofibrosis with myeloid metaplasia, for the IWG for Myelofibrosis Research and Treatment (IWG-MRT). IWG for Myelofibrosis Research and Treatment (IWG-MRT). Blood 2006; 108: 1497-1503
16. Tefferi A. Primary myelofibrosis: 2013 update on diagnosis, risk-stratification, and management. Am J Hematol 2013; 88: 141-150
17. Quintás-Cardama A, Kantarjian HM, Manshouri T, Thomas D, Cortes J, Ravandi F et al. Lenalidomide plus prednisone results in durable clinical, histopathologic, and molecular responses in patients with myelofibrosis. J Clin Oncol 2009; 27: 4760-4766
18. Thapalibya P, Tefferi A, Pardanani A, Steensma DP, Camoriano J, Wu W et al. International working group for myelofibrosis research and treatment response assessment and long-term follow-up of 50 myelofibrosis patients treated with thalidomide-prednisone based regimens. Am J Hematol 2011; 86: 96-98
19. Santos F, Verstovsek S. Efficacy of ruxolitinib for myelofibrosis. Expert Opin Pharmacother 2014; 15: 1465-1473
20. Lacy MQ, McCurdy AR. Pomalidomide. Blood 2013; 122: 2305-2309
21. Moutouh de Parseval LA, Verhelle D, Glezer E, Jensen-Pergakes K, Ferguson GD, Corral LG et al. Pomalidomide and lenalidomide regulate erythropoiesis and fetal hemoglobin production in human CD34+ cells. J Clin Invest 2008; 118: 248-258
22. Dulmovitis BM, Appiah-Kubi AO, Papain J, Hale J, He M, Al-Abed Y et al. Pomalidomide reverses γ-globulin silencing through the transcriptional reprogramming of adult hematopoietic progenitors. Blood 2016; 127: 1481-1492
23. Tefferi A, Verstovsek S, Barosi G, Passamonti F, Roboz GJ, Gisslinger H et al. Pomalidomide is active in the treatment of anemia associated with myelofibrosis. J Clin Oncol 2009; 20: 4563-4569
24. Mesa RA, Pardanani AD, Hussein K, Wu W, Schwager S, Litzow MR et al. Phase1/-2 study of pomalidomide in myelofibrosis. Am J Hematol 2010; 85: 129-130
25. Begna KH, Mesa RA, Pardanani A, Hogan WJ, Litzow MR, McClure RF et al. A phase-2 trial of low-dose pomalidomide in myelofibrosis. Leukemia 2011; 25: 301-304
26. Gale RP, Barosi G, Barbui T, Cervantes F, Dohner K, Dupriez B et al. What are RBCtransfusion-dependence and-independence? Leuk Res 2011; 35: 8-11
27. Gale RP, Barosi G, Barbui T, Cervantes F, Dohner K, Dupriez B et al. RBC-transfusion guidelines update. Leuk Res 2012; 36: 659-660
28. Gowin KL, Mesa RL. Profile of pomalidomide and its potential in the treatment of myelofibrosis. Ther Clin Risk Manage 2015; 11: 549-556
29. Schlenk R, Stegelmann F, Reiter A, Jost E, Gattermann N, Hebart H et al. Pomalidomide in MPN-associated myelofibrosis. Leukemia 2017; 31: 889-895
30. Andrei M, Sindhu H, Wang JC. Two cases of myelofibrosis with severe thrombocytopenia and symptomology treated with a combination of pomalidomide and ruxolitinib. Leuk Lymphoma 2015; 56: 524-526
31. Tefferi A, Cervantes F, Mesa R, Passamonti F, Verstovsek S, Vannucchi AM et al. Revised response criteria for myelofibrosis: International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) and European LeukemiaNet (ELN) consensus report. Blood 2013; 122: 8
32. Elena C, Passamonti F, Rumi E, Malcovati L et al. Red blood cell transfusiondependency implies a poor survival in primary myelofibrosis irrespective of IPSS and DIPSS. Haematologica 2011; 96: 167-170