Is there a role for pomalidomide in the treatment of myelofibrosis?

References

• Tefferi A. Essential thrombocythemia, polycythemia vera, and myelofibrosis: current management and the prospect of targeted therapy. Am J Hematol. 2008;83(6):491–497.
   PubMed Web of Science ®Google Scholar
• Harrison C, Kiladjian -J-J, Al-Ali HK, et al. JAK inhibition with ruxolitinib versus best available therapy for myelofibrosis. N Engl J Med. 2012;366(9):787–798.
   PubMed Web of Science ®Google Scholar
• Levine RL, Gilliland DG. Myeloproliferative disorders. Blood. 2008;112(6):2190–2198.
   PubMed Web of Science ®Google Scholar
• Mesa RA, Li C-Y, Ketterling RP, et al. Leukemic transformation in myelofibrosis with myeloid metaplasia: a single-institution experience with 91 cases. Blood. 2005;105(3):973–977.
   PubMed Web of Science ®Google Scholar
• Vainchenker W, Delhommeau F, Constantinescu SN, et al. New mutations and pathogenesis of myeloproliferative neoplasms. Blood. 2011;118(7):1723–1735.
   PubMed Web of Science ®Google Scholar
• Tefferi A, Lasho TL, Finke CM, et al. CALR vs JAK2 vs MPL-mutated or triple-negative myelofibrosis: clinical, cytogenetic and molecular comparisons. Leukemia. 2014;28(7):1472–1477.
   PubMed Web of Science ®Google Scholar
• Randhawa J, Ostojic A, Vrhovac R, et al. Splenomegaly in myelofibrosis–new options for therapy and the therapeutic potential of Janus kinase 2 inhibitors. J Hematol Oncol. 2012;5:43.
   PubMed Web of Science ®Google Scholar
• Mesa RA, Niblack J, Wadleigh M, et al. The burden of fatigue and quality of life in myeloproliferative disorders (MPDs): an international internet-based survey of 1179 MPD patients. Cancer. 2007;109(1):68–76.
   PubMed Web of Science ®Google Scholar
• Tefferi A. How I treat myelofibrosis. Blood. 2011;117(13):3494–3504.
   PubMed Web of Science ®Google Scholar
• Mesa RA. How I treat symptomatic splenomegaly in patients with myelofibrosis. Blood. 2009;113(22):5394–5400.
   PubMed Web of Science ®Google Scholar
• Geyer HL, Dueck AC, Scherber RM, et al. Impact of inflammation on myeloproliferative neoplasm symptom development. Mediators Inflamm. 2015;2015:9. Article ID: 284706.
   PubMed Web of Science ®Google Scholar
• Cervantes F. How I treat myelofibrosis. Blood. 2014;124(17):2635–2642.
   PubMed Web of Science ®Google Scholar
• Alimam S, McLornan D, Harrison C. The use of JAK inhibitors for low-risk myelofibrosis. Expert Rev Hematol. 2015;8(5):551–553.
   PubMed Web of Science ®Google Scholar
• Martinez-Trillos A, Gaya A, Maffioli M, et al. Efficacy and tolerability of hydroxyurea in the treatment of the hyperproliferative manifestations of myelofibrosis: results in 40 patients. Ann Hematol. 2010;89(12):1233–1237.
   PubMed Web of Science ®Google Scholar
• Quintas-Cardama A, Kantarjian H, Manshouri T, et al. Pegylated interferon alfa-2a yields high rates of hematologic and molecular response in patients with advanced essential thrombocythemia and polycythemia vera. J Clin Oncol. 2009;27(32):5418–5424.
   PubMed Web of Science ®Google Scholar
• Kiladjian JJ, Cassinat B, Chevret S, et al. Pegylated interferon-alfa-2a induces complete hematologic and molecular responses with low toxicity in polycythemia vera. Blood. 2008;112(8):3065–3072.
   PubMed Web of Science ®Google Scholar
• Ianotto J-C, Boyer-Perrard F, Gyan E, et al. Efficacy and safety of pegylated-interferon α-2a in myelofibrosis: a study by the FIM and GEM French cooperative groups. Br J Haematol. 2013;162(6):783–791.
   PubMed Web of Science ®Google Scholar
• Silver RT, Vandris K. Recombinant interferon alpha (rIFN alpha-2b) may retard progression of early primary myelofibrosis. Leukemia. 2009;23(7):1366–1369.
   PubMed Web of Science ®Google Scholar
• Silver RT, Vandris K, Goldman JJ. Recombinant interferon-α may retard progression of early primary myelofibrosis: a preliminary report. Blood. 2011;117(24):6669–6672.
   PubMed Web of Science ®Google Scholar
• Cervantes F, Alvarez-Larran A, Hernandez-Boluda JC, et al. Erythropoietin treatment of the anaemia of myelofibrosis with myeloid metaplasia: results in 20 patients and review of the literature. Br J Haematol. 2004;127(4):399–403.
   PubMed Web of Science ®Google Scholar
• Cervantes F, Alvarez-Larran A, Hernandez-Boluda JC, et al. Darbepoetin-alpha for the anaemia of myelofibrosis with myeloid metaplasia. Br J Haematol. 2006;134(2):184–186.
   PubMed Web of Science ®Google Scholar
• Cervantes F. How I treat splenomegaly in myelofibrosis. Blood Cancer J. 2011;1(10):e37.
   PubMed Web of Science ®Google Scholar
• Barbui T, Barosi G, Birgegard G, et al. Philadelphia-negative classical myeloproliferative neoplasms: critical concepts and management recommendations from European LeukemiaNet. J Clin Oncol. 2011;29(6):761–770.
   PubMed Web of Science ®Google Scholar
• Cervantes F, Hernandez-Boluda JC, Alvarez A, et al. Danazol treatment of idiopathic myelofibrosis with severe anemia. Haematologica. 2000;85(6):595–599.
   PubMed Web of Science ®Google Scholar
• Fontana V, Dudkiewicz P, Ahn ER, et al. Danazol therapy combined with intermittent application of chemotherapy induces lasting remission in myeloproliferative disorder (MPD): an alternative for the elderly with advanced MPD. Hematology. 2011;16(2):90–94.
   PubMed Web of Science ®Google Scholar
• Gowin KL, Dueck AC, Mascarenhas JO, et al. Interim analysis of a phase II pilot trial of ruxolitinib combined with danazol for patients with primary myelofibrosis (MF), post essential thrombocythemia-myelofibrosis (Post ET), and post polycythemia vera myelofibrosis (PV MF) suffering from anemia. Blood. 2014;21(124):3206.
   Google Scholar
• Barugola G, Cavallini A, Lipari G, et al. The role of splenectomy in myelofibrosis with myeloid metaplasia. Minerva Chir. 2010;65(6):619–625.
   PubMed Web of Science ®Google Scholar
• Verstovsek S, Mesa RA, Gotlib J, et al. A double-blind, placebo-controlled trial of ruxolitinib for myelofibrosis. N Engl J Med. 2012;366(9):799–807.
   PubMed Web of Science ®Google Scholar
• Cervantes F, Vannucchi AM, Kiladjian -J-J, et al. Three-year efficacy, safety, and survival findings from COMFORT-II, a phase 3 study comparing ruxolitinib with best available therapy for myelofibrosis. Blood. 2013;122(25):4047–4053.
   PubMed Web of Science ®Google Scholar
• Verstovsek S, Mesa RA, Gotlib J, et al. Efficacy, safety and survival with ruxolitinib in patients with myelofibrosis: results of a median 2-year follow-up of COMFORT-I. Haematologica. 2013;98(12):1865–1871.
   PubMed Web of Science ®Google Scholar
• Verstovsek S, Mesa RA, Gotlib J, et al. Efficacy, safety, and survival with ruxolitinib in patients with myelofibrosis: results of a median 3-year follow-up of COMFORT-I. Haematologica. 2015;100(4):479–488.
   PubMed Web of Science ®Google Scholar
• Vannucchi AM, Kantarjian HM, Kiladjian -J-J, et al. A pooled analysis of overall survival in COMFORT-I and COMFORT-II, 2 randomized phase III trials of ruxolitinib for the treatment of myelofibrosis. Haematologica. 2015;100(9):1139–1145.
   PubMed Web of Science ®Google Scholar
• Talpaz M, Paquette R, Afrin L, et al. Interim analysis of safety and efficacy of ruxolitinib in patients with myelofibrosis and low platelet counts. J Hematol Oncol. 2013;6(1):81.
   PubMed Web of Science ®Google Scholar
• Komrokji RS, Seymour JF, Roberts AW, et al. Results of a phase 2 study of pacritinib (SB1518), a JAK2/JAK2(V617F) inhibitor, in patients with myelofibrosis. Blood. 2015;125(17):2649–2655.
   PubMed Web of Science ®Google Scholar
• Beauverd Y, McLornan DP, Harrison CN. Pacritinib: a new agent for the management of myelofibrosis? Expert Opin Pharmacother. 2015;16(15):2381–2390.
   PubMed Web of Science ®Google Scholar
• Harrison C, Szoke A, Suvorov A, et al. editors. Persist-1: a phase III study of pacritinib (Pac) vs best available therapy (BAT) in primary myelofibrosis (PMF), post-polycythemia vera MF (PPV-MF) or post-essential thrombocythemia MF (PET-MF). Haematologica. 2015;100:103. FERRATA STORTI FOUNDATION VIA GIUSEPPE BELLI 4, 27100 PAVIA, ITALY.
   Web of Science ®Google Scholar
• Pardanani A, Laborde RR, Lasho TL, et al. Safety and efficacy of CYT387, a JAK1 and JAK2 inhibitor, in myelofibrosis. Leukemia. 2013;27(6):1322–1327.
   PubMed Web of Science ®Google Scholar
• Abdelrahman RA, Begna KH, Al-Kali A, et al. Momelotinib treatment-emergent neuropathy: prevalence, risk factors and outcome in 100 patients with myelofibrosis. Br J Haematol. 2015;169(1):77–80.
   PubMed Web of Science ®Google Scholar
• Mascarenhas J. Rationale for combination therapy in myelofibrosis. Best Pract Res Clin Haematol. 2014;27(2):197–208.
   PubMed Web of Science ®Google Scholar
• Bartalucci N, Tozzi L, Bogani C, et al. Co-targeting the PI3K/mTOR and JAK2 signalling pathways produces synergistic activity against myeloproliferative neoplasms. J Cell Mol Med. 2013;17(11):1385–1396.
   PubMed Web of Science ®Google Scholar
• Bartalucci N, Guglielmelli P, Vannucchi AM. Rationale for targeting the PI3K/Akt/mTOR pathway in myeloproliferative neoplasms. Clin Lymphoma Myeloma Leuk. 2013;13(Suppl 2):S307–9.
   PubMedGoogle Scholar
• Durrant ST, Nagler A, Vannucchi AM, et al. An open-label, multicenter, 2-arm, dose-finding, phase 1b study of the combination of ruxolitinib and buparlisib (BKM120) in patients with myelofibrosis: results from HARMONY study. Blood. 2015;126(23):827.
   PubMed Web of Science ®Google Scholar
• Harrison CN, Kiladjian -J-J, Heidel FH, et al. Efficacy, safety, and confirmation of the recommended phase 2 starting dose of the combination of ruxolitinib (RUX) and panobinostat (PAN) in patients (Pts) with myelofibrosis (MF). Blood. 2015;126(23):4060.
   Web of Science ®Google Scholar
• Tibes R, Mesa RA. Targeting hedgehog signaling in myelofibrosis and other hematologic malignancies. J Hematol Oncol. 2014;7:18.
   PubMed Web of Science ®Google Scholar
• Mwirigi A, Galli S, Keohane C, et al. Combination therapy with ruxolitinib plus 5-azacytidine or continuous infusion of low dose cytarabine is feasible in patients with blast-phase myeloproliferative neoplasms. Br J Haematol. 2014;167(5):714–716.
   PubMed Web of Science ®Google Scholar
• Daver NCJ, Zhou L, Pemmaraju N, et al. Ruxolitinib (RUX) in combination with 5-azacytidine (AZA) as therapy for patients (PTS) with myelofibrosis (MF). Haematologica. 2015;100(1):2.
   Google Scholar
• Elliott MA, Mesa RA, Li C-Y, et al. Thalidomide treatment in myelofibrosis with myeloid metaplasia. Br J Haematol. 2002;117(2):288–296.
   PubMed Web of Science ®Google Scholar
• Chou JM, Li C-Y, Tefferi A. Bone marrow immunohistochemical studies of angiogenic cytokines and their receptors in myelofibrosis with myeloid metaplasia. Leuk Res. 2003;27(6):499–504.
   PubMed Web of Science ®Google Scholar
• Tefferi A, Cortes J, Verstovsek S, et al. Lenalidomide therapy in myelofibrosis with myeloid metaplasia. Blood. 2006;108(4):1158–1164.
   PubMed Web of Science ®Google Scholar
• Emanuel RM, Dueck AC, Geyer HL, et al. Myeloproliferative neoplasm (MPN) symptom assessment form total symptom score: prospective international assessment of an abbreviated symptom burden scoring system among patients with MPNs. J Clin Oncol. 2012;30(33):4098–4103.
   PubMed Web of Science ®Google Scholar
• Gorgun G, Calabrese E, Soydan E, et al. Immunomodulatory effects of lenalidomide and pomalidomide on interaction of tumor and bone marrow accessory cells in multiple myeloma. Blood. 2010;116(17):3227–3237.
   PubMed Web of Science ®Google Scholar
• Zhu YX, Kortuem KM, Stewart AK. Molecular mechanism of action of immune-modulatory drugs thalidomide, lenalidomide and pomalidomide in multiple myeloma. Leuk Lymphoma. 2013;54(4):683–687.
   PubMed Web of Science ®Google Scholar
• Lacy MQ, Rajkumar SV. Pomalidomide: a new IMiD with remarkable activity in both multiple myeloma and myelofibrosis. Am J Hematol. 2010;85(2):95–96.
   PubMed Web of Science ®Google Scholar
• Singhal S, Mehta J, Desikan R, et al. Antitumor activity of thalidomide in refractory multiple myeloma. N Engl J Med. 1999;341(21):1565–1571.
   PubMed Web of Science ®Google Scholar
• Merup M, Kutti J, Birgergard G, et al. Negligible clinical effects of thalidomide in patients with myelofibrosis with myeloid metaplasia. Med Oncol. 2002;19(2):79–86.
   PubMed Web of Science ®Google Scholar
• Barosi G, Elliott M, Canepa L, et al. Thalidomide in myelofibrosis with myeloid metaplasia: a pooled-analysis of individual patient data from five studies. Leuk Lymphoma. 2002;43(12):2301–2307.
   PubMed Web of Science ®Google Scholar
• Canepa L, Ballerini F, Varaldo R, et al. Thalidomide in agnogenic and secondary myelofibrosis. Br J Haematol. 2001;115(2):313–315.
   PubMed Web of Science ®Google Scholar
• Mesa RA, Steensma DP, Pardanani A, et al. A phase 2 trial of combination low-dose thalidomide and prednisone for the treatment of myelofibrosis with myeloid metaplasia. Blood. 2003;101(7):2534–2541.
   PubMed Web of Science ®Google Scholar
• Jabbour E, Thomas D, Kantarjian H, et al. Comparison of thalidomide and lenalidomide as therapy for myelofibrosis. Blood. 2011;118(4):899–902.
   PubMed Web of Science ®Google Scholar
• Marchetti M, Barosi G, Balestri F, et al. Low-dose thalidomide ameliorates cytopenias and splenomegaly in myelofibrosis with myeloid metaplasia: a phase II trial. J Clin Oncol. 2004;22(3):424–431.
   PubMed Web of Science ®Google Scholar
• Tefferi A, Lasho TL, Mesa RA, et al. Lenalidomide therapy in del(5)(q31)-associated myelofibrosis: cytogenetic and JAK2V617F molecular remissions. Leukemia. 2007;21(8):1827–1828.
   PubMed Web of Science ®Google Scholar
• Maeng H, Kantarjian HM, Manshouri T, et al. Effective very long term (6+ Years) lenalidomide therapy for myelofibrosis. Blood. 2014;124(21):3203.
   Web of Science ®Google Scholar
• Quintas-Cardama A, Kantarjian HM, Manshouri T, et al. Lenalidomide plus prednisone results in durable clinical, histopathologic, and molecular responses in patients with myelofibrosis. J Clin Oncol. 2009;27(28):4760–4766.
   PubMed Web of Science ®Google Scholar
• Engelhardt M, Wasch R, Reinhardt H, et al. Pomalidomide. Recent Results Cancer Res. 2014;201:359–372.
   PubMedGoogle Scholar
• Gowin KL, Mesa RA. Profile of pomalidomide and its potential in the treatment of myelofibrosis. Ther Clin Risk Manag. 2015;11:549–556.
   PubMed Web of Science ®Google Scholar
• Quintas-Cardama A, Kantarjian HM, Thomas DA, et al. Combined therapy with lenalidomide and prednisone renders durable clinical, histopathological, and molecular responses in patients with myelofibrosis. Blood. 2008;112(11):247.
   Web of Science ®Google Scholar
• Mesa RA, Pardanani AD, Hussein K, et al. Phase1/-2 study of Pomalidomide in myelofibrosis. Am J Hematol. 2010;85(2):129–130.
   PubMed Web of Science ®Google Scholar
• Tefferi A, Verstovsek S, Barosi G, et al. Pomalidomide is active in the treatment of anemia associated with myelofibrosis. J Clin Oncol. 2009;27(27):4563–4569.
   PubMed Web of Science ®Google Scholar
• Tefferi A, Barosi G, Mesa RA, 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). Blood. 2006;108(5):1497–1503.
   PubMed Web of Science ®Google Scholar
• Begna KH, Pardanani A, Mesa R, et al. Long-term outcome of pomalidomide therapy in myelofibrosis. Am J Hematol. 2012;87(1):66–68.
   PubMed Web of Science ®Google Scholar
• Begna KH, Mesa RA, Pardanani A, et al. A phase-2 trial of low-dose pomalidomide in myelofibrosis. Leukemia. 2011;25(2):301–304.
   PubMed Web of Science ®Google Scholar
• Daver N, Shastri A, Kadia T, et al. Modest activity of pomalidomide in patients with myelofibrosis and significant anemia. Leuk Res. 2013;37(11):1440–1444.
   PubMed Web of Science ®Google Scholar
• Daver N, Shastri A, Kadia T, et al. Phase II study of pomalidomide in combination with prednisone in patients with myelofibrosis and significant anemia. Leuk Res. 2014;38(9):1126–1129.
   PubMed Web of Science ®Google Scholar
• Stegelmann F, Reiter A, Jost E, et al. Pomalidomide In MPN–associated myelofibrosis with cytopenia: results of the Mpnsg 01-09 study. Blood. 2013;122(21):2822.
   Web of Science ®Google Scholar
• Passamonti F, Barbui T, Barosi G, et al. Phase 3 study of pomalidomide in myeloproliferative neoplasm (MPN)-associated myelofibrosis with RBC-transfusion-dependence. Blood. 2013;122(21):394.
   PubMed Web of Science ®Google Scholar
• Stegelmann F, Bangerter M, Heidel FH, et al. A phase-Ib/II study of ruxolitinib plus pomalidomide in myelofibrosis. Blood. 2015;126(23):826.
   PubMed Web of Science ®Google Scholar
• Daver N, Cortes J, Newberry K, et al. Ruxolitinib in combination with lenalidomide as therapy for patients with myelofibrosis. Haematologica. 2015;100(8):1058–1063.
   PubMed Web of Science ®Google Scholar