Pomalidomide therapy for multiple myeloma and myelofibrosis: an update

References

• Jemal A, Siegel R, Xu J, Ward E. Cancer statistics, 2010. CA Cancer J Clin 2010;60:277–300.
   PubMed Web of Science ®Google Scholar
• Singhal S, Mehta J, Desikan R, et al. Antitumor activity of thalidomide in refractory multiple myeloma [see comments]. N Engl J Med 1999;341:1565–1571.
   PubMed Web of Science ®Google Scholar
• Kumar SK, Rajkumar SV, Dispenzieri A, et al. Improved survival in multiple myeloma and the impact of novel therapies. Blood 2008;111:2516–2520.
   PubMed Web of Science ®Google Scholar
• von Lilienfeld-Toal M, Hahn-Ast C, Furkert K, et al. A systematic review of phase II trials of thalidomide/dexamethasone combination therapy in patients with relapsed or refractory multiple myeloma. Eur J Haematol 2008;81:247–252.
   PubMed Web of Science ®Google Scholar
• Dimopoulos M, Spencer A, Attal M, et al. Lenalidomide plus dexamethasone for relapsed or refractory multiple myeloma. N Engl J Med 2007;357:2123–2132.
   PubMed Web of Science ®Google Scholar
• Weber DM, Chen C, Niesvizky R, et al. Lenalidomide plus dexamethasone for relapsed multiple myeloma in North America. N Engl J Med 2007;357:2133–2142.
   PubMed Web of Science ®Google Scholar
• Richardson PG, Barlogie B, Berenson J, et al. A phase 2 study of bortezomib in relapsed, refractory myeloma. N Engl J Med 2003;348:2609–2617.
   PubMed Web of Science ®Google Scholar
• Davies FE, Wu P, Jenner M, Srikanth M, Saso R, Morgan GJ. The combination of cyclophosphamide, velcade and dexamethasone induces high response rates with comparable toxicity to velcade alone and velcade plus dexamethasone. Haematologica 2007;92:1149–1150.
   PubMed Web of Science ®Google Scholar
• Kropff M, Bisping G, Schuck E, et al. Bortezomib in combination with intermediate-dose dexamethasone and continuous low-dose oral cyclophosphamide for relapsed multiple myeloma. Br J Haematol 2007;138:330–337.
   PubMed Web of Science ®Google Scholar
• Orlowski RZ, Nagler A, Sonneveld P, et al. Randomized phase III study of pegylated liposomal doxorubicin plus bortezomib compared with bortezomib alone in relapsed or refractory multiple myeloma: combination therapy improves time to progression. J Clin Oncol 2007;25:3892–3901.
   PubMed Web of Science ®Google Scholar
• Palumbo A, Ambrosini MT, Benevolo G, et al. Bortezomib, melphalan, prednisone, and thalidomide for relapsed multiple myeloma. Blood 2007;109:2767–2772.
   PubMed Web of Science ®Google Scholar
• Reece DE, Rodriguez GP, Chen C, et al. Phase I-II trial of bortezomib plus oral cyclophosphamide and prednisone in relapsed and refractory multiple myeloma. J Clin Oncol 2008;26:4777–4783.
   PubMed Web of Science ®Google Scholar
• D'Amato RJ, Loughnan MS, Flynn E, Folkman J. Thalidomide is an inhibitor of angiogenesis. Proc Natl Acad Sci USA 1994;91:4082–4085.
   PubMed Web of Science ®Google Scholar
• Keifer JA, Guttridge DC, Ashburner BP, Baldwin ASJr. Inhibition of NF-kappa B activity by thalidomide through suppression of IkappaB kinase activity. J Biol Chem 2001;276:22382–22387.
   PubMed Web of Science ®Google Scholar
• Mitsiades N, Mitsiades CS, Poulaki V, et al. Apoptotic signaling induced by immunomodulatory thalidomide analogs in human multiple myeloma cells: therapeutic implications. Blood 2002;99:4525–4530.
   PubMed Web of Science ®Google Scholar
• Escoubet-Lozach L, Lin IL, Jensen-Pergakes K, et al. Pomalidomide and lenalidomide induce p21 WAF-1 expression in both lymphoma and multiple myeloma through a LSD1-mediated epigenetic mechanism. Cancer Res 2009;69:7347–7356.
   PubMed Web of Science ®Google Scholar
• Davies FE, Raje N, Hideshima T, et al. Thalidomide and immunomodulatory derivatives augment natural killer cell cytotoxicity in multiple myeloma. Blood 2001;98:210–216.
   PubMed Web of Science ®Google Scholar
• Reddy N, Hernandez-Ilizalituri FJ, Deeb G, et al. Immunomodulatory drugs stimulate natural killer-cell function, alter cytokine production by dendritic cells, and inhibit angiogenesis enhancing the anti-tumour activity of rituximab. Br J Haematol 2008;140:36–45.
   PubMed Web of Science ®Google Scholar
• Corral LG, Haslett PA, Muller GW, et al. Differential cytokine modulation and T cell activation by two distinct classes of thalidomide analogues that are potent inhibitors of TNF-alpha. J Immunol 1999;163:380–386.
   PubMed Web of Science ®Google Scholar
• Haslett PA, Corral LG, Albert M, Kaplan G. Thalidomide costimulates primary human T lymphocytes, preferentially inducing proliferation, cytokine production, and cytotoxic responses in the CD8+ subset. J Exp Med 1998;187:1885–1892.
   PubMed Web of Science ®Google Scholar
• Anderson G, Gries M, Kurihara N, et al. Thalidomide derivative CC-4047 inhibits osteoclast formation by down-regulation of PU.1. Blood 2006;107:3098–3105.
   PubMed Web of Science ®Google Scholar
• Koh KR, Janz M, Mapara MY, et al. Immunomodulatory derivative of thalidomide (IMiD CC-4047) induces a shift in lineage commitment by suppressing erythropoiesis and promoting myelopoiesis. Blood 2005;105:3833–3840.
   PubMed Web of Science ®Google Scholar
• Lentzsch S, LeBlanc R, Podar K, et al. Immunomodulatory analogs of thalidomide inhibit growth of Hs Sultan cells and angiogenesis in vivo. Leukemia 2003;17:41–44.
   PubMed Web of Science ®Google Scholar
• D'Amato RJ, Lentzsch S, Anderson KC, Rogers MS. Mechanism of action of thalidomide and 3-aminothalidomide in multiple myeloma. Semin Oncol 2001;28:597–601.
   PubMed Web of Science ®Google Scholar
• Ferguson G, Jensen-Pergakes K, Wilkey C, et al. Immunomodulatory drug CC-4047 is a cell-type and stimulus-selective transcriptional inhibitor of cyclooxygenase 2. J Clin Immunol 2007;27:210–220.
   PubMed Web of Science ®Google Scholar
• Schey SA, Fields P, Bartlett JB, et al. Phase I study of an immunomodulatory thalidomide analog, CC-4047, in relapsed or refractory multiple myeloma. J Clin Oncol 2004;22:3269–3276.
   PubMed Web of Science ®Google Scholar
• Streetly MJ, Gyertson K, Daniel Y, Zeldis JB, Kazmi M, Schey SA. Alternate day pomalidomide retains anti-myeloma effect with reduced adverse events and evidence of in vivo immunomodulation. Br J Haematol 2008;141:41–51.
   PubMed Web of Science ®Google Scholar
• Richardson P, Siegel D, Baz R, et al. A phase 1/2 multi-center, randomized, open label dose escalation study to determine the maximum tolerated dose, safety, and efficacy of pomalidomide alone or in combination with low-dose dexamethasone in patients with relapsed and refractory multiple myeloma who have received prior treatment that includes lenalidomide and bortezomib. Blood 2009;114(Suppl. 1): Abstract 301.
   Google Scholar
• Lacy MQ, Hayman SR, Gertz MA, et al. Pomalidomide (CC4047) plus low-dose dexamethasone as therapy for relapsed multiple myeloma. J Clin Oncol 2009;27:5008–5014.
   PubMed Web of Science ®Google Scholar
• Lacy MQ, Hayman SR, Gertz MA, et al. Pomalidomide (CC4047) plus low dose dexamethasone (Pom/dex) is active and well tolerated in lenalidomide refractory multiple myeloma (MM). Leukemia 2010;24:1934–1939.
   PubMed Web of Science ®Google Scholar
• Kumar S, Blade J, Crowley J, et al. Natural history of multiple myeloma relapsing after therapy with IMiDs and bortezomib: a multicenter International Myeloma Working Group Study. Blood 2009;114(Suppl. 1): Abstract 2878.
   Google Scholar
• Richardson PG, Siegel D, Baz R, et al. A phase 1/2 multi-center, randomized, open label dose escalation study to determine the maximum tolerated dose, safety, and efficacy of pomalidomide alone or in combination with low-dose dexamethasone in patients with relapsed and refractory multiple myeloma who have received prior treatment that includes lenalidomide and bortezomib. Blood 2010;116(Suppl. 1): Abstract 864.
   Google Scholar
• Leleu X, Attal M, Moreau P, et al. Phase 2 study of 2 modalities of pomalidomide (CC4047) plus low-dose dexamethasone as therapy for relapsed multiple myeloma. IFM 2009-02. Blood 2010;116(Suppl. 1): Abstract 859.
   PubMed Web of Science ®Google Scholar
• Lacy M, Mandrekar S, Gertz MAA, et al. Pomalidomide plus low-dose dexamethasone in myeloma refractory to both bortezomib and lenalidomide: comparison of two dosing strategies in dual-refractory disease. Blood 2010;116(Suppl. 1): Abstract 863.
   Google Scholar
• Short KD, Rajkumar SV, Buadi F, et al. Increased incidence of extramedullary plasmacytomas in patients with multiple myeloma in the era of novel therapy and effect of pomalidomide on extramedullary disease. Blood 2010;116(Suppl. 1): Abstract 3047.
   Google Scholar
• Geyer H, Viggiano R, Lacy M, et al. Acute lung toxicity related to pomalidomide. Chest2011; in press
   Google Scholar
• Tefferi A, Vardiman JW. Classification and diagnosis of myeloproliferative neoplasms: the 2008 World Health Organization criteria and point-of-care diagnostic algorithms. Leukemia 2008;22:14–22.
   PubMed Web of Science ®Google Scholar
• Tefferi A. Novel mutations and their functional and clinical relevance in myeloproliferative neoplasms: JAK2, MPL, TET2, ASXL1, CBL, IDH and IKZF1. Leukemia 2010;24:1128–1138.
   PubMed Web of Science ®Google Scholar
• Tefferi A. Myelofibrosis with myeloid metaplasia. N Engl J Med 2000;342:1255–1265.
   PubMed Web of Science ®Google Scholar
• Gangat N, Caramazza D, Vaidya R, et al. DIPSS-plus: a refined Dynamic International Prognostic Scoring System (DIPSS) for primary myelofibrosis that incorporates prognostic information from karyotype, platelet count and transfusion status. J Clin Oncol 2010 Dec 13. [Epub ahead of print].
   PubMed Web of Science ®Google Scholar
• Ballen KK, Shrestha S, Sobocinski KA, et al. Outcome of transplantation for myelofibrosis. Biol Blood Marrow Transplant 2010;16:358–367.
   PubMed Web of Science ®Google Scholar
• Tefferi A. Essential thrombocythemia, polycythemia vera, and myelofibrosis: current management and the prospect of targeted therapy. Am J Hematol 2008;83:491–497.
   PubMed Web of Science ®Google Scholar
• Mishchenko E, Tefferi A. Treatment options for hydroxyurea-refractory disease complications in myeloproliferative neoplasms: JAK2 inhibitors, radiotherapy, splenectomy and transjugular intrahepatic portosystemic shunt. Eur J Haematol 2010;85:192–199.
   PubMed Web of Science ®Google Scholar
• Verstovsek S, Kantarjian H, Mesa RA, et al. Safety and efficacy of INCB018424, a JAK1 and JAK2 inhibitor, in myelofibrosis. N Engl J Med 2010;363:1117–1127.
   PubMed Web of Science ®Google Scholar
• Pardanani A, Gotlib JR, Jamieson C, et al. Safety and efficacy of TG101348, a selective JAK2 inhibitor, in myelofibrosis. J Clin Oncol 2011; Published Ahead of Print on January 10, 2011 as 10.1200/JCO.2010.32.8021.
   Google Scholar
• Tefferi A. Pathogenesis of myelofibrosis with myeloid metaplasia. J Clin Oncol 2005;23:8520–8530.
   PubMed Web of Science ®Google Scholar
• Mesa RA, Hanson CA, Rajkumar SV, Schroeder G, Tefferi A. Evaluation and clinical correlations of bone marrow angiogenesis in myelofibrosis with myeloid metaplasia. Blood 2000;96:3374–3380.
   PubMed Web of Science ®Google Scholar
• Vaidya R, Caramazza D, Finke C, Lasho T, Pardanani A, Tefferi A. Circulating IL-2R, IL-8, IL-15 and CXCL10 levels are independently prognostic in primary myelofibrosis: a comprehensive cytokine profiling study. Blood 2010;116(Suppl. 1): Abstract 3068.
   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;101:2534–2541.
   PubMed Web of Science ®Google Scholar
• Thomas DA, Giles FJ, Albitar M, et al. Thalidomide therapy for myelofibrosis with myeloid metaplasia. Cancer 2006;106:1974–1984.
   PubMed Web of Science ®Google Scholar
• Tefferi A, Cortes J, Verstovsek S, et al. Lenalidomide therapy in myelofibrosis with myeloid metaplasia. Blood 2006;108:1158–1164.
   PubMed 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;27:4760–4766.
   PubMed Web of Science ®Google Scholar
• Mesa RA, Yao X, Cripe LD, et al. Lenalidomide and prednisone for myelofibrosis: Eastern Cooperative Oncology Group (ECOG) phase 2 trial E4903. Blood 2010;116:4436–4438.
   PubMed Web of Science ®Google Scholar
• Elliott MA, Mesa RA, Li CY, et al. Thalidomide treatment in myelofibrosis with myeloid metaplasia. Br J Haematol 2002;117:288–296.
   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;108:1497–1503.
   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;22:424–431.
   PubMed Web of Science ®Google Scholar
• Thapaliya P, Tefferi A, Pardanani A, et al. Inter- national 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.
   PubMed Web of Science ®Google Scholar
• Tefferi A, Lasho TL, Mesa RA, Pardanani A, Ketterling RP, Hanson CA. Lenalidomide therapy in del(5)(q31)-associated myelofibrosis: cytogenetic and JAK2V617F molecular remissions. Leukemia 2007;21:1827–1828.
   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;27:4563–4569.
   PubMed Web of Science ®Google Scholar
• Mesa RA, Pardanani AD, Hussein K, et al. Phase1/-2 study of pomalidomide in myelofibrosis. Am J Hematol 2010;85:129–130.
   PubMed Web of Science ®Google Scholar
• Begna KH, Mesa RA, Pardanani A, et al. A phase-2 trial of low-dose pomalidomide in myelofibrosis with anemia. Leukemia 2010 Nov 5. [Epub ahead of print].
   Web of Science ®Google Scholar