Thalidomide and Revlimid for myelofibrosis – pro

Myelofibrosis characterized by splenomegaly and bone marrow dysfunction leading ultimately to acute leukemia. Except for allogeneic and autologous stem cell transplantation, treatment is unsatisfactory.  Non-transplant treatment modalities have not improved the average 3-5 year survival associated with this disease. The usual palliative treatments include erythropoietin, androgens, hydroxyurea, and splenectomy. Myelofibrosis ( also called agnogenic myeloid metaplasia ) is a myeloproliferative disorder in which the bone marrow is initially over-active but then develops fibrosis.

There are a number of studies suggesting that thalidomide and revlimid with or without prednisone can ameliorate cytopenias of myelofibrosis with or withotu myelod metaplasia, although the latter group do not respond as well. There is supporting phase II evidence. A Phase II study evaluated Revlimid ( Lenalidomide ) in previously treated patients with myelofibrosis, a rare blood disorder that can be fatal if untreated. It was reported at the 47th American Society of Hematology ( ASH ) Meeting in Atlanta.The patients with a median age of 65 ( range, 42 to 83 ) received Lenalidomide at 10 mg/day orally ( 5 mg daily for patients with a platelet count less than 100,000 at the start of the study ). Thirty-two patients were evaluable for response or toxicity. Responses were observed in nineteen patients ( 46% ), including complete response ( CR ) achieved in three patients defined by normalization of Hgb and WBC, respectively.Partial response ( PR ) was achieved in five patients as a result of improvement in platelets and Hgb, +/- spleen, and hematologic improvement was observed in eleven patients based on improvement in platelets, spleen, WBC and BM blasts. Five of thirteen transfusion-dependent patients became transfusion independent.The median time to response was six weeks ( range, 1 to 22 ). Responses were sustained for a median of thirty-one weeks ( range, 1 to 40 weeks ).

Revlimid is not listed by DrugDex for this indication but Thaldomid is. At this point, Revlimid is not FDA approved or guideline recommended for myelofibrosis but there are several supporting studies and in 2009 several additional supporting articles have appeared, which I reference.

Ayalew Tefferi, Jorge Cortes, Srdan Verstovsek, Ruben A. Mesa, Deborah Thomas, Terra L. Lasho, William J. Hogan, Mark R. Litzow, Jacob B. Allred, Dan Jones, Catriona Byrne, Jerome B. Zeldis, Rhett P. Ketterling, Rebecca F. McClure, Francis Giles, and Hagop M. Kantarjian Lenalidomide therapy in myelofibrosis with myeloid metaplasia Blood 108: 1158-1164;

Tefferi, Ayalew Pathogenesis of Myelofibrosis With Myeloid Metaplasia J Clin Oncol 2005 23: 8520-8530

A. Quintas-Cardama, H. M. Kantarjian, T. Manshouri, D. Thomas, J. Cortes, F. Ravandi, G. Garcia-Manero, A. Ferrajoli, C. Bueso-Ramos, and S. Verstovsek Lenalidomide Plus Prednisone Results in Durable Clinical, Histopathologic, and Molecular Responses in Patients With Myelofibrosis J. Clin. Oncol., October 1, 2009; 27(28): 4760 – 4766.
R. Vaidya, S. Siragusa, J. Huang, S. M. Schwager, C. A. Hanson, K. Hussein, A. Pardanani, and A. Tefferi
Mature Survival Data for 176 Patients Younger Than 60 Years With Primary Myelofibrosis Diagnosed Between 1976 and 2005: Evidence for Survival Gains in Recent Years Mayo Clin. Proc., December 1, 2009; 84(12): 1114 – 1119.
A. Tefferi, S. Verstovsek, G. Barosi, F. Passamonti, G. J. Roboz, H. Gisslinger, R. L. Paquette, F. Cervantes, C. E. Rivera, H. J. Deeg, et al. Pomalidomide Is Active in the Treatment of Anemia Associated With Myelofibrosis
J. Clin. Oncol., September 20, 2009; 27(27): 4563 – 4569.

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