A phase-2 trial of low-dose pomalidomide in myelofibrosis

K. H. Begna, R. A. Mesa, A. Pardanani, W. J. Hogan, M. R. Litzow, R. F. McClure, A. Tefferi

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Abstract

In a previous study, we reported on the safety and efficacy of low-dose (0.5 mg) pomalidomide and prednisone and pomalidomide alone (2 mg/day), for the treatment of anemia associated with myelofibrosis (MF). The current study examined the value of low-dose pomalidomide alone. The main eligibility criterion was transfusion-dependency or hemoglobin 10 gm per 100 ml. Anemia response was assessed by International Working Group criteria. Pomalidomide (0.5 mg/day) was given to 58 patients (median age 68 years); 46 (79%) were transfusion-dependent and 42 were JAK2V617F positive. Anemia response was documented only in the presence of JAK2V617F (24 vs 0%; P0.03) but was not further affected by mutant allele burden (P0.39); 9 of the 10 anemia responders became transfusion independent. Anemia response in JAK2V617F-positive patients was predicted by the presence of pomalidomide-induced basophilia in the first month of therapy (38 vs 6%; P0.02) or absence of marked splenomegaly (38 vs 11%; P0.05). A total of 14 (58%) of 24 patients with a platelet count of 100 × 10 9 cells/l experienced a 50% increment in platelet count. There were no spleen responses. Grade 3 or 4 thrombocytopenia/neutropenia occurred in 2%/0% of patients. Low-dose pomalidomide is effective in the treatment of anemia associated with JAK2V617F-positive MF; response is predicted by early drug-induced basophilia.

Original languageEnglish (US)
Pages (from-to)301-304
Number of pages4
JournalLeukemia
Volume25
Issue number2
DOIs
StatePublished - Feb 2011
Externally publishedYes

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Keywords

  • JAK2
  • lenalidomide
  • myelofibrosis
  • thalidomide
  • treatment

ASJC Scopus subject areas

  • Hematology
  • Oncology
  • Cancer Research

Cite this

Begna, K. H., Mesa, R. A., Pardanani, A., Hogan, W. J., Litzow, M. R., McClure, R. F., & Tefferi, A. (2011). A phase-2 trial of low-dose pomalidomide in myelofibrosis. Leukemia, 25(2), 301-304. https://doi.org/10.1038/leu.2010.254