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

Leukemia. 2011 Feb;25(2):301-4. doi: 10.1038/leu.2010.254. Epub 2010 Nov 5.

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%; P=0.03) but was not further affected by mutant allele burden (P=0.39); 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%; P=0.02) or absence of marked splenomegaly (38 vs 11%; P=0.05). 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.

Publication types

  • Clinical Trial, Phase II

MeSH terms

  • Aged
  • Aged, 80 and over
  • Anemia / drug therapy*
  • Anemia / pathology
  • Anemia / therapy
  • Basophils / pathology
  • Erythrocyte Transfusion
  • Female
  • Humans
  • Janus Kinase 2 / genetics
  • Male
  • Middle Aged
  • Mutation
  • Primary Myelofibrosis / drug therapy*
  • Primary Myelofibrosis / pathology
  • Primary Myelofibrosis / therapy
  • Thalidomide / administration & dosage
  • Thalidomide / analogs & derivatives*

Substances

  • Thalidomide
  • pomalidomide
  • JAK2 protein, human
  • Janus Kinase 2