Thalidomide therapy for myelofibrosis with myeloid metaplasia

Deborah A. Thomas, Francis J. Giles, Maher Albitar, Jorge E. Cortes, Srdan Verstovsek, Stefan Faderl, Susan M. O'Brien, Guillermo Garcia-Manero, Michael J. Keating, Sherry Pierce, Jerome Zeldis, Hagop M. Kantarjian

Research output: Contribution to journalArticlepeer-review

89 Scopus citations


BACKGROUND. Thalidomide is a putative antiangiogenesis agent with activity in several hematologic malignancies. METHODS. Forty-four patients who had myelofibrosis with myeloid metaplasia received treatment with thalidomide in a Phase II clinical trial at a dose of 200 mg daily with escalation by 200 mg weekly until the best tolerated dose (maximum, 800 mg) was reached. RESULTS. Seventeen of 41 evaluable patients (41%) who received treatment for at least 15 days had a response. A complete response (without reversal of bone marrow fibrosis) was achieved in 4 patients (10%), a partial response was achieved in 4 patients (10%), and hematologic improvements in anemia, thrombopenia, and/or splenomegaly were observed in 9 patients (21%). Improvements in anemia occurred in 7 of 35 patients (20%) with hemoglobin levels <10.0 g/dL, and improvements in thrombopenia occurred in 5 of 24 patients (21%) with platelet counts <100 × 109/L. Five of 24 patients (21%) became transfusion- independent. Major or minor regression of splenomegaly was noted in 9 of 29 evaluable patients (31%), and complete regression was noted in 5 patients. Responders had a lower baseline median vascular endothelial growth factor levels (77.9 pg/mL vs. 97.7 pg/mL; P < .01) and higher median basis fibroblast growth factor levels (60.8 pg/mL vs. 37.4 pg/mL; P < .01) compared with nonresponders. Nine patients (22%) had deterioration that was attributed to thalidomide (resolved after withdrawal) with either progressive cytopenias or excessive proliferation. Two patients developed Grade 3 neutropenia with recovery and resumed therapy with dose reductions, and both later achieved a complete response. Dose-related toxicities included fatigue (50%), constipation (48%), rash or pruritis (37%), sedation (35%), peripheral edema (29%), tremors (23%), peripheral neuropathy (22%), and orthotasis (16%). CONCLUSIONS. Thalidomide warrants further evaluation in patients with MMM, particularly in combination regimens, along with the investigation of newer analogs.

Original languageEnglish (US)
Pages (from-to)1974-1984
Number of pages11
Issue number9
StatePublished - May 1 2006
Externally publishedYes


  • Agnogenic myaloid metaplasia
  • Basic fibroblast growth factor
  • Myelofibrosis
  • Thalidomide
  • Tumor necrosis factor α
  • Vascular endothelial growth factor

ASJC Scopus subject areas

  • Oncology
  • Cancer Research


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