Phase 2 trial of imatinib mesylate in myelofibrosis with myeloid metaplasia

Ayalew Tefferi, Ruben A. Mesa, Leigh A. Gray, David P. Steensma, John K. Camoriano, Michelle A. Elliott, Animesh Pardanani, Stephen M. Ansell, Timothy G. Call, Gerardo Colon-Otero, Georgene Schroeder, Curtis A. Hanson, Gordon W. Dewald, Scott H. Kaufmann

Research output: Contribution to journalArticlepeer-review

63 Scopus citations


In a phase 2 study, 23 patients with myelofibrosis with myeloid metaplasia were treated with imatinib mesylate at a constant dose of 400 mg/d. Treatment was held in 16 patients (70%), after 1 to 12 weeks, because of side effects (neutropenia, 6 patients; musculoskeletal pain, 5 patients; thrombocytosis, 4 patients; edema, 3 patients; diarrhea and hyperbilirubinemia, 1 patient). Including patients in whom retreatment at a reduced dose was possible, 11 patients (48%) were able to continue treatment beyond 3 months. None of the patients experienced a response in anemia, and only 2 had partial responses in splenomegaly. A greater than 50% increase in platelet count was documented in 11 (48%) patients, but not in those with baseline platelet counts of less than 100 × 109/L. In vitro, imatinib mesylate caused variable degrees of growth suppression of myeloid and erythroid progenitors that unfortunately did not translate into clinical benefit.

Original languageEnglish (US)
Pages (from-to)3854-3856
Number of pages3
Issue number10
StatePublished - May 15 2002

ASJC Scopus subject areas

  • Biochemistry
  • Immunology
  • Hematology
  • Cell Biology


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