title: Phase 2 trial of imatinib mesylate in myelofibrosis with myeloid metaplasia.
abstract: 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 x 10(9)/L. In vitro, imatinib mesylate caused variable degrees of growth suppression of myeloid and erythroid progenitors that unfortunately did not translate into clinical benefit.
authors: Mayo Clinic, Rochester, MN 55905, USA. tefferi.ayalew@mayo.edu
authors: Ayalew
authors: A
authors: Tefferi
authors: Ruben A
authors: RA
authors: Mesa
authors: Leigh A
authors: LA
authors: Gray
authors: David P
authors: DP
authors: Steensma
authors: John K
authors: JK
authors: Camoriano
authors: Michelle A
authors: MA
authors: Elliott
authors: Animesh
authors: A
authors: Pardanani
authors: Stephen M
authors: SM
authors: Ansell
authors: Timothy G
authors: TG
authors: Call
authors: Gerardo
authors: G
authors: Colon-Otero
authors: Georgene
authors: G
authors: Schroeder
authors: Curtis A
authors: CA
authors: Hanson
authors: Gordon W
authors: GW
authors: Dewald
authors: Scott H
authors: SH
authors: Kaufmann
mesh_terms: D000328:Adult; D000368:Aged; D001549:Benzamides; D003114:Colony-Forming Units Assay; D004305:Dose-Response Relationship, Drug; D004791:Enzyme Inhibitors / Q000009:adverse effects / Q000494:pharmacology / Q000627:therapeutic use*; D005260:Female; D006801:Humans; D000068877:Imatinib Mesylate; D008297:Male; D008875:Middle Aged; D023461:Myeloid Progenitor Cells / Q000187:drug effects; D010879:Piperazines / Q000009:adverse effects / Q000494:pharmacology / Q000627:therapeutic use*; D055728:Primary Myelofibrosis / Q000175:diagnosis / Q000188:drug therapy*; D011505:Protein-Tyrosine Kinases / Q000037:antagonists & inhibitors; D011743:Pyrimidines / Q000009:adverse effects / Q000494:pharmacology / Q000627:therapeutic use*
chemical_list: D001549:Benzamides; D004791:Enzyme Inhibitors; D010879:Piperazines; D011743:Pyrimidines; D000068877:Imatinib Mesylate; D011505:Protein-Tyrosine Kinases