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Prepublished online as a Blood First Edition Paper on April 17, 2002; DOI 10.1182/blood-2001-12-0154.
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Blood, 15 May 2002, Vol. 99, No. 10, pp. 3854-3856
BRIEF REPORT
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, and
Scott H. Kaufmann
From the Mayo Clinic, Rochester, MN; the Mayo Clinic,
Scottsdale, AZ; and the Mayo Clinic, Jacksonville, FL.
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.

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