A Phase II Pilot Study to Determine the Safety and Preliminary Efficacy of Imatinib Mesylate (Gleevec) in Patients With Myelofibrosis With Myeloid Metaplasia
- Determine the safety, efficacy, and tolerability of imatinib mesylate in patients with
myelofibrosis with myeloid metaplasia.
- Determine the 3-, 6-, and 12-month major and minor erythroid response rates in patients
treated with this drug.
- Determine reduction in marrow fibrosis in patients treated with this drug.
- Determine decrease in spleen size in patients treated with this drug.
OUTLINE: This is a multicenter, open-label, nonrandomized, pilot study.
Patients receive oral imatinib mesylate once daily for 1 year in the absence of disease
progression or unacceptable toxicity. Patients who do not experience a minor erythroid
response or a 50% reduction in spleen size after 6 months of treatment are removed from the
study. Patients experiencing clinical benefit (e.g., ongoing erythroid response) after 1
year of treatment may continue treatment with imatinib mesylate as above at the discretion
of the principal investigator.
PROJECTED ACCRUAL: A total of 20 patients will be accrued for this study.
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Percentage of Participants With Major and/or Minor Erythroid Responses at 3, 6, and 12 Months of Therapy
A major response = transfusion independent or a>2.0g/dl rise in hemoglobin without transfusion maintained for at least 8 weeks. Minor response= > 1 to 2.0g/dl incremental rise in hemoglobin maintained for at lease 8 weeks with a decrease in transfusion requirements of at least 50% compared to the mean transfusion requirement during the 8 week pre-study period.
At 3,6, and 12 months of therapy
Michael Mauro, MD
OHSU Knight Cancer Institute
United States: Federal Government
|OHSU Knight Cancer Institute||Portland, Oregon 97239|