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A Phase II Pilot Study to Determine the Safety and Preliminary Efficacy of Imatinib Mesylate (Gleevec) in Patients With Myelofibrosis With Myeloid Metaplasia

Phase 2
18 Years
Not Enrolling
Chronic Myeloproliferative Disorders

Thank you

Trial Information

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.

Inclusion Criteria


- Diagnosis of myelofibrosis with myeloid metaplasia (MMM), defined by all of the

- Leukoerythroblastic blood picture

- Fibrosis involving > 1/3 sectional area of bone marrow biopsy

- Splenomegaly (unless patient has undergone prior splenectomy)

- Philadelphia chromosome negative

- No myelodysplastic syndrome

- No systemic disorders associated with marrow fibrosis

- Red blood cell transfusion dependent, defined by 1 of the following:

- Patient has required ≥ 2 units of red blood cells every 4 weeks within the past
8 weeks

- Hemoglobin ≤ 8 g/dL on ≥ 3 occasions (≥ 2 weeks apart ) over the past 8 weeks

- No evidence of disease transformation to acute myelogenous leukemia, defined as > 20%
blasts in bone marrow and/or peripheral blood


Performance status

- ECOG 0-3

Life expectancy

- Not specified


- Absolute neutrophil count > 1,000/mm^3

- Platelet count > 50,000/mm^3


- Bilirubin ≤ 1.5 times upper limit of normal (ULN)

- AST or ALT ≤ 2 times ULN (unless due to extramedullary hematopoiesis in the liver)


- Creatinine ≤ 1.5 times ULN


- No New York Heart Association grade III-IV heart disease


- Not pregnant or nursing

- Negative pregnancy test

- Fertile patients must use effective barrier method contraception during and for 3
months after completion of study treatment

- No serious, uncontrolled medical condition

- No patients who are considered potentially unreliable or with a history of
noncompliance to medical regimens


Biologic therapy

- More than 2 weeks since prior interferon alfa


- No concurrent chemotherapy except hydroxyurea to control elevated blood counts

Endocrine therapy

- More than 4 weeks since prior corticosteroids, danazol, or other androgens for MMM


- More than 4 weeks since other prior treatment for MMM

- No other concurrent experimental drug therapy for MMM

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Percentage of Participants With Major and/or Minor Erythroid Responses at 3, 6, and 12 Months of Therapy

Outcome Description:

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.

Outcome Time Frame:

At 3,6, and 12 months of therapy

Safety Issue:


Principal Investigator

Michael Mauro, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

OHSU Knight Cancer Institute


United States: Federal Government

Study ID:




Start Date:

August 2005

Completion Date:

October 2011

Related Keywords:

  • Chronic Myeloproliferative Disorders
  • chronic idiopathic myelofibrosis
  • polycythemia vera
  • essential thrombocythemia
  • Primary Myelofibrosis
  • Myeloproliferative Disorders



OHSU Knight Cancer Institute Portland, Oregon  97239