Thalidomide in Treating Anemia in Patients With Myelodysplastic Syndrome

• Status: Completed
• Trial end date: 1969-12-31
• Target enrollment: 0
• Participant gender: All

Summary

RATIONALE: Thalidomide may be an effective treatment for anemia caused by myelodysplastic syndrome.

PURPOSE: Randomized phase II trial to study the effectiveness of thalidomide in treating anemia in patients who have myelodysplastic syndrome.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Roswell Park Cancer Institute
• Collaborator: National Cancer Institute (NCI)
• Treatments: Thalidomide

Criteria

DISEASE CHARACTERISTICS:

• Diagnosis of myelodysplastic syndromes (MDS) of at least 12 weeks duration

• Refractory anemia (RA)

• RA with ringed sideroblasts

• RA with excess blasts

• Chronic myelomonocytic

• No therapy-related MDS

• No myelosclerosis or myelofibrosis occupying more than 30% of marrow space (or assessed as grade 3+ or greater)

• No transformation to acute myeloid leukemia

• No more than 20% blasts in bone marrow

• No more than 5% blasts in peripheral blood

• Patients with an erythropoietin level 100 mU/mL or less must have failed epoetin alfa treatment (i.e., at least 30,000 units of epoetin alfa weekly for at least 6 weeks)

• Transfusion-dependent (received at least 2 units of packed RBCs or whole blood within the past 8 weeks) OR

• Transfusion-independent (no packed RBC or whole blood transfusions within the past 8 weeks with 2 hemoglobin levels (at least 7 days apart) less than 11 g/dL)

• No iron deficiency (e.g., absent bone marrow iron store)

• If marrow aspirate is not evaluable, transferrin saturation must be at least 20% and ferritin at least 50 ng/mL

• No uncorrected B12 or folate deficiency

• No other contributing causes of anemia (e.g., autoimmune or hereditary hemolytic disorders or gastrointestinal blood loss)

PATIENT CHARACTERISTICS:

Age:

• 18 and over

Performance status:

• ECOG 0-2 OR

• Zubrod 0-2

Life expectancy:

• At least 6 months

Hematopoietic:

• See Disease Characteristics

• Absolute neutrophil count at least 500/mm^3

Hepatic:

• Bilirubin no greater than 2.0 mg/dL

• AST and ALT less than 2 times upper limit of normal (ULN)

• Hepatitis B surface antigen negative

• Hepatitis C negative

Renal:

• Creatinine no greater than 1.5 times ULN

Cardiovascular:

• No uncontrolled hypertension

• No clinically significant, symptomatic, unstable cardiovascular disease unrelated to MDS

Pulmonary:

• No clinically significant, symptomatic, unstable pulmonary disease unrelated to MDS

Neurologic:

• No clinically significant, symptomatic, unstable neurologic disease unrelated to MDS

• No history of epilepsy

• No sustained neurologic deficit (e.g., stroke)

• No grade 2 or greater peripheral neuropathy

Other:

• Not pregnant or nursing

• Negative pregnancy test

• Fertile patients must use at least 1 highly effective and 1 additional effective method of contraception for 4 weeks prior to, during, and for 4 weeks after study participation

• HIV negative

• No clinically significant, symptomatic, unstable endocrine, gastrointestinal, or genitourinary disease unrelated to MDS

• No other malignancy within the past 5 years except basal cell or squamous cell skin cancer or carcinoma in situ of the cervix

• No life-threatening or active infection requiring parenteral antibiotics

• No other serious concurrent illness

PRIOR CONCURRENT THERAPY:

Biologic therapy:

• See Disease Characteristics

• More than 7 days since prior hematopoietic growth factors (e.g., epoetin alfa, filgrastim (G-CSF), sargramostim (GM-CSF), or interleukin-3)

• No prior thalidomide

• No prior agents intended to inhibit vascular endothelial growth factor or tumor necrosis factor alfa (e.g., etanercept or infliximab)

• No concurrent epoetin alfa

Chemotherapy:

• No concurrent chemotherapy that may be active against MDS

Endocrine therapy:

• More than 30 days since prior androgens

• No requirement for ongoing therapy with systemic corticosteroids

Radiotherapy:

• Not specified

Surgery:

• Not specified

Other:

• More than 30 days since prior treatment for MDS except RBC transfusion or epoetin alfa

• More than 30 days since prior participation in another experimental clinical trial

• More than 30 days since prior experimental drugs

• No other concurrent investigational agents or treatments