Anemia, Leukemia, Myelodysplastic/Myeloproliferative Neoplasms, Myelodysplastic Syndromes
Conditions
Keywords
de novo myelodysplastic syndromes, secondary myelodysplastic syndromes, previously treated myelodysplastic syndromes, anemia, atypical chronic myeloid leukemia, BCR-ABL1 negative, myelodysplastic/myeloproliferative neoplasm, unclassifiable, childhood myelodysplastic syndromes
Brief summary
RATIONALE: Thalidomide may stop or slow the growth of cancer cells. Epoetin alfa may stimulate red blood cell production. Combining thalidomide with epoetin alfa may improve anemia, decrease the need for blood transfusions, and improve the quality of life in patients with myelodysplastic syndrome. PURPOSE: Phase II trial to study the effectiveness of combining thalidomide with epoetin alfa in treating anemia in patients who have myelodysplastic syndrome.
Detailed description
OBJECTIVES: * Determine whether the combination of epoetin alfa and thalidomide improves the anemia and/or decreases the need for red cell transfusion in patients with low- or intermediate-risk myelodysplastic syndromes. * Determine whether this regimen improves the bone marrow morphology and cytogenetics, alters the natural history of the disease, and reduces the frequency of leukemic transformation in these patients. * Evaluate whether this regimen improves pathophysiologic parameters (e.g., apoptosis, tumor necrosis factor-alpha concentration, microvessel density, vascular endothelial growth factor, and cytotoxic T lymphocytes) in the bone marrow of these patients. * Determine the safety of this regimen in these patients. OUTLINE: Patients receive epoetin alfa subcutaneously (SC) once weekly for 8 weeks. After 8 weeks, patients unresponsive to epoetin alfa alone receive oral thalidomide once daily in addition to epoetin alfa SC once weekly for a maximum of 24 weeks in the absence of disease progression or unacceptable toxicity. PROJECTED ACCRUAL: A total of 30-40 patients will be accrued for this study within 2 years..
Interventions
BIOLOGICALepoetin alfa
DRUGthalidomide
Sponsors
Fallon Clinic
Study design
Primary purpose
SUPPORTIVE_CARE
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
21 Years to No maximum
Healthy volunteers
No
Inclusion criteria
DISEASE CHARACTERISTICS: * Diagnosis of myelodysplastic syndromes * Newly diagnosed OR * Prior treatment was unsuccessful, including treatment with chemotherapy * International prognostic scoring system score no greater than 1.5 * Hemoglobin no greater than 10 g/dL (untransfused) AND/OR * Received at least 3 units of packed red blood cells for symptomatic anemia within the past 6 weeks PATIENT CHARACTERISTICS: Age * Over 21 Performance status * Karnofsky 70-100% Life expectancy * At least 6 months Hematopoietic * See Disease Characteristics * No prior bleeding disorder Hepatic * Bilirubin less than 2 mg/dL * ALT/AST less than 2 times upper limit of normal Renal * Creatinine less than 1.5 mg/dL Cardiovascular * No prior clinically significant heart disease * No uncontrolled hypertension * No recent thromboembolic disease (e.g., deep vein thrombosis) * Prior thromboembolic events allowed provided event occurred at least 6 weeks prior to study and patient is on anticoagulants and is clinically stable Pulmonary * No unstable pulmonary disease * No recent pulmonary embolism * No active pulmonary infection Neurologic * No pre-existing peripheral neuropathy greater than grade 2 * No sustained neurologic deficit * No epilepsy Other * Not pregnant or nursing * Negative pregnancy test * Fertile patients must use 2 effective methods (including 1 highly effective method) of contraception for at least 4 weeks before, during, and for at least 4 weeks after study completion * No active infection * No concurrent illness that would obscure toxicity or dangerously alter drug metabolism * No other serious concurrent medical illness * No uncontrolled diabetes mellitus * No other malignant disease (except non-melanoma skin cancer or carcinoma in situ of the cervix) unless in complete remission and off therapy for that disease for more than 1 year * No known hypersensitivity to mammalian cell-derived products or human albumin PRIOR CONCURRENT THERAPY: Biologic therapy * Not specified Chemotherapy * See Disease Characteristics Endocrine therapy * Not specified Radiotherapy * Not specified Surgery * Not specified Other * At least 4-6 weeks since prior therapy
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Clinical response | — |
Countries
United States
Outcome results
None listed