Thalidomide and Epoetin Alfa in Treating Anemia in Patients With Myelodysplastic Syndrome

Study Overview
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Brief Summary
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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
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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..

Official Title
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A Phase II Study on the Effectiveness of Thalomid (Thalidomide) Combined With Procrit (Erythropoietin) for the Treatment of Anemia in Patients With Low and Intermediate Risk-1 (IPSS Score Less Than or Equal to 1.5) Myelodysplastic Syndromes

Conditions
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Anemia, Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms

Intervention / Treatment
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* Biological: epoetin alfa
* Drug: thalidomide


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 21 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Supportive Care
Masking: None (Open Label)


Arms and Interventions

| Intervention/Treatment |
| --- |
|Biological: epoetin alfa|nan|
|Drug: thalidomide|nan|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Clinical response |  |  | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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anemia, de novo myelodysplastic syndromes, secondary myelodysplastic syndromes, previously treated myelodysplastic syndromes, atypical chronic myeloid leukemia, BCR-ABL1 negative, myelodysplastic/myeloproliferative neoplasm, unclassifiable, childhood myelodysplastic syndromes