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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00053001
Recruitment Status : Completed
First Posted : January 28, 2003
Last Update Posted : June 26, 2013
Information provided by:
National Cancer Institute (NCI)

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.

Condition or disease Intervention/treatment Phase
Anemia Leukemia Myelodysplastic Syndromes Myelodysplastic/Myeloproliferative Neoplasms Biological: epoetin alfa Drug: thalidomide Phase 2

Detailed Description:


  • 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..

Study Type : Interventional  (Clinical Trial)
Masking: None (Open Label)
Primary Purpose: Supportive Care
Official Title: 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
Study Start Date : June 2001
Actual Study Completion Date : October 2007

Primary Outcome Measures :
  1. Clinical response

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Ages Eligible for Study:   21 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • 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



  • Over 21

Performance status

  • Karnofsky 70-100%

Life expectancy

  • At least 6 months


  • See Disease Characteristics
  • No prior bleeding disorder


  • Bilirubin less than 2 mg/dL
  • ALT/AST less than 2 times upper limit of normal


  • Creatinine less than 1.5 mg/dL


  • 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


  • No unstable pulmonary disease
  • No recent pulmonary embolism
  • No active pulmonary infection


  • No pre-existing peripheral neuropathy greater than grade 2
  • No sustained neurologic deficit
  • No epilepsy


  • 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


Biologic therapy

  • Not specified


  • See Disease Characteristics

Endocrine therapy

  • Not specified


  • Not specified


  • Not specified


  • At least 4-6 weeks since prior therapy

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00053001

United States, Massachusetts
Fallon Clinic at Worcester Medical Center
Worcester, Massachusetts, United States, 01608
UMASS Memorial Cancer Center - University Campus
Worcester, Massachusetts, United States, 01655
Sponsors and Collaborators
Fallon Clinic
Study Chair: Laszlo Leb, MD Fallon Clinic Identifier: NCT00053001     History of Changes
Other Study ID Numbers: FALLON-PR01-09-010
CDR0000258753 ( Registry Identifier: PDQ (Physician Data Query) )
First Posted: January 28, 2003    Key Record Dates
Last Update Posted: June 26, 2013
Last Verified: October 2007

Keywords provided by National Cancer Institute (NCI):
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

Additional relevant MeSH terms:
Myelodysplastic Syndromes
Myeloproliferative Disorders
Myelodysplastic-Myeloproliferative Diseases
Pathologic Processes
Hematologic Diseases
Bone Marrow Diseases
Precancerous Conditions
Epoetin Alfa
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Leprostatic Agents
Anti-Bacterial Agents
Anti-Infective Agents
Angiogenesis Inhibitors
Angiogenesis Modulating Agents
Growth Substances
Growth Inhibitors
Antineoplastic Agents