Protocol for Prospective Phase II Study of Rabbit Antithymocyte Globulin (r-ATG/Thymoglobulin) and Cyclosporine (CsA) as a First Line Immunosuppressive (IS) Therapy for Severe Aplastic Anemia (sAA)
PRIMARY OBJECTIVES: To determine the response rate of r-ATG and CsA in the first line
setting. SECONDARY OBJECTIVES: To determine the level of IS as assessed by Immuknow assay in
responders and compare it to non-responders. OUTLINE:Patients receive anti-thymocyte
globulin IV over 4-24 hours daily on days 1-5. Beginning on day 6, patients receive oral
cyclosporine twice daily for 6 months followed by a taper. Treatment continues in the
absence of disease progression or unacceptable toxicity.
Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Patients Treated With Rabbit Antithymocyte Globulin (r-ATG/Thymoglobulin) and Cyclosporine (CsA) Achieving at Least a Partial Remission (PR) at 6 Months
Patients will be classified as responders if they have transfusion independence and meet two of the following three criteria: ANC greater than 500/mm3; platelet count greater than 20,000/mm3; and reticulocyte count greater than 40,000/mm3. Transfusion independence is defined as no need for transfusions for one month prior to response assessment.
At 6 months
Cleveland Clinic Taussig Cancer Institute, Case Comprehensive Cancer Center
United States: Institutional Review Board
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