Anti-thymocyte Globulin and Cyclosporine as First-Line Therapy in Treating Patients With Severe Aplastic Anemia

• Status: Completed
• Trial end date: 2010-12-01
• Target enrollment: 0
• Participant gender: All

Summary

RATIONALE: Immunosuppressive therapies, such as anti-thymocyte globulin and cyclosporine, may improve bone marrow function and increase blood cell counts. PURPOSE: This phase II trial is studying how well giving anti-thymocyte globulin together with cyclosporine as first-line therapy works in treating patients with severe aplastic anemia.

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

Details

• Lead Sponsor: The Cleveland Clinic
• Treatments: Antilymphocyte Serum; Cyclosporine; Cyclosporins; Immunoglobulins; Thymoglobulin

Criteria

Inclusion Criteria:

• All patients with sAA as defined by Camitta who are candidates for IS therapy; these criteria include bone marrow cellularity < 25% or 25-50% with < 30% of hematopoietic cells; it should also have two of the following three parameters: peripheral blood neutrophils < 0.5 x 10^9/L, platelets < 20 x 10^9/L and reticulocytes < 60 x 10^9/L in anemic patients

• If cytogenetic testing has been done, it should show normal karyotype or be not informative

• Patients should be either unwilling or otherwise ineligible (age, comorbidities, lack of donor) for bone marrow transplantation as a therapeutic modality

• Not previously treated with ATG for sAA

• Patients must have ECOG performance status of 0, 1, or 2

• Vitamin B12 and folic acid deficiency must be ruled out by measurement of serum levels

• Patients must have had a bone marrow biopsy examination in the three months prior to enrolling in the study

• Must be able to provide informed consent

• Systemic and other hematologic causes of pancytopenia, based on clinical presentation, must have been ruled out

Exclusion Criteria:

• Patients with clinically evident congestive heart failure, serious cardiac arrhythmias; symptoms of coronary artery disease must be cleared by cardiology prior to therapy

• Patients who have had chemotherapy, radiotherapy, or immunotherapy or other investigational drug use within 3 weeks prior to study entry

• Pregnant women

• All females of childbearing potential must have a blood test or urine study within two weeks prior to induction registration to rule out pregnancy

• Women of childbearing potential are strongly advised to use an accepted and effective method of contraception

• Patients who have medical, psychological, or social conditions that may interfere with the patient's participation in the study or evaluation of the study results