Comparing Therapies for the Treatment of Severe Aplastic Anemia

Launched by NATIONAL HEART, LUNG, AND BLOOD INSTITUTE (NHLBI) · Nov 3, 1999

Keywords

ClinConnect Summary

Severe aplastic anemia (SAA) is a disorder with a poor prognosis if untreated. Current accepted therapeutic strategies include bone marrow transplantation (BMT) and immunosuppression, both offering cure or amelioration in the majority of patients. Although BMT is successful using human leukocyte antigen (HLA) matched sibling bone marrow, the 25% probability of finding an HLA identical sibling within a family renders this approach available to only a minority of patients. BMT utilizing HLA-matched, unrelated donors carries a high risk of treatment failure along with considerable toxicity. Wh...

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Eligibility criteria

• * INCLUSION CRITERIA:
• Severe aplastic anemia confirmed at NIH by:
• • 1. Bone marrow cellularity less than thirty percent (excluding lymphocytes).
• 2. At least two of the following:
• • Absolute neutrophil count less that 500/mm(3);
• • Platelet count less than 20,000/mm(3);
• • Reticulocyte count less than 60,000/mm(3).
• EXCLUSION CRITERIA:
• • Serum creatinine greater than to 2.5 mg/dl.
• • Cardiac ejection fraction less than 45% by MUGA.
• • Underlying carcinoma (except local cervical, basal cell, squamous cell or melanoma).
• • Current pregnancy or unwilling to take oral contraceptives.
• • Diagnosis of Fanconi anemia or other congenital bone marrow failure syndromes.
• • Evidence of a clonal disorder on cytogenetics.
• • HIV positivity.
• • Inability to understand the investigational nature of the study.
• • Patients who are moribund or have hepatic, renal, cardiac, metabolic or other concurrent diseases of such severity that death within 7-10 days is likely.
• • Previous treatment with ATG, or cyclophosphamide.

Trial Officials