Phase I Study of Amifostine in Patients With Bone Marrow Failure Related to Fanconi's Anemia

Inclusion Criteria

• •PROTOCOL ENTRY CRITERIA:
• •-Disease Characteristics--
• •Diagnosis of Fanconi's anemia by conventional diepoxybutane or mitomycin sensitivity assays, or molecular testing
• •Bone marrow failure verified on at least 3 occasions in the preceding 3 months by any one of the following: hemoglobin less than 8 g/dL; absolute neutrophil count less than 1,000/mm3; platelet count less than 30,000/mm3; symptomatic bone marrow failure (e.g., exercise limitation from anemia or spontaneous bleeding from thrombocytopenia)
• •Evidence consistent with myelodysplastic syndrome allowed if less than 5% blasts on bone marrow aspiration; clonality on bone marrow cytogenetic analysis OR morphological changes on bone marrow aspirate
• •Refusal of or unsuccessful with prior conventional therapies
• •-Prior/Concurrent Therapy--
• •Biologic therapy: No prior bone marrow transplantation; no concurrent hematopoietic growth factors
• •Endocrine therapy: No concurrent androgens
• •-Patient Characteristics--
• •Hepatic: Bilirubin no greater than 2 times normal AST no greater than 3 times normal ALT no greater than 6 times normal
• •Renal: Creatinine no greater than 2 times normal
• •Other: No prior malignancy; no active bacterial, viral, or fungal infection requiring therapy other than prophylaxis; not pregnant; negative pregnancy test