Phase I Study of Amifostine in Patients With Bone Marrow Failure Related to Fanconi's Anemia
Fanconi's Anemia
About this trial
This is an interventional treatment trial for Fanconi's Anemia focused on measuring Fanconi's anemia, aplastic anemia, hematologic disorders, rare disease
Eligibility 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
Sites / Locations
- Dana-Farber Cancer Institute