Medical Treatment for Diamond Blackfan Anemia

INCLUSION CRITERIA: Diagnosis of DBA as characterized by a hyporegenerative anemia presenting in early childhood with reticulocytopenia, and low or absent erythroid precursors in the bone marrow. Transfusion-dependence due to steroid failure or intolerance of steroid side effects. Ineligible for or declining an allogeneic transplant. Ages 3 to 75. EXCLUSION CRITERIA: Serum creatinine greater than 2 times normal or a creatinine clearance less than 50% normal. SGPT or SGOT greater than 5 times normal. History of epilepsy (any seizures besides childhood febrile seizures). Current pregnancy or unwillingness to take oral contraceptives if menstruating. Positive diepoxybutane (DEB) test for Fanconi anemia. HIV positivity. Inability or unwillingness to sign an informed consent, either by the patient, or in the case of a minor, by the parent or guardian responsible for the patient. Underlying organ failure and/or those with a Karnofsky performance status of less than 1. Treatment with androgens, prednisone greater than 10 mg/day, growth factors, or other immunosuppressive therapies within one month of protocol entry. Ongoing treatment with Beta-adrenergic blocking drugs. Previous treatment with ATG and concurrent CSA. Previous treatment with either drug alone is acceptable if greater than one year prior to study entry.