Phase I/II Study of Induction of Stable Mixed Chimerism After Bone Marrow Transplantation From HLA-Identical Donors in Children With Sickle Cell Disease

Date de début de l'étude : 1 décembre 1999

PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Diagnosis of sickle cell anemia with clinically severe disease manifestations defined by: Recurrent painful events (at least 2 painful events in past year) which cannot be explained by other causes Pain lasts at least 4 hours Requires treatment with parenteral narcotics, equianalgesic dose of oral narcotics, or parenteral nonsteroidal antiinflammatory drugs Acute chest syndrome (ACS) with at least 2 episodes within past 2 years that required hospitalization, oxygen, and RBC transfusion Any combination of painful events and ACS episodes that total 2 events within the past year Abnormal cerebral MRI, abnormal angiography (MR or conventional), and abnormal neuropsychologic testing performance No stage III or IV sickle cell lung disease Genotypically HLA identical sibling donor available --Prior/Concurrent Therapy-- No prior transfusions with greater than 5 units RBC --Patient Characteristics-- Performance status: Karnofsky 70-100% Hepatic: No active hepatitis No moderate/severe portal fibrosis Renal: Glomerular filtration rate at least 30% predicted for age Neurologic: No severe residual functional neurologic impairment Hemiplegia alone allowed Other: HIV negative Not pregnant or nursing Fertile patients must use effective contraception