Bone Marrow Transplantation in Treating Children With Sickle Cell Disease

You may be eligible if

• 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