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

Inclusion Criteria

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