BONE-MARROW TRANSPLANTATION IN A YOUNG-CHILD WITH SICKLE-CELL-ANEMIA

Bone marrow transplantation (BMT) is the only curative therapy availab le for hemoglobinopathies, BMT was performed on a young child with sic kle cell anemia (SCA) after approximately 9 months of transfusion ther apy following her initial stroke, The patient received a matched sibli ng donor (sickle trait) BMT, The conditioning regimen consisted of bus ulfan 4 mg/kg/day x 4, cyclophosphamide 50 mg/kg/day x 4, Graft vs, ho st disease prophylaxis was daily cyclosporine for 6 months, There were no significant complications during BMT, Engraftment occurred on day +17 and the patient was transfusion independent since day +45, Pre-BMT cerebral arteriography showed multiple stenotic cerebral vessels and a moya-moya pattern, Perfusion MRI demonstrated reduced capillary perf usion, Approximately 170 days after BMT the patient experienced episod es of transient left-sided weakness and speech problems, Neuroimaging revealed progression of large vessel pathology by angiography despite significant improvement in cortical perfusion (MR perfusion scan), Mol ecular analysis by PCR and DNA fingerprinting confirmed absence of mix ed mosaicism, Rheologic evaluation showed normal corrected bulk viscos ity, It is possible that progression of large vessel pathology and ret urn of clinical symptoms in the face of normal theologic parameters ma y be due to worsening of the already damaged cerebral vessels by the B MT conditioning regimen, Further evaluations of patients with SCA unde rgoing BMT after a stroke are needed to answer this question. (C) 1995 Wiley-Liss, Inc.