Article révisé par les pairs
Résumé : Forty five patients with homozygous sickle cell anaemia (SCA) underwent bone marrow transplantation (BMT) between april 1986 and april 1996 in Belgium. Their age ranged from 11 months to 23 years (median: 7 years) and the follow up from 3 months to 10 years (median: 4 yrs). Donors were HLA identical relatives (sibling marrow: 42, sibling cord blood: 2, father marrow: 1). All patients had chronic haemolysis, 44 suffered from vaso-occlusive crises, 17 from acute chest syndrome, 5 from strokes or seizures. Two had osteonecrosis from shoulders or hips and 7 were on a chronic transfusion program. The conditioning regimen consisted of Busulfan and Cyclophosphamide. The survival rate is 95%, the disease free survival (DFS) 86%. A bone marrow rejection was observed in 5 patients. A second BMT was successfully performed in one. Two patients died: one 3 months after BMT of gr III aGVHD, CMV infection and aspergillosis, the other 6 years after BMT (sudden death) despite a complete chimerism and a recovery of the spleen function. Short term secondary events include a high incidence of neurological complications (16/45). Acute GVHD was observed in 19 patients (gr I and II: 18; III: 1) and chronic GVHD in 7 (localised: 4, extensive: 3). Growth was normal or improved after BMT in 27/29 patients. Two patients with cGVHD treated with steroids had a transient decrease in growth velocity. The thyroid function was normal in all the patients tested (22/22). Among 7 evaluable girls, primary and secondary amenorrhoea were found in 6. The pubertal development and basal levels of gonadal hormones were normal in 5/5 evaluable boys. The general status of these patients has been dramatically improved by BMT with resolution of haemolysis and vaso-occlusive episodes in all of them and a partial recovery of spleen function in 10/12 studied patients.