[Results and current indications of bone marrow allograft in sickle cell disease]

Since 1988, 34 pediatric patients with severe sickle cell disease have received bone marrow transplantation (BMT) from HLA-identical siblings in France. After 1992, documentation of the favorable effect of hydroxyurea therapy on the frequency of vasoocclusive crisis (VOC) left a history of stroke (n = 16) as the main indication for BMT. Among patients treated by genoidentical BMT, 85% were cured and 9% died. All deaths were due to severe graft-versus-host disease. The rejection rate fell from 25% to 5% after addition of ATG to the conditioning regimen. BMT reversed some sickle cell disease-related abnormalities: splenic function improved and some cases of osteonecrosis showed a favorable course. Fifteen of 16 patients with a history of stroke showed no stroke recurrence after BMT, and arterial stenoses improved. BMT should be offered to sickle cell anemia patients with a history of stroke if a genoidentical donor is available. To prevent stroke-related residual impairment and transfusion-related complications, BMT should be considered early in patients with Doppler or MRI evidence of silent stroke associated with cognitive function impairment, failure to respond to hydroxyurea therapy (frequent VOCs, severe anemia and thrombocytosis, multifocal osteonecrosis), or polyerythroalloimmunization. Storage of frozen of cord blood samples from siblings should be considered.