Results and current indications for allogeneic bone marrow grafting in sickle cell disease patients.

Since 1988, 34 pediatric patients with severe sickle cell disease have rece ived bone marrow transplantation (BMT) from HLA-identical siblings in Franc e. 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 genoidentica l BMT, 85% were cured and 9% died. All deaths were due to severe graft-vers us-host disease. The rejection rate fell from 25% to 5% after addition of A TG to the conditioning regimen. BMT reversed some sickle cell disease-relat ed 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 i mpairment and transfusion-related complications, BMT should be considered e arly in patients with Doppler or MRI evidence of silent stroke associated w ith cognitive function impairment, failure to respond to hydroxyurea therap y (frequent VOCs, severe anemia and thrombocytosis, multifocal osteonecrosi s), or polyerythroalloimmunization. Storage of frozen of cord blood samples from siblings should be considered.