Haematopoietic stem cell transplantation for severe sickle cell disease in childhood: a single centre experience of 50 patients

Summary Despite improvements in medical management, sickle cell disease ( SCD ) remains associated with severe morbidity and decreased survival. Allogeneic haematopoietic stem cell transplantation ( HSCT ) remains the only curative approach. We report the outcome of 50 consecutive children with severe SCD that received HSCT in our unit between N ovember 1988 and A pril 2013. The stem cell source was bone marrow ( n  = 39), cord blood ( n  = 3), bone marrow and cord blood ( n  = 7) and peripheral blood stem cells ( n  = 1). All patients had ≥1 severe manifestation: 37 presented with recurrent vaso‐occlusive crises/acute chest syndrome, 27 cerebral vasculopathy and 1 nephropathy. The conditioning regimen consisted of busulfan + cyclophosphamide ( B u C y) before November 1991 and B u C y + rabbit antithymocyte globulin after that date. Since 1995, all patients have been treated with hydroxycarbamide ( HC ) prior to transplantation for a median duration of 2·7 years. Median age at transplantation and median follow‐up was 8·3 and 7·7 years, respectively. Acute graft‐versus‐host disease ( GVHD ) and chronic GVHD were observed in 11 and 10 patients, respectively. An excellent outcome was achieved, with 8‐year overall survival and event‐free survival ( EFS ) rates of 94·1% and 85·6%, respectively. Since HC introduction, no graft failure occurred and EFS reached 97·4%. Prior treatment with HC may have contributed to successful engraftment.