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

Abstract

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 November 1988 and April 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 (BuCy) before November 1991 and BuCy + 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.

Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation

“…Five-year survival is excellent, considering that these transplants were performed worldwide and reported to observational registries and that the majority of patients were not enrolled on clinical trials. [22][23][24][25][26][27] Further, we were able The adjusted Cox regression analysis was stratified by registry (EBMT and CIBMTR); age was considered as a continuous variable, and when considering the graft source, PB and CB were compared, separately, with BM (baseline) for the EFS.…”

Neuroimaging findings in sickle cell disease

“…PRES has also been reported following allogenous bone marrow transplantation (ABMT) to treat severe SCD, whereby myeloablative neurotoxicity has been postulated as a mechanism. 28,29 A further possible complication of ABMT is graft-versus-host disease, which may present as cerebrovascular disease, demyelination or immune-mediated encephalitis. Cerebral fat embolism secondary to breakdown of infarcted bone is a rare but feared complication of SCD.…”

Alternative donor hematopoietic stem cell transplantation for sickle cell disease

“…3 Myeloablative HLA-matched sibling donor transplantation for pediatric SCD has been successful, with survival rates now approaching more than 95%. [4][5][6][7] Most patients who could be cured of SCD by stem cell transplantation do not have a HLA-matched sibling donor or an 8/8 matched unrelated donor. less successful than HLA-matched sibling transplantation as a result of graft failure or GVHD, resulting in failure to cure the SCD and/or poor survival.…”