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Haematopoietic stem cell transplantation for severe sickle cell disease in childhood: a single centre experience of 50 patients
Author(s) -
Dedeken Laurence,
Lê Phu Q.,
Azzi Nadira,
Brachet Cécile,
Heijmans Catherine,
Huybrechts Sophie,
Devalck Christine,
Rozen Laurence,
Ngalula Malou,
Ferster Alina
Publication year - 2014
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/bjh.12737
Subject(s) - medicine , busulfan , transplantation , cyclophosphamide , fludarabine , cord blood , surgery , hematopoietic stem cell transplantation , stem cell , gastroenterology , haematopoiesis , bone marrow , chemotherapy , biology , genetics
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.