TY - JOUR
T1 - Haematopoietic stem cell transplantation for Shwachman-Diamond disease
T2 - a study from the European Group for blood and marrow transplantation
AU - Cesaro, Simone
AU - Oneto, Rosi
AU - Messina, Chiara
AU - Gibson, Brenda E
AU - Buzyn, Agnes
AU - Steward, Colin
AU - Gluckman, Eliane
AU - Bredius, Robbert
AU - Breddius, Robbert
AU - Boogaerts, Marc
AU - Vermylen, Christiane
AU - Veys, Paul
AU - Marsh, Judith
AU - Badell, Isabel
AU - Michel, Gerard
AU - Güngör, Tayfun
AU - Niethammer, Dietrich
AU - Bordigoni, Pierre
AU - Oswald, Cecilia
AU - Favre, Claudio
AU - Passweg, Jakob
AU - Dini, Giorgio
AU - EBMT Severe Aplastic Anaemia and Paediatric Diseases Working Party
PY - 2005
Y1 - 2005
N2 - This report assessed the results of allogeneic stem cell transplantation (allo-SCT) in 26 patients with Shwachman-Diamond disease (SDS) and severe bone marrow abnormalities. The conditioning regimen was based on busulphan (54%), total body irradiation (23%), fludarabine (15%) or other chemotherapy combinations (8%). Standard prevention of graft versus host disease (GVHD) with cyclosporin +/- methotrexate was adopted in 54% of the patients whilst in vivo or in vitro T-cell depletion was used in 17 and four patients respectively. Neutrophil and platelet engraftment were achieved in 21 (81%) and 17 (65%) of 26 patients after a median time of 18 days and 29 days respectively. The incidence of grade III and IV acute GVHD was 24% and of chronic GVHD 29%. Nine patients died after a median time of 70 d, post-SCT. After a median follow-up of 1.1 years, the transplant-related mortality was 35.5% (95% CI 17-54) whilst the overall survival was 64.5% (95% CI 45.7-83.2). Allo-SCT was found to be successful in more than half of SDS patients with severe bone marrow dysfunction. Further improvements would be anticipated by a better definition of the optimum time in the course of disease to transplant and by the adoption of less toxic conditioning regimens.
AB - This report assessed the results of allogeneic stem cell transplantation (allo-SCT) in 26 patients with Shwachman-Diamond disease (SDS) and severe bone marrow abnormalities. The conditioning regimen was based on busulphan (54%), total body irradiation (23%), fludarabine (15%) or other chemotherapy combinations (8%). Standard prevention of graft versus host disease (GVHD) with cyclosporin +/- methotrexate was adopted in 54% of the patients whilst in vivo or in vitro T-cell depletion was used in 17 and four patients respectively. Neutrophil and platelet engraftment were achieved in 21 (81%) and 17 (65%) of 26 patients after a median time of 18 days and 29 days respectively. The incidence of grade III and IV acute GVHD was 24% and of chronic GVHD 29%. Nine patients died after a median time of 70 d, post-SCT. After a median follow-up of 1.1 years, the transplant-related mortality was 35.5% (95% CI 17-54) whilst the overall survival was 64.5% (95% CI 45.7-83.2). Allo-SCT was found to be successful in more than half of SDS patients with severe bone marrow dysfunction. Further improvements would be anticipated by a better definition of the optimum time in the course of disease to transplant and by the adoption of less toxic conditioning regimens.
U2 - 10.1111/j.1365-2141.2005.05758.x
DO - 10.1111/j.1365-2141.2005.05758.x
M3 - Article (Academic Journal)
C2 - 16197455
SN - 0007-1048
VL - 131
SP - 231
EP - 236
JO - British Journal of Haematology
JF - British Journal of Haematology
IS - 2
ER -