Haematopoietic stem cell transplantation for Shwachman–Diamond disease: a study from the European Group for blood and marrow transplantation
Year: 2005
Type: article
Study design: Observational Study
Abstract: 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.
Source: British Journal of Haematology
Authors: Simone Cesaro, Rosi Oneto, Chiara Messina, Brenda E. Gibson, Agnes Buzyn, Colin Steward, Eliane Gluckman, Robbert Breddius, Marc Boogaerts, Christiane Vermylen, Paul Veys, Judith Marsh, Isabel Badell, Gerard Michel, Tayfun Güngör, Dietrich Niethammer, Pierre Bordigoni, Cecilia Oswald, Claudio Favre, Jakob Passweg, Giorgio Dini, the EBMT Severe Aplastic Anaemia and Paediatric Diseases Working Party
Institutions: University of Padua, Ospedale Policlinico San Martino, Royal Hospital for Children, Hôpital Necker-Enfants Malades, Inserm, Bristol Royal Hospital for Children, Hôpital Saint-Louis, Leiden University Medical Center, Universitair Ziekenhuis Leuven, Cliniques Universitaires Saint-Luc, Great Ormond Street Hospital, St George's Hospital, Hospital de Sant Pau, Hôpital de la Timone, University Children's Hospital Zurich, University Children's Hospital Tübingen, Hôpital d'Enfants, Sydney Children's Hospital, Azienda Ospedaliera Universitaria Pisana, University Hospital of Basel, Istituto Giannina Gaslini
Language: English
Subfield: Hematology
Field: Medicine
Domain: Health Sciences
Open Access status: green
OpenAlex ID: W2147802275