Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel.

Abstract

Thalassemia major and sickle cell disease are the two most widely disseminated hereditary hemoglobinopathies in the world. The outlook for affected individuals has improved in recent years due to advances in medical management in the prevention and treatment of complications. However, hematopoietic stem cell transplantation is still the only available curative option. The use of hematopoietic stem cell transplantation has been increasing, and outcomes today have substantially improved compared with the past three decades. Current experience world-wide is that more than 90% of patients now survive hematopoietic stem cell transplantation and disease-free survival is around 80%. However, only a few controlled trials have been reported, and decisions on patient selection for hematopoietic stem cell transplantation and transplant management remain principally dependent on data from retrospective analyses and on the clinical experience of the transplant centers. This consensus document from the European Blood and Marrow Transplantation Inborn Error Working Party and the Paediatric Diseases Working Party aims to report new data and provide consensus-based recommendations on indications for hematopoietic stem cell transplantation and transplant management.

DOI: 10.3324/haematol.2013.099747

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@article{Angelucci2014HematopoieticSC, title={Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel.}, author={Emanuele Angelucci and Susanne Matthes-Martin and Donatella Baronciani and Françoise Bernaudin and Sonia Bonanomi and Maria Domenica Cappellini and J H Dalle and Paolo di Bartolomeo and Cristina D{\'i}az de Heredia and Roswitha Dickerhoff and Claudio Giardini and Eliane Gluckman and Ayad Achmed Hussein and Naynesh R. Kamani and Milen Minkov and Franco Locatelli and Vanderson Geraldo Rocha and Petr Sedl{\'a}cek and Frans J W Smiers and Isabelle Thuret and Isaac Yaniv and Marina Cavazzana and C Peters}, journal={Haematologica}, year={2014}, volume={99 5}, pages={811-20} }