Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel | oneSCDvoice
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guides & guidelines

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

key information

source: Haematologica

year: 2014

authors: Angelucci E, Matthes-Martin S, Baronciani D, Bernaudin F, Bonanomi S, Cappellini MD, Dalle JH, Di Bartolomeo P, de Heredia CD, Dickerhoff R, Giardini C, Gluckman E, Hussein AA, Kamani N, Minkov M, Locatelli F, Rocha V, Sedlacek P, Smiers F, Thuret I, Yaniv I, Cavazzana M, Peters C

summary/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.

organisation: Ospedale Oncologico di Riferimento Regionale “Armando Businco”, Cagliari; St. Anna Children’s Hospital,Austria; Centre de Référence de Drépanocytose du CHI-Creteil; Hospital San Gerardo, Monza; IRCCS Foundation Policlinico Hospital, Milano; Hôpital Robert Debré, Paris; BMT-Center,Pescara; Hopital Materno-Infantil Val d’Hebron, Barcelona; University Düsseldorf, Germany; Hematology and Stem Cell Transplant Center, Pesaro; Hôpital Saint Louis, Paris; King Hussein Cancer Center, Amman; Children’s National Medical Center, Washington, DC; IRCCS Ospedale Pediatrico Bambino Gesù, Rome; Charles University Motol Prague, Czech Republic; Schneider Children’s Medical Center of Israel; U768 INSERM, Paris; AP-HP, Hôpital Universitaire Necker – Enfants Malades, Paris; Université Paris Descartes, Sorbonne Paris Cité; EBMT Paediatric Diseases Working Party

DOI: 10.3324/haematol.2013.099747

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