Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel
Emanuele Angelucci,
Susanne Matthes-Martin,
Donatella Baronciani,
Françoise Bernaudin,
Sonia Bonanomi,
Maria Domenica Cappellini,
Jean-Hugues Dalle,
Paolo Di Bartolomeo,
Cristina Díaz de Heredia,
Roswitha Dickerhoff,
Claudio Giardini,
Eliane Gluckman,
Ayad Achmed Hussein,
Naynesh Kamani,
Milen Minkov,
Franco Locatelli,
Vanderson Rocha,
Petr Sedlacek,
Frans Smiers,
Isabelle Thuret,
Isaac Yaniv,
Marina Cavazzana,
Christina Peters
Affiliations
Emanuele Angelucci
Hematology, Ospedale Oncologico di Riferimento Regionale “Armando Businco”, Cagliari, Italy
Susanne Matthes-Martin
St. Anna Children’s Hospital, Department of Pediatrics, Medical University, Vienna, Austria
Donatella Baronciani
Ospedale Oncologico di Riferimento Regionale “Armando Businco”, Cagliari, Italy
Françoise Bernaudin
Centre de Référence de Drépanocytose du CHI-Creteil, France
Sonia Bonanomi
Hospital San Gerardo, Monza, Italy
Maria Domenica Cappellini
Dipartimento di Scienze Cliniche e di Comunità, IRCCS Foundation Policlinico Hospital, Milano, Italy
Jean-Hugues Dalle
Service d’Hémato-Immunologie, Hôpital Robert Debré, Paris, France
Paolo Di Bartolomeo
BMT-Center, Ospedale Civile, Pescara, Italy
Cristina Díaz de Heredia
Hopital Materno-Infantil Val d’Hebron, Barcelona, Spain
Roswitha Dickerhoff
Paediatric Unit for Haematology, Oncology and Immunology, University Düsseldorf, Germany
Claudio Giardini
Hematology and Stem Cell Transplant Center, Pesaro, Italy
Eliane Gluckman
Hôpital Saint Louis, Paris, France
Ayad Achmed Hussein
King Hussein Cancer Center, Amman, Jordan
Naynesh Kamani
Division of BMT/Immunology, Children’s National Medical Center, Washington, DC, USA
Milen Minkov
St. Anna Children’s Hospital, Department of Pediatrics, Medical University, Vienna, Austria
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.
