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Cited by 399

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

Open access
Angelucci, E., Matthes-Martin, S., Baronciani, D., Bernaudin, F., Bonanomi, S., Cappellini, M. D., Dalle, J.-H., Di Bartolomeo, P., de Heredia, C. D., Dickerhoff, R., Giardini, C., Gluckman, E., Hussein, A. A., Kamani, N., Minkov, M., Locatelli, F., Rocha, V., Sedlacek, P., Smiers, F., Thuret, I., Yaniv, I., Cavazzana, M., Peters, C., on behalf of the EBMT Inborn Error and EBMT Paediatric Working Parties·2014·Haematologica

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.

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Metadata

Authors
Angelucci, E., Matthes-Martin, S., Baronciani, D., Bernaudin, F., Bonanomi, S., Cappellini, M. D., Dalle, J.-H., Di Bartolomeo, P., de Heredia, C. D., Dickerhoff, R., Giardini, C., Gluckman, E., Hussein, A. A., Kamani, N., Minkov, M., Locatelli, F., Rocha, V., Sedlacek, P., Smiers, F., Thuret, I., Yaniv, I., Cavazzana, M., Peters, C., & Parties, O. B. O. T. E. I. E. A. E. P. W.
Venue
Haematologica
Published
2014-04-30
Volume
99
Issue
5
Pages
811-820
ISSN
  • 0390-6078 (ISSN-L)
  • 1592-8721
OpenAlex
W2028135932
Cited by
399
References
4
FWCI
13.05
Type
journal article
Language
en
Open access
gold

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CoChat. (2026). Citation check: Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel. https://app.cochat.ai/papers/10.3324/haematol.2013.099747
The paper
Angelucci, E., Matthes-Martin, S., Baronciani, D., Bernaudin, F., Bonanomi, S., Cappellini, M. D., Dalle, J.-H., Di Bartolomeo, P., de Heredia, C. D., Dickerhoff, R., Giardini, C., Gluckman, E., Hussein, A. A., Kamani, N., Minkov, M., Locatelli, F., Rocha, V., Sedlacek, P., Smiers, F., . . . Parties, O. B. O. T. E. I. E. A. E. P. W. (2014). Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel. Haematologica. https://doi.org/10.3324/haematol.2013.099747

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Bibliographic data: Semantic Scholar, OpenAlex, Crossref.

Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).

Semantic Scholar’s data is described in: Kinney, R., et al. (2023). The Semantic Scholar Open Data Platform. arXiv:2301.10140.