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Allogeneic/Matched Related Transplantation for β-Thalassemia and Sickle Cell Anemia

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Gene and Cell Therapies for Beta-Globinopathies

Part of the book series: Advances in Experimental Medicine and Biology ((ASGCT,volume 1013))

Abstract

Allogeneic hematopoietic stem cell transplantation (HSCT) can cure single gene disorders such as thalassemia and sickle cell anemia (SCA). These non-malignant diseases have in common severe hemolytic anemia and high proliferative bone marrow, requiring frequent transfusions. The risk of rejection is high and graft-vs-host disease is not desirable. Important progress has been made in the management of these diseases, including leukocyte depletion of blood products, and chelation therapy, for both diseases, and erythrocytapheresis and hydroxycarbamide for SCA. However, morbidity and quality of life are still of concern. Results have also significantly improved for HSCT, with the reduction of rejection by using anti-thymocyte globulin (ATG), which also decreases the risk of chronic graft-vs-host disease. Current data show a more than 90% chance of cure with myeloablative conditioning in children with hemoglobinopathy and a geno-identical donor. Results are similar whether the cell source is cord blood or bone marrow. Because of the risk of conditioning-related infertility, ovarian and/or testis cryopreservation should be discussed. Non-myeloablative conditioning regimens have also been successfully developed in adults with SCA and organ dysfunction, making cure possible. These encouraging results should incite to perform HLA typing early in families with hemoglobinopathies, and to systematically propose sibling cord blood cryopreservation for those without geno-identical donor.

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Authors and Affiliations

  1. Pediatrics Department, Referral Center for Sickle Cell Disease, Centre Hospitalier Intercommunal de Creteil (CHIC), 40 avenue de Verdun, Créteil, NA, 94010, France

    Françoise Bernaudin & Corinne Pondarré

  2. Pediatric Hematology Department, Referral Thalassemia Center, La Timone, Marseille, France

    Claire Galambrun & Isabelle Thuret

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  1. Françoise Bernaudin
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  2. Corinne Pondarré
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  3. Claire Galambrun
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  4. Isabelle Thuret
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Correspondence to Françoise Bernaudin .

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  1. Cincinnati Children’s Hospital, Comprehensive Sickle Cell Program, Cincinnati, Ohio, USA

    Punam Malik

  2. National Institutes of Health and Human Services, Bethesda, Maryland, USA

    John Tisdale

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Bernaudin, F., Pondarré, C., Galambrun, C., Thuret, I. (2017). Allogeneic/Matched Related Transplantation for β-Thalassemia and Sickle Cell Anemia. In: Malik, P., Tisdale, J. (eds) Gene and Cell Therapies for Beta-Globinopathies. Advances in Experimental Medicine and Biology(), vol 1013. Springer, New York, NY. https://doi.org/10.1007/978-1-4939-7299-9_4

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