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Review
. 2018 Apr;32(2):317-328.
doi: 10.1016/j.hoc.2017.11.011.

Hematopoietic Stem Cell Transplantation in Thalassemia

Luisa Strocchio  1 , Franco Locatelli  2
Affiliations
Review

Hematopoietic Stem Cell Transplantation in Thalassemia

Luisa Strocchio et al. Hematol Oncol Clin North Am. 2018 Apr.

Abstract

Although recent advances in gene therapy are expected to increase the chance of disease cure in thalassemia major, at present hematopoietic stem cell transplantation (HSCT) remains the only consolidated curative approach for this disorder. The widest experience has been obtained in the HLA-matched family donor (MFD) setting, with probabilities of overall and thalassemia-free survival exceeding 90% and 85%, respectively. As for most patients a suitable MFD is not available, alternative donors (HLA-matched unrelated donor, unrelated cord blood, HLA-haploidentical relative) have been increasingly explored, translating into the expansion of the number of patients treatable with HSCT.

Keywords: Cord blood transplantation; Haploidentical transplantation; Hematopoietic stem cell transplantation; Sibling donor transplantation; Thalassemia; Unrelated donor transplantation.

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