Hematopoietic SCT is currently the only curative therapy for a range of benign inherited and acquired primary hematologic disorders in children, including BM failure syndromes and hemoglobinopathies. The preferred HLA-matched sibling donor is available for only about 25% of such children. However, there has been substantial progress over the last four decades in the use of alternative donors for those without a matched sibling—including HLA-matched unrelated donors, HLA-haploidentical related donors and unrelated-donor umbilical cord blood—so that it is now possible to find a donor for almost every child requiring an allograft. Below, we summarize the relative merits and limitations of the different alternative donors for benign hematologic conditions, first generally, and then in relation to specific disorders, and suggest recommendations for selecting such an alternative donor.
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This study was supported by grants from the NCI (#1R13CA177155-01), NHLBI (#5U10HL069254-13), Pediatric Cancer Research Foundation, Children’s Cancer Fund, NMDP Foundation and St Baldrick’s Foundation.
The authors declare no conflict of interest.
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Tolar, J., Sodani, P. & Symons, H. Alternative donor transplant of benign primary hematologic disorders. Bone Marrow Transplant 50, 619–627 (2015). https://doi.org/10.1038/bmt.2015.1
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