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Comparison of haploidentical and MSD HCT in pediatric hematologic malignancies

Filioglou D, Kovacs K, Lafleur BJ, et al. Haploidentical Versus Matched Sibling Donor HCT in Racially Diverse Pediatric and AYA Patients With Hematologic Malignancies: A Single-Center Comparison. Transplantation and Cellular Therapy. 2025; (doi: 10.1016/j.jtct.2025.09.014).

A single-site analysis concludes that using haploidentical donors for pediatric allogeneic hematopoietic cell transplantation (allo-HCT) is safe and effective when matched sibling donors (MSDs) are not possible. A MSD is unavailable for as many as 70% of patients, and this difficulty is more significant for racial and ethnic minorities. Studies have already shown haploidentical-HCT to be a reasonable option for adult transplantation recipients in these circumstances, but whether the same is true for children is uncertain. To investigate, researchers conducted a retrospective study of 29 pediatric and adolescent/young adult participants who underwent allo-HCT with MSD for hematologic malignancies and 43 others whose procedures involved a haploidentical donor. The largely Hispanic sample, aged 0-28 years, underwent transplantation between October 2013 and March 2025. At 48.7 and 39.4 months of follow-up, respectively, key outcomes with haploidentical donors were comparable to those seen with MSDs. Overall survival was measured at 70.1% vs. 74.5%; leukemia-free survival was 67.8% vs. 70.6%; relapse occurred at a rate of 23.0% vs. 26.8%; and non-relapse mortality was 9.8% vs. 13.1%. Although haplo-HCT recipients were more susceptible to cytomegalovirus infection, at 40% vs. 7%, graft-versus-host disease rates were similar and considered manageable. Collectively, the data point to haplo-HCT as an accessible transplant alternative for young minorities without an MSD.

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