ArticleBlood advances2026
Outcome of second conditioned allogeneic stem cell transplant in children with non-SCID inborn errors of immunity.
Article in Blood advances, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
abstractGraft failure (GF) after allogeneic hematopoietic stem cell transplant (HSCT) is a life-threatening complication. The Inborn Errors Working Party conducted a retrospective study to examine the outcome of a second HSCT for children with nonsevere combined immunodeficiency (SCID) inborn errors of immunity (IEI); 159 children from 37 centers who received a second transplant between 2009 and 2020 were included in this analysis. The median interval between first and second HSCT was 6.9 months (0.7-155.2 months). The 5-year overall survival (OS) and event-free survival (EFS) rates were 78% and 69%, respectively. The second HSCT for primary GF had a significantly lower OS (69%, 55%-83% vs secondary GF, 81%, 73%-89%; P = .044) and EFS (52%, 37%-68% vs secondary GF, 75%, 67%-84%; P< .001). Improved EFS was observed in patients who received myeloablative conditioning/reduced-toxicity conditioning (74%, 66%-81%) compared with nonmyeloablative conditioning (50%, 29%-71%; P = .003), but this association was not observed in OS. Upon multivariable analysis, cord blood was the only independent negative predictor for EFS (hazard ratio, 4.9, 1.3-18.7; P = .020). The 1-year cumulative incidence (CIN) of all GF after the second HSCT was 13% (95% confidence interval, 8%-19%). The day-100 CINs of grade 2 and grade 3 to 4 acute graft-versus-host disease were 19% (13%-25%) and 8% (4%-13%), respectively. Whole-blood chimerism >90% was reported in 84.3% at last follow-up. We report, to our knowledge, the first international experience with the largest cohort of second conditioned HSCT in non-SCID IEI to date. This study provides valuable insights into the clinical outcomes following second transplant, identifying key predictors of survival.
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