Outcome of Second Conditioned Allogeneic Stem Cell Transplant in Children with Non-SCID Inborn Errors of Immunity - PubMed
8 hours ago
- #inborn errors of immunity
- #stem cell transplant
- #pediatrics
- Study examines outcomes of second allogeneic stem cell transplants in children with non-SCID inborn errors of immunity (IEI).
- 159 children from 37 centers who received a second transplant between 2009-2020 were analyzed.
- The median interval between first and second HSCT was 6.9 months.
- 5-year overall survival (OS) and event-free survival (EFS) rates were 78% and 69%, respectively.
- Primary graft failure had significantly lower OS (69%) and EFS (52%) compared to secondary graft failure (81% and 75%).
- Viral infections at second HSCT resulted in significantly lower EFS but not OS.
- Improved EFS was observed with MAC/RTC conditioning compared to NMA.
- Cord blood was an independent negative predictor for EFS (HR 4.9).
- 1-year cumulative incidence of all graft failure post-second HSCT was 13%.
- Day-100 cumulative incidence of grade II and grade III-IV aGvHD was 19% and 8%, respectively.
- Whole blood chimerism >90% was reported in 84.3% at last follow-up.