Pretransplant immune dysregulation and survival after HSCT in pediatric inborn errors of immunity.
- 1Division of Pediatrics, Department of Infectious Diseases and Immunology, School of Medicine, Pontificia Universidad Católica de Chile, Santiago, Chile.
- 2Red de Salud UC CHRISTUS , Santiago, Chile.
- 3Division of Pediatrics, Section of Hematology, Oncology and Stem Cell Transplantation, School of Medicine, Pontificia Universidad Católica de Chile, Santiago, Chile.
Abstract
Allogeneic hematopoietic stem cell transplantation (HSCT) is curative for many inborn errors of immunity (IEIs), but the prognostic role of pretransplant immune dysregulation in pediatric patients remains unclear. We retrospectively studied 29 pediatric patients with genetically confirmed IEI undergoing HSCT between 2021 and 2025. Immune dysregulation was quantified using IDDA v2.1; HCT-CI and PARI were also calculated. 2-year overall survival (OS) was 79%, and event-free survival was 59%. Higher IDDA v2.1 scores were associated with inferior OS in univariate analysis (HR: 2.23 per five-point increase, P = 0.043) and remained associated in an exploratory multivariable model (HR: 2.76, P = 0.041). Harrell's C-statistic for OS was 0.722 for IDDA v2.1, 0.661 for HCT-CI, and 0.772 for PARI. Exploratory quadrant analyses suggested that IDDA v2.1 provides complementary information, identifying patients classified as lower risk by comorbidity indices who nonetheless had adverse outcomes. These findings support IDDA v2.1 as an adjunct pretransplant risk stratification tool in pediatric IEI.
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BibTeX
@article{soffia2026pretransplant,
title = {{Pretransplant immune dysregulation and survival after HSCT in pediatric inborn errors of immunity.}},
author = {Andrés Soffia and Arturo Borzutzky and Cristián Sotomayor},
journal = {Journal of human immunity},
year = {2026},
month = oct,
doi = {10.70962/jhi.20260093},
url = {https://europepmc.org/article/MED/42895471},
note = {CC BY},
}
RIS
TY - JOUR
TI - Pretransplant immune dysregulation and survival after HSCT in pediatric inborn errors of immunity.
AU - Andrés Soffia
AU - Arturo Borzutzky
AU - Cristián Sotomayor
PY - 2026
DA - 2026/10/19
JO - Journal of human immunity
DO - 10.70962/jhi.20260093
UR - https://europepmc.org/article/MED/42895471
AB - Allogeneic hematopoietic stem cell transplantation (HSCT) is curative for many inborn errors of immunity (IEIs), but the prognostic role of pretransplant immune dysregulation in pediatric patients remains unclear. We retrospectively studied 29 pediatric patients with genetically confirmed IEI undergoing HSCT between 2021 and 2025. Immune dysregulation was quantified using IDDA v2.1; HCT-CI and PARI were also calculated. 2-year overall survival (OS) was 79%, and event-free survival was 59%. Higher IDDA v2.1 scores were associated with inferior OS in univariate analysis (HR: 2.23 per five-point increase, P = 0.043) and remained associated in an exploratory multivariable model (HR: 2.76, P = 0.041). Harrell's C-statistic for OS was 0.722 for IDDA v2.1, 0.661 for HCT-CI, and 0.772 for PARI. Exploratory quadrant analyses suggested that IDDA v2.1 provides complementary information, identifying patients classified as lower risk by comorbidity indices who nonetheless had adverse outcomes. These findings support IDDA v2.1 as an adjunct pretransplant risk stratification tool in pediatric IEI.
SN - 3065-8993
ER -