Cord Blood Transplantation for Very Early-Onset Inflammatory Bowel Disease Caused by Interleukin-10 Receptor Deficiency.

Abstract

PURPOSE:

Interleukin-10 receptor (IL-10R) deficiency can result in life-threatening very early-onset inflammatory bowel disease (VEO-IBD). Umbilical cord blood transplantation (UCBT) is a curative therapy for patients with IL-10R deficiency. This study aimed to investigate the efficacy of UCBT in treating IL-10R deficiency and develop a predictive model based on pre-transplant factors.

METHODS:

Eighty patients with IL-10R deficiency who underwent UCBT between July 2015 and April 2023 were retrospectively analyzed. Cox proportional hazards regression and random survival forest were used to develop a predictive model.

RESULTS:

Median age at transplant was 13.0 months (interquartile range [IQR], 8.8-25.3 months). With a median follow-up time of 29.4 months (IQR, 3.2-57.1 months), the overall survival (OS) rate was 65.0% (95% confidence interval [CI], 55.3%-76.3%). The engraftment rate was 85% (95% CI, 77%-93%). The cumulative incidences of acute and chronic graft-versus-host disease were 48.2% (95% CI, 37.1%-59.4%) and 12.2% (95% CI, 4.7%-19.8%), respectively. VEO-IBD-associated clinical symptoms were resolved in all survivors. The multivariate analysis showed that IL-6 and stool occult blood were independent prognostic risk factors. The multivariate Cox proportional hazards regression model with stool occult blood, length- or height-for-age Z-score, medical history of sepsis, and cord blood total nucleated cells showed good discrimination ability, with a bootstrap concordance index of 0.767-0.775 in predicting OS.

CONCLUSION:

Better inflammation control before transplantation and higher cord blood total nucleated cell levels can improve patient prognosis. The nomogram can successfully predict OS in patients with IL-10R deficiency undergoing UCBT.

• Authors: Ping Wang, Xiaowen Qian, Wenjin Jiang, Hongsheng Wang, Yuhuan Wang, Ying Zhou, Ye Zhang, Ying Huang, Xiaowen Zhai
• Journal: Journal of clinical immunology (J Clin Immunol) Vol. 44 Issue 3 Pg. 67 (Feb 19 2024) ISSN: 1573-2592 [Electronic] Netherlands
• PMID: 38372823 (Publication Type: Journal Article)
• Copyright: © 2024. The Author(s), under exclusive licence to Springer Science+Business Media, LLC, part of Springer Nature.

Chemical References

• Receptors, Interleukin-10

Topics

• Humans
• Infant
• Child, Preschool
• Cord Blood Stem Cell Transplantation
• Retrospective Studies
• Hematopoietic Stem Cell Transplantation
• Receptors, Interleukin-10
• Inflammatory Bowel Diseases (diagnosis)