Your browser doesn't support javascript.
loading
Cord Blood Transplantation for Very Early-Onset Inflammatory Bowel Disease Caused by Interleukin-10 Receptor Deficiency.
Wang, Ping; Qian, Xiaowen; Jiang, Wenjin; Wang, Hongsheng; Wang, Yuhuan; Zhou, Ying; Zhang, Ye; Huang, Ying; Zhai, Xiaowen.
Afiliación
  • Wang P; Department of Hematology/Oncology, Children's Hospital of Fudan University, National Children's Medical Center, Shanghai, 201102, China.
  • Qian X; Department of Hematology/Oncology, Children's Hospital of Fudan University, National Children's Medical Center, Shanghai, 201102, China.
  • Jiang W; Department of Hematology/Oncology, Children's Hospital of Fudan University, National Children's Medical Center, Shanghai, 201102, China.
  • Wang H; Department of Hematology/Oncology, Children's Hospital of Fudan University, National Children's Medical Center, Shanghai, 201102, China.
  • Wang Y; Department of Gastroenterology, Children's Hospital of Fudan University, National Children's Medical Center, Shanghai, 201102, China.
  • Zhou Y; Department of Gastroenterology, Children's Hospital of Fudan University, National Children's Medical Center, Shanghai, 201102, China.
  • Zhang Y; Department of Gastroenterology, Children's Hospital of Fudan University, National Children's Medical Center, Shanghai, 201102, China.
  • Huang Y; Department of Gastroenterology, Children's Hospital of Fudan University, National Children's Medical Center, Shanghai, 201102, China.
  • Zhai X; Department of Hematology/Oncology, Children's Hospital of Fudan University, National Children's Medical Center, Shanghai, 201102, China. xwzhai@fudan.edu.cn.
J Clin Immunol ; 44(3): 67, 2024 Feb 19.
Article en En | MEDLINE | ID: mdl-38372823
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.
Asunto(s)
Palabras clave
Texto completo
Añadir a Mi BVS
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Enfermedades Inflamatorias del Intestino / Trasplante de Células Madre Hematopoyéticas / Trasplante de Células Madre de Sangre del Cordón Umbilical Límite: Child, preschool / Humans / Infant Idioma: En Revista: J Clin Immunol Año: 2024 Tipo del documento: Article País de afiliación: China Pais de publicación: Países Bajos
Texto completo
Añadir a Mi BVS
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Enfermedades Inflamatorias del Intestino / Trasplante de Células Madre Hematopoyéticas / Trasplante de Células Madre de Sangre del Cordón Umbilical Límite: Child, preschool / Humans / Infant Idioma: En Revista: J Clin Immunol Año: 2024 Tipo del documento: Article País de afiliación: China Pais de publicación: Países Bajos