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西罗莫司治疗小儿肝移植中慢性和激素抵抗的移植物排斥反应相关纤维化。

Sirolimus to treat chronic and steroid-resistant allograft rejection-related fibrosis in pediatric liver transplantation.

机构信息

Pediatric Hepatology and Liver Transplant Department, ERN Rare Liver - ERN TrasplantChild, Hospital Universitari Vall d'Hebron, Barcelona, Spain.

Pediatric Intensive Care Unit, Hospital Universitari Vall d'Hebron, Barcelona, Spain.

出版信息

J Pediatr Gastroenterol Nutr. 2024 Nov;79(5):962-968. doi: 10.1002/jpn3.12286. Epub 2024 Jul 8.

Abstract

This study aimed to report our experience with the use of Sirolimus (SRL) in pediatric liver transplant patients with chronic rejection or steroid-resistant rejection with hepatic fibrosis, focusing on their histological evolution. All pediatric liver transplant recipients who received off-label treatment with SRL for chronic ductopenic rejection or cortico-resistant rejection between July 2003 and July 2022 were included in the study. All nine patients included in the study showed improvement in liver enzymes and cholestasis parameters as soon as 1-month after post-SRL introduction. A decrease in fibrosis stage was observed in 7/9 (77.7%) patients at 36 months. All but one patient experienced an improvement in the Rejection Activity Index and ductopenia at 12 months. A single patient had to discontinue SRL treatment owing to nephrotic proteinuria. In conclusion, SRL may be a safe and effective treatment for chronic and steroid-resistant rejection and may improve allograft rejection-related fibrosis and ductal damage.

摘要

本研究旨在报告我们在患有慢性排斥反应或伴有肝纤维化的类固醇耐药排斥反应的儿科肝移植患者中使用西罗莫司(SRL)的经验,重点关注其组织学演变。所有在 2003 年 7 月至 2022 年 7 月期间因慢性胆管发育不良排斥反应或皮质类固醇耐药排斥反应而接受 SRL 标签外治疗的儿科肝移植受者均纳入本研究。研究中纳入的 9 名患者在 SRL 治疗后 1 个月内均显示出肝功能和胆汁淤积参数的改善。7/9(77.7%)名患者在 36 个月时纤维化分期降低。除 1 名患者外,所有患者在 12 个月时排斥活动指数和胆管减少均有改善。仅 1 例患者因肾病性蛋白尿而停止 SRL 治疗。总之,SRL 可能是治疗慢性和类固醇耐药排斥反应的安全有效方法,可改善移植物排斥反应相关纤维化和胆管损伤。

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