Torres Clarivet, Badalyan Vahe, Mohan Parvathi
Intestinal Rehabilitation Program, Division of Gastroenterology, Hepatology, and Nutrition, Children's National Hospital, Washington, DC, USA.
George Washington University School of Medicine, Washington, DC, USA.
JPEN J Parenter Enteral Nutr. 2022 Jan;46(1):197-206. doi: 10.1002/jpen.2112. Epub 2021 Jun 8.
Our aim was to analyze the outcomes in children with short-bowel syndrome (SBS), parenteral nutrition dependence (PND), and intestinal failure-associated liver disease (IFALD) treated in our Intestinal Rehabilitation Program (IRP) during 2007-2018. We retrospectively reviewed charts of 135 patients with SBS-PND at the time of enrollment in IRP; of these, 89 (66%) had IFALD, defined as conjugated bilirubin (CB) of ≥2 mg/dl at enrollment and/or abnormal liver biopsy showing stage 2-4 fibrosis. Outcomes included resolution of CB, enteral autonomy, laboratory parameters (platelets, aspartate aminotransferase to platelet ratio index), growth trends, transplant rates, and mortality. Of the 89 patients, 74 had elevated CB at enrollment; the other 15 had normalized CB but had fibrosis on liver biopsy. Thirty-eight patients had liver biopsies: 36 (95%) had fibrosis, including 21/36 with bridging fibrosis/cirrhosis. The median proportion of residual small bowel was 23% (interquartile range, 13%-38%) of the expected length for age and median, daily energy requirement by PN was 100%. Two received a transplant, three died (one posttransplant), and the remaining 85 survived; 69 (81%) achieved enteral autonomy. Seventy-three (99%) of the 74 patients with hyperbilirubinemia normalized their CB with medical treatment. In a subset of eight of 89 patients with initial platelet count of <100,000/μl(median 50,500/μl) and median CB of 21 mg/dl, seven achieved CB normalization and had improved platelet count. Overall survival was 97% (censored 96.3%). We demonstrate high transplant-free survival and enteral autonomy rates among children with SBS-IFALD relying on low-dose soybean lipid emulsion.
我们的目的是分析2007年至2018年期间在我们的肠道康复项目（IRP）中接受治疗的短肠综合征（SBS）、肠外营养依赖（PND）和肠衰竭相关肝病（IFALD）患儿的治疗结果。我们回顾性分析了135例在IRP登记时患有SBS-PND的患者的病历；其中89例（66%）患有IFALD，定义为登记时结合胆红素（CB）≥2mg/dl和/或肝活检异常显示2-4期纤维化。结果包括CB恢复正常、肠内自主、实验室参数（血小板、天冬氨酸转氨酶与血小板比值指数）、生长趋势、移植率和死亡率。在这89例患者中，74例登记时CB升高；另外15例CB已恢复正常，但肝活检显示有纤维化。38例患者进行了肝活检：36例（95%）有纤维化，其中21/36例有桥接纤维化/肝硬化。残余小肠的中位比例为预期年龄长度的23%（四分位间距，13%-38%），PN的中位每日能量需求为100%。2例接受了移植，3例死亡（1例移植后死亡），其余85例存活；69例（81%）实现了肠内自主。74例高胆红素血症患者中有73例（99%）通过药物治疗使CB恢复正常。在89例初始血小板计数<100,000/μl（中位值50,500/μl）且中位CB为21mg/dl的患者亚组中，7例实现了CB正常化且血小板计数有所改善。总体生存率为97%（截尾率96.3%）。我们证明了依赖低剂量大豆脂质乳剂的SBS-IFALD患儿具有较高的无移植生存率和肠内自主率。
