Section of Pediatric Surgery, Pediatric Liver and Gut Research Group, Pediatric Research Center, Children's Hospital, University of Helsinki and Helsinki University Hospital, Helsinki, Finland.
J Pediatr Gastroenterol Nutr. 2020 May;70(5):628-634. doi: 10.1097/MPG.0000000000002643.
Outcomes of pediatric-onset portal hypertension are poorly defined. We aimed to assess population-based long-term outcomes of pediatric-onset portal hypertension complicated by varices.
All children with esophageal varices (n = 126) were identified from 14,144 single nationwide referral center endoscopy reports during 1987 to 2013, and followed up through national health care and death registers. A questionnaire was sent to survivors (n = 94) of whom 65 (69%) responded.
Nineteen underlying disorders included biliary atresia (35%), extrahepatic portal vein obstruction (35%), autosomal recessive polycystic kidney disease (7%), and other disorders (23%). During median follow-up of 15.2 (range 0.5-43.1) years patients underwent median 9 (1-74) upper gastrointestinal endoscopies. Esophageal varices were first observed at a median age of 4.0 (0.3-18.2) years, 112 (89%) patients underwent median 6 (1-56) sclerotherapy/banding sessions, and 61 (48%) experienced median 2 (range 1-20) variceal bleeding episodes. Forty-eight surgical shunt procedures were performed to 41 (36%) patients and 38% underwent liver transplantation. Portal hypertensive biliopathy was diagnosed in 4 patients. Hepatopulmonary syndrome necessitated liver transplantation in 2 patients, hepatic encephalopathy in 2, and hepatorenal syndrome in 1. No patient died of variceal bleeding. Patient-reported perception of health on a scale of 1 to 10 was 9 (range 4-10), and 86% reported no current symptoms attributable to esophageal varices.
Pediatric-onset portal hypertension is a heterogeneous disease with significant long-term morbidity, requiring multimodal approach with considerable resources and continuation of follow-up in adulthood. Although mortality to variceal bleeding was avoided, bleeding episodes recurred also in adulthood, while patient-reported health of long-term survivors was encouraging.
儿童期发病的门静脉高压症的预后情况尚不清楚。本研究旨在评估儿童期发病的门静脉高压症伴食管静脉曲张患者的人群长期预后。
从 1987 年至 2013 年期间，14144 例全国性单一转诊中心内镜报告中确定了所有患有食管静脉曲张的儿童（n=126），并通过国家卫生保健和死亡登记进行随访。对幸存者（n=94）发送了一份调查问卷，其中 65 人（69%）做出了回应。
19 种潜在疾病包括胆道闭锁（35%）、肝外门静脉阻塞（35%）、常染色体隐性多囊肾病（7%）和其他疾病（23%）。中位随访时间为 15.2 年（范围 0.5-43.1 年），患者接受了中位 9 次（1-74 次）上消化道内镜检查。食管静脉曲张首次观察到的中位年龄为 4.0 岁（0.3-18.2 岁），112 例（89%）患者接受了中位 6 次（1-56 次）硬化/套扎治疗，61 例（48%）经历了中位 2 次（范围 1-20 次）静脉曲张出血发作。48 例门体分流术用于 41 例（36%）患者，38%的患者接受了肝移植。4 例患者诊断为门静脉高压性胆病。2 例患者因肝肺综合征需要进行肝移植，2 例患者因肝性脑病需要进行肝移植，1 例患者因肝肾综合征需要进行肝移植。没有患者死于静脉曲张出血。患者报告的健康评分范围为 1 至 10，得分为 9 分（范围 4-10 分），86%的患者报告目前没有因食管静脉曲张引起的症状。
儿童期发病的门静脉高压症是一种异质性疾病，具有显著的长期发病率，需要多模式治疗，并且需要在成年期继续进行随访。尽管避免了因静脉曲张出血导致的死亡率，但出血事件在成年期仍会再次发生，而长期幸存者的报告健康状况令人鼓舞。
