{"title":"儿童原发性硬化性胆管炎的长期良好疗效","authors":"","doi":"10.1016/j.jhepr.2024.101123","DOIUrl":null,"url":null,"abstract":"<div><h3>Background & Aims</h3><p>Primary sclerosing cholangitis (PSC) is a rare progressive liver disease associated with inflammatory bowel disease (IBD). It is usually diagnosed in adults but can also present in children. Data on long-term outcomes of pediatric PSC are limited. Our aim was to study the natural history of pediatric PSC in Sweden.</p></div><div><h3>Methods</h3><p>This is a cohort study, including all children (<18 years), diagnosed with PSC between January 2000 and December 2015 at the Pediatric Liver Unit at Karolinska University Hospital, Stockholm. Patients were followed until liver transplantation, death or last date of follow-up (August 2021).</p></div><div><h3>Results</h3><p>We identified 124 children with a median age of 14 (1.9–17.8) years at PSC diagnosis. Sixty percent were boys, 93% had IBD. Median follow-up time was 13 years (5.7–21.6). Overall event-free survival in the cohort was 91% (95% CI 0.84–0.95) at 5 years and 77% (95% CI 0.68–0.84) at 10 years after diagnosis. Autoimmune hepatitis (AIH) was present in 31% (n = 39). Portal hypertension developed in 13% (n = 16), biliary complications in 24% (n = 30), cholangiocarcinoma (CCA) in 0.8% (n = 1), while 13% (n = 16) underwent liver transplantation and three patients died. Transplant-free survival was 91% after 10 years. Individuals with a high SCOPE index at diagnosis had a 2.3-fold increased risk of requiring liver transplantation (hazard ratio 2.35, 95% CI 1.18–4.66, c-statistics = 0.70). Patients with an additional diagnosis of autoimmune hepatitis had slightly higher risk of reaching transplantation during follow-up (hazard ratio 2.85, 95% CI 1.06–7.67, <em>p</em> = 0.038).</p></div><div><h3>Conclusions</h3><p>Children diagnosed with PSC have a good prognosis during the first decade after diagnosis. A high SCOPE index at diagnosis was associated with a less favorable outcome.</p></div><div><h3>Impact and implications:</h3><p>Data on long-term outcome in pediatric primary sclerosing cholangitis bridging over to adulthood is limited. There is a great need among children with primary sclerosing cholangitis and their parents for more knowledge about the natural history of this disease and what they can expect from the future. We hope that the data presented in this study may help counsel health professionals, young individuals and families affected by this disease.</p></div>","PeriodicalId":14764,"journal":{"name":"JHEP Reports","volume":null,"pages":null},"PeriodicalIF":9.5000,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2589555924001277/pdfft?md5=237b31cbbeaa0ee743f239cbe1482beb&pid=1-s2.0-S2589555924001277-main.pdf","citationCount":"0","resultStr":"{\"title\":\"Good long-term outcomes of primary sclerosing cholangitis in childhood\",\"authors\":\"\",\"doi\":\"10.1016/j.jhepr.2024.101123\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div><h3>Background & Aims</h3><p>Primary sclerosing cholangitis (PSC) is a rare progressive liver disease associated with inflammatory bowel disease (IBD). It is usually diagnosed in adults but can also present in children. Data on long-term outcomes of pediatric PSC are limited. Our aim was to study the natural history of pediatric PSC in Sweden.</p></div><div><h3>Methods</h3><p>This is a cohort study, including all children (<18 years), diagnosed with PSC between January 2000 and December 2015 at the Pediatric Liver Unit at Karolinska University Hospital, Stockholm. Patients were followed until liver transplantation, death or last date of follow-up (August 2021).</p></div><div><h3>Results</h3><p>We identified 124 children with a median age of 14 (1.9–17.8) years at PSC diagnosis. Sixty percent were boys, 93% had IBD. Median follow-up time was 13 years (5.7–21.6). Overall event-free survival in the cohort was 91% (95% CI 0.84–0.95) at 5 years and 77% (95% CI 0.68–0.84) at 10 years after diagnosis. Autoimmune hepatitis (AIH) was present in 31% (n = 39). Portal hypertension developed in 13% (n = 16), biliary complications in 24% (n = 30), cholangiocarcinoma (CCA) in 0.8% (n = 1), while 13% (n = 16) underwent liver transplantation and three patients died. Transplant-free survival was 91% after 10 years. Individuals with a high SCOPE index at diagnosis had a 2.3-fold increased risk of requiring liver transplantation (hazard ratio 2.35, 95% CI 1.18–4.66, c-statistics = 0.70). Patients with an additional diagnosis of autoimmune hepatitis had slightly higher risk of reaching transplantation during follow-up (hazard ratio 2.85, 95% CI 1.06–7.67, <em>p</em> = 0.038).</p></div><div><h3>Conclusions</h3><p>Children diagnosed with PSC have a good prognosis during the first decade after diagnosis. A high SCOPE index at diagnosis was associated with a less favorable outcome.</p></div><div><h3>Impact and implications:</h3><p>Data on long-term outcome in pediatric primary sclerosing cholangitis bridging over to adulthood is limited. There is a great need among children with primary sclerosing cholangitis and their parents for more knowledge about the natural history of this disease and what they can expect from the future. We hope that the data presented in this study may help counsel health professionals, young individuals and families affected by this disease.</p></div>\",\"PeriodicalId\":14764,\"journal\":{\"name\":\"JHEP Reports\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":9.5000,\"publicationDate\":\"2024-08-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.sciencedirect.com/science/article/pii/S2589555924001277/pdfft?md5=237b31cbbeaa0ee743f239cbe1482beb&pid=1-s2.0-S2589555924001277-main.pdf\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"JHEP Reports\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://www.sciencedirect.com/science/article/pii/S2589555924001277\",\"RegionNum\":1,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"GASTROENTEROLOGY & HEPATOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"JHEP Reports","FirstCategoryId":"3","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S2589555924001277","RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"GASTROENTEROLOGY & HEPATOLOGY","Score":null,"Total":0}
引用次数: 0
摘要
背景& 目的原发性硬化性胆管炎(PSC)是一种罕见的进行性肝病,与炎症性肠病(IBD)有关。它通常在成人中确诊,但也可在儿童中出现。有关小儿 PSC 长期预后的数据非常有限。方法这是一项队列研究,包括2000年1月至2015年12月期间在斯德哥尔摩卡罗林斯卡大学医院小儿肝病科确诊为PSC的所有儿童(18岁)。对患者进行了随访,直至肝移植、死亡或最后随访日期(2021 年 8 月)。结果我们发现 124 名儿童确诊为 PSC,中位年龄为 14(1.9-17.8)岁。其中60%为男孩,93%患有IBD。中位随访时间为 13 年(5.7-21.6)。确诊后 5 年和 10 年的总体无事件生存率分别为 91% (95% CI 0.84-0.95) 和 77% (95% CI 0.68-0.84)。31%的患者(n = 39)患有自身免疫性肝炎(AIH)。13%的患者(16人)出现门静脉高压,24%的患者(30人)出现胆道并发症,0.8%的患者(1人)出现胆管癌(CCA),13%的患者(16人)接受了肝移植,3名患者死亡。10年后无移植生存率为91%。诊断时SCOPE指数较高的患者需要进行肝移植的风险增加了2.3倍(危险比2.35,95% CI 1.18-4.66,c统计量=0.70)。在随访期间,附加诊断为自身免疫性肝炎的患者接受移植手术的风险略高(危险比 2.85,95% CI 1.06-7.67,p = 0.038)。结论:确诊为原发性硬化性胆管炎的儿童在确诊后的前十年预后良好,而确诊时SCOPE指数高则预后较差。原发性硬化性胆管炎患儿及其家长非常需要更多地了解这种疾病的自然病史以及他们对未来的预期。我们希望本研究中提供的数据能够为医疗专业人员、年轻人和受此病影响的家庭提供帮助。
Good long-term outcomes of primary sclerosing cholangitis in childhood
Background & Aims
Primary sclerosing cholangitis (PSC) is a rare progressive liver disease associated with inflammatory bowel disease (IBD). It is usually diagnosed in adults but can also present in children. Data on long-term outcomes of pediatric PSC are limited. Our aim was to study the natural history of pediatric PSC in Sweden.
Methods
This is a cohort study, including all children (<18 years), diagnosed with PSC between January 2000 and December 2015 at the Pediatric Liver Unit at Karolinska University Hospital, Stockholm. Patients were followed until liver transplantation, death or last date of follow-up (August 2021).
Results
We identified 124 children with a median age of 14 (1.9–17.8) years at PSC diagnosis. Sixty percent were boys, 93% had IBD. Median follow-up time was 13 years (5.7–21.6). Overall event-free survival in the cohort was 91% (95% CI 0.84–0.95) at 5 years and 77% (95% CI 0.68–0.84) at 10 years after diagnosis. Autoimmune hepatitis (AIH) was present in 31% (n = 39). Portal hypertension developed in 13% (n = 16), biliary complications in 24% (n = 30), cholangiocarcinoma (CCA) in 0.8% (n = 1), while 13% (n = 16) underwent liver transplantation and three patients died. Transplant-free survival was 91% after 10 years. Individuals with a high SCOPE index at diagnosis had a 2.3-fold increased risk of requiring liver transplantation (hazard ratio 2.35, 95% CI 1.18–4.66, c-statistics = 0.70). Patients with an additional diagnosis of autoimmune hepatitis had slightly higher risk of reaching transplantation during follow-up (hazard ratio 2.85, 95% CI 1.06–7.67, p = 0.038).
Conclusions
Children diagnosed with PSC have a good prognosis during the first decade after diagnosis. A high SCOPE index at diagnosis was associated with a less favorable outcome.
Impact and implications:
Data on long-term outcome in pediatric primary sclerosing cholangitis bridging over to adulthood is limited. There is a great need among children with primary sclerosing cholangitis and their parents for more knowledge about the natural history of this disease and what they can expect from the future. We hope that the data presented in this study may help counsel health professionals, young individuals and families affected by this disease.
期刊介绍:
JHEP Reports is an open access journal that is affiliated with the European Association for the Study of the Liver (EASL). It serves as a companion journal to the highly respected Journal of Hepatology.
The primary objective of JHEP Reports is to publish original papers and reviews that contribute to the advancement of knowledge in the field of liver diseases. The journal covers a wide range of topics, including basic, translational, and clinical research. It also focuses on global issues in hepatology, with particular emphasis on areas such as clinical trials, novel diagnostics, precision medicine and therapeutics, cancer research, cellular and molecular studies, artificial intelligence, microbiome research, epidemiology, and cutting-edge technologies.
In summary, JHEP Reports is dedicated to promoting scientific discoveries and innovations in liver diseases through the publication of high-quality research papers and reviews covering various aspects of hepatology.