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Abstract：
OBJECTIVE: The impact of dominant stricture (DS) on the outcomes of paediatric-onset primary sclerosing cholangitis (PSC) is unknown. This study was aimed at investigating the impact of DS on the clinical course and prognosis of patients with paediatric-onset PSC.
METHODS: Patients with paediatric-onset PSC diagnosed between January 1993 and May 2017 were identified from hospital records or our PSC registry. Data including clinical, laboratory, cholangiography, and cytology at diagnosis and during follow-up (until July 2023) were reviewed. We graphed the Kaplan-Meier failure function and fitted crude and multivariable Cox model to calculate hazard ratios (HR) and 95% confidence intervals (CI) for selected variables. In these analyses, DS was treated as a time-varying variable.
RESULTS: We identified 68 patients (42 males) with paediatric-onset PSC (median age at diagnosis 15 years). The median follow-up was 13 years and the median age at the last follow-up was 27 years. In total, 35 (51%) had concomitant autoimmune hepatitis. DS was diagnosed in 33 patients (48%): in eight at the time of PSC diagnosis (12%) and in 25 (37%) by the end of follow-up. In patients with DS, two developed cirrhosis, seven were transplanted and one patient was operated for a biliary mass with low-grade dysplasia. In patients without a DS, two developed cirrhosis, and four were transplanted; one female was excluded from survival analysis because she already had cirrhosis at the time of PSC diagnosis. Cirrhosis or biliary dysplasia or needing liver transplantation for these indications were more frequent after the development of DS (10/33, adjusted HR 4.26, 95%CI: 1.26-14.4). No cholangiocarcinomas or deaths occurred during the follow-up.
CONCLUSIONS: DS was present at diagnosis or developed during follow-up in about half of the patients with paediatric-onset PSC and was associated with impaired outcome.
METHODS: Patients with paediatric-onset PSC diagnosed between January 1993 and May 2017 were identified from hospital records or our PSC registry. Data including clinical, laboratory, cholangiography, and cytology at diagnosis and during follow-up (until July 2023) were reviewed. We graphed the Kaplan-Meier failure function and fitted crude and multivariable Cox model to calculate hazard ratios (HR) and 95% confidence intervals (CI) for selected variables. In these analyses, DS was treated as a time-varying variable.
RESULTS: We identified 68 patients (42 males) with paediatric-onset PSC (median age at diagnosis 15 years). The median follow-up was 13 years and the median age at the last follow-up was 27 years. In total, 35 (51%) had concomitant autoimmune hepatitis. DS was diagnosed in 33 patients (48%): in eight at the time of PSC diagnosis (12%) and in 25 (37%) by the end of follow-up. In patients with DS, two developed cirrhosis, seven were transplanted and one patient was operated for a biliary mass with low-grade dysplasia. In patients without a DS, two developed cirrhosis, and four were transplanted; one female was excluded from survival analysis because she already had cirrhosis at the time of PSC diagnosis. Cirrhosis or biliary dysplasia or needing liver transplantation for these indications were more frequent after the development of DS (10/33, adjusted HR 4.26, 95%CI: 1.26-14.4). No cholangiocarcinomas or deaths occurred during the follow-up.
CONCLUSIONS: DS was present at diagnosis or developed during follow-up in about half of the patients with paediatric-onset PSC and was associated with impaired outcome.
摘要：
目的：显性狭窄（DS）对小儿原发性硬化性胆管炎（PSC）结局的影响尚不清楚。这项研究旨在研究DS对儿科发病PSC患者的临床病程和预后的影响。
方法：从医院记录或我们的PSC登记中确定了1993年1月至2017年5月诊断为儿科发病的PSC患者。数据包括临床，实验室,胆道造影,对诊断时和随访期间(至2023年7月)的细胞学进行了回顾.我们绘制了Kaplan-Meier失效函数，并拟合了粗变量和多变量Cox模型，以计算选定变量的风险比（HR）和95％置信区间（CI）。在这些分析中,DS被视为时变变量。
结果：我们确定了68例（42例男性）患有儿科发病的PSC（诊断时的中位年龄15岁）。中位随访时间为13年，最后一次随访的中位年龄为27岁。总的来说,35（51％）伴有自身免疫性肝炎。在33例患者（48％）中诊断出DS：在PSC诊断时8例（12％），在随访结束时25例（37％）。在DS患者中，两个发达的肝硬化，7例接受了移植，1例患者接受了低度发育不良的胆道肿块手术。在没有DS的患者中，两个发达的肝硬化，4例进行了移植;1例女性被排除在生存分析之外,因为她在PSC诊断时已经患有肝硬化.发生DS后，肝硬化或胆道发育不良或需要肝移植的发生率更高（10/33，调整后的HR4.26，95CI：1.26-14.4）。随访期间未发生胆管癌或死亡。
结论：约半数儿科发病的PSC患者在诊断时出现或随访期间出现DS，并与预后受损相关。
方法：从医院记录或我们的PSC登记中确定了1993年1月至2017年5月诊断为儿科发病的PSC患者。数据包括临床，实验室,胆道造影,对诊断时和随访期间(至2023年7月)的细胞学进行了回顾.我们绘制了Kaplan-Meier失效函数，并拟合了粗变量和多变量Cox模型，以计算选定变量的风险比（HR）和95％置信区间（CI）。在这些分析中,DS被视为时变变量。
结果：我们确定了68例（42例男性）患有儿科发病的PSC（诊断时的中位年龄15岁）。中位随访时间为13年，最后一次随访的中位年龄为27岁。总的来说,35（51％）伴有自身免疫性肝炎。在33例患者（48％）中诊断出DS：在PSC诊断时8例（12％），在随访结束时25例（37％）。在DS患者中，两个发达的肝硬化，7例接受了移植，1例患者接受了低度发育不良的胆道肿块手术。在没有DS的患者中，两个发达的肝硬化，4例进行了移植;1例女性被排除在生存分析之外,因为她在PSC诊断时已经患有肝硬化.发生DS后，肝硬化或胆道发育不良或需要肝移植的发生率更高（10/33，调整后的HR4.26，95CI：1.26-14.4）。随访期间未发生胆管癌或死亡。
结论：约半数儿科发病的PSC患者在诊断时出现或随访期间出现DS，并与预后受损相关。
