Division of Gastroenterology and Hepatology, University of California Davis School of Medicine, Sacramento, California.
PSC Partners Seeking a Cure, Greenwood Village, Colorado.
Clin Gastroenterol Hepatol. 2019 Jun;17(7):1372-1378. doi: 10.1016/j.cgh.2018.04.047. Epub 2018 Apr 26.
BACKGROUND & AIMS: Primary sclerosing cholangitis (PSC) is a fibrostenosing disease of the bile ducts associated with inflammatory bowel disease (IBD), for which the only treatment is liver transplantation. PSC progression has been defined in cohorts from referral centers or single-nation population databases. However, observations made from these cohorts have limited applicability owing to referral bias and demographic confounders. We analyzed data from the worldwide PSC Partners Patient Registry, an international online database established in 2014 to obtain information from individuals with PSC or their caretakers and compare symptoms, disease progression, and treatments of PSC in the United States and other countries.
We analyzed demographic and clinical characteristics, symptoms, and clinical outcomes of patients with PSC using the PSC Partners Patient Registry. Participants completed an online standardized questionnaire and electronic case report, providing information on age, age at symptom onset, age at PSC diagnosis, methods of diagnosis, concurrent diagnoses, family history, and medication use.
Of 873 registrants, 811 (92.9%) had completed questionnaires and 528 (65.1%) had their PSC diagnosis confirmed; we found no significant demographic or clinical differences between patients with vs without a confirmed diagnosis. In contrast to other studies, we found a higher proportion of individuals with PSC to be female (52.5%). However, the mean age at PSC diagnosis (32.4 ± 14.7 y) and the proportion of individuals with PSC and IBD (67.1%) were similar to those from prior reports. Most cases in the database were from the United States (74.9%). More than half of the participants reported having pruritus, abdominal pain, fatigue, or sleep disturbances; rates were not significantly different among participants within vs outside the United States. There was no significant difference in treatment with ursodeoxycholic acid between participants within vs outside the United States (50.0% and 57.8%; P = .07). The median time of transplant-free survival was 21 years; transplant-free survival was associated with female sex and Crohn's disease.
Our findings from an analysis of data from the PSC Partners Patient Registry confirm those from previous studies, although we found a higher proportion of individuals with PSC to be female. In addition to allowing efficient collection of patient-reported outcomes, the patient-driven registry allows for inclusion of previously under-represented cases of PSC.
原发性硬化性胆管炎（PSC）是一种与炎症性肠病（IBD）相关的胆管纤维性狭窄疾病，其唯一的治疗方法是肝移植。PSC 的进展已在转诊中心或单一国家人群数据库的队列中进行了定义。然而，由于转诊偏倚和人口统计学混杂因素，这些队列中的观察结果的适用性有限。我们分析了 2014 年建立的国际在线数据库 PSC 合作伙伴患者注册处（PSC Partners Patient Registry）的数据，该数据库旨在从 PSC 患者或其护理人员处获取信息，并比较美国和其他国家 PSC 的症状、疾病进展和治疗情况。
我们使用 PSC 合作伙伴患者注册处分析了 PSC 患者的人口统计学和临床特征、症状和临床结局。参与者完成了在线标准化问卷和电子病例报告，提供了年龄、症状发作年龄、PSC 诊断年龄、诊断方法、并发诊断、家族史和药物使用情况等信息。
在 873 名注册者中，811 名（92.9%）完成了问卷调查，528 名（65.1%）的 PSC 诊断得到了确认；我们发现有或没有确诊诊断的患者之间在人口统计学或临床方面没有显著差异。与其他研究相比，我们发现女性 PSC 患者的比例更高（52.5%）。然而，数据库中 PSC 诊断的平均年龄（32.4±14.7 岁）和 PSC 与 IBD 共存的比例（67.1%）与之前的报告相似。数据库中的大多数病例来自美国（74.9%）。超过一半的参与者报告有瘙痒、腹痛、疲劳或睡眠障碍；美国内外的参与者之间的发生率没有显著差异。美国内外参与者接受熊去氧胆酸治疗的比例没有显著差异（50.0%和 57.8%；P=0.07）。无移植生存的中位数为 21 年；无移植生存与女性和克罗恩病相关。
我们对 PSC 合作伙伴患者注册处数据的分析结果与之前的研究结果一致，尽管我们发现女性 PSC 患者的比例更高。除了能够有效地收集患者报告的结果外，患者驱动的注册处还允许纳入以前代表性不足的 PSC 病例。
