Primary sclerosing cholangitis (PSC) is a chronic liver disease associated with inflammation and biliary fibrosis that leads to cholangitis, cirrhosis, and impaired quality of life. Our objective was to develop and validate a PSC‐specific patient‐reported outcome (PRO) instrument. We developed a 42‐item PSC PRO instrument that contains two modules (Symptoms and Impact of Symptoms) and conducted an external validation. Reliability and validity were evaluated using clinical data and a battery of other validated instruments. Test‐retest reliability was assessed in a subgroup of patients who repeated the PSC PRO after the first administration. One hundred two PSC subjects (44 ± 13 years; 32% male, 74% employed, 39% with cirrhosis, 14% with a history of decompensated cirrhosis, 38% history of depression, and 68% with inflammatory bowel disease [IBD]) completed PSC PRO and other PRO instruments (Short Form 36 V2 [SF‐36], Chronic Liver Disease Questionnaire [CLDQ], Primary Biliary Cholangitis – 40 [PBC‐40], and five dimensions [5‐D Itch]). PSC PRO demonstrated excellent internal consistency (Cronbach alphas, 0.84‐0.94) and discriminant validity (41 of 42 items had the highest correlations with their own domains). There were good correlations between PSC PRO domains and relevant domains of SF‐36, CLDQ, and PBC‐40 (R = 0.69‐0.90; all P < 0.0001), but lower (R = 0.31‐0.60; P < 0.001) with 5‐D Itch. Construct validity showed that PSC PRO can differentiate patients according to the presence and severity of cirrhosis and history of depression (P < 0.05), but not by IBD (P > 0.05). Test‐retest reliability was assessed in 53 subjects who repeated PSC PRO within a median (interquartile range) of 37 (27‐47) days. There was excellent reliability for most domains with intraclass correlations (0.71‐0.88; all P < 0.001). Conclusion: PSC PRO is a self‐administered disease‐specific instrument developed according to U.S. Food and Drug Administration guidelines. This preliminary validation study suggests good psychometric properties. Further validation of the instrument in a larger and more diverse sample of PSC patients is needed. (Hepatology 2018;68:155‐165).

中文翻译：

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原发性硬化性胆管炎患者报告结果工具的开发和验证：PSC PRO

原发性硬化性胆管炎 (PSC) 是一种与炎症和胆管纤维化相关的慢性肝病，可导致胆管炎、肝硬化和生活质量下降。我们的目标是开发和验证 PSC 特定的患者报告结果 (PRO) 工具。我们开发了一个包含两个模块（症状和症状影响）的 42 项 PSC PRO 仪器，并进行了外部验证。使用临床数据和一系列其他经过验证的仪器来评估可靠性和有效性。在第一次给药后重复 PSC PRO 的患者亚组中评估了重测信度。102 名 PSC 受试者（44 ± 13 岁；32% 男性，74% 就业，39% 有肝硬化，14% 有失代偿期肝硬化病史，38% 有抑郁病史，68% 患有炎症性肠病 [IBD]）完成 PSC PRO 和其他 PRO 工具（Short Form 36 V2 [SF-36]、慢性肝病问卷 [CLDQ]、原发性胆汁性胆管炎 – 40 [PBC-40]，以及 5尺寸 [5-D 痒]）。PSC PRO 表现出出色的内部一致性（Cronbach alpha，0.84-0.94）和区分效度（42 个项目中的 41 个与他们自己的领域具有最高的相关性）。PSC PRO 域与 SF-36、CLDQ 和 PBC-40 的相关域之间存在良好的相关性（R = 0.69-0.90；均 P < 0.0001），但较低（R = 0.31-0.60；P < 0.001），有 5 -沟。结构效度表明，PSC PRO 可以根据肝硬化的存在和严重程度以及抑郁症史（P < 0.05）来区分患者（P < 0.05），但不能根据 IBD 来区分（P > 0.05）。在 37 (27-47) 天的中位数（四分位距）内重复 PSC PRO 的 53 名受试者中评估了重测信度。大多数具有类内相关性的领域具有出色的可靠性（0.71-0.88；所有 P < 0.001）。结论：PSC PRO 是一种根据美国食品和药物管理局指南开发的自我管理的疾病特异性仪器。该初步验证研究表明具有良好的心理测量特性。需要在更大和更多样化的 PSC 患者样本中进一步验证该仪器。（肝病学 2018 年；68：155-165）。食品和药物管理局指南。该初步验证研究表明具有良好的心理测量特性。需要在更大和更多样化的 PSC 患者样本中进一步验证该仪器。（肝病学 2018 年；68：155-165）。食品和药物管理局指南。该初步验证研究表明具有良好的心理测量特性。需要在更大和更多样化的 PSC 患者样本中进一步验证该仪器。（肝病学 2018 年；68：155-165）。