Gwaltney Consulting, 1 Bucks Trail, Westerly, RI, 02891, USA.
Patient-Centered Solutions, IQVIA, New York, NY, USA.
Adv Ther. 2022 Nov;39(11):5126-5143. doi: 10.1007/s12325-022-02261-8. Epub 2022 Sep 6.
Understanding how patients experience their disease is a vital step in optimal disease management, and patient- and observer-reported outcome (PRO and ObsRO, respectively) measures can add important details to clinical information that is obtained as novel treatments are developed. Instruments that measure meaningful symptoms and impacts from the perspective of pediatric patients with cholestatic liver disease or their caregivers are needed. This study aimed to identify salient concepts in pediatric cholestatic liver disease, develop novel PRO and ObsRO instruments, and establish the instruments' content validity.
Relevant signs, symptoms, and impacts of cholestatic liver disease were identified through a literature review, interviews with expert clinicians, and concept elicitation interviews with children and caregivers of children who had progressive familial intrahepatic cholestasis (PFIC), Alagille syndrome, biliary atresia, or primary sclerosing cholangitis. Additional cognitive debriefing interviews with patients and caregivers were performed to ensure that participants could understand the instructions, questions, and response scales of the PRO and ObsRO instruments, with modifications made as necessary to improve comprehension and/or usability.
A total of 36 interviews with patients and caregivers were conducted. Pruritus and sleep disturbance (e.g., difficulty falling or staying asleep due to itch) were identified as the most problematic symptom and significant impact, respectively, of the pediatric cholestatic liver diseases assessed. The ObsRO and PRO instruments, called PRUCISION, focus on these key disease features in the morning and evening. Several modifications were made to the draft instruments following cognitive interviews. The final PRUCISION PRO and ObsRO measures are designed as an electronic diary to be completed twice daily. The response scales include pictorial, verbal, and numeric scales.
Novel PRO and ObsRO PRUCISION instruments were created that evaluate the patient experience of cholestatic pruritus in children with PFIC and other cholestatic liver diseases. The content validity of the PRUCISION instruments is established.
了解患者如何体验自身疾病是优化疾病管理的重要步骤，患者报告结局（PRO）和观察报告结局（ObsRO）测量可以为临床信息提供重要补充，而这些临床信息是在开发新型治疗方法时获得的。因此，需要开发一种能从患有胆汁淤积性肝病的儿科患者及其照护者的角度来测量有意义的症状和影响的工具。本研究旨在确定儿科胆汁淤积性肝病的重要概念，开发新的 PRO 和 ObsRO 工具，并建立这些工具的内容效度。
通过文献回顾、与专家临床医生的访谈以及对患有进行性家族性肝内胆汁淤积症（PFIC）、Alagille 综合征、胆道闭锁或原发性硬化性胆管炎的儿童及其照护者的概念启发式访谈，确定了胆汁淤积性肝病的相关体征、症状和影响。还对患者和照护者进行了额外的认知深度访谈，以确保患者能够理解 PRO 和 ObsRO 工具的说明、问题和反应量表，并在必要时进行修改以提高理解能力和/或可用性。
共进行了 36 次患者和照护者的访谈。瘙痒和睡眠障碍（例如，因瘙痒而难以入睡或保持睡眠）被确定为评估的儿科胆汁淤积性肝病中最严重的症状和最重要的影响。ObsRO 和 PRO 工具称为 PRUCISION，重点关注这些关键疾病特征在早上和晚上的情况。在认知访谈后，对草稿工具进行了一些修改。最终的 PRUCISION PRO 和 ObsRO 测量工具设计为电子日记，每天完成两次。反应量表包括图像、文字和数字量表。
创建了新的 PRO 和 ObsRO PRUCISION 工具，用于评估 PFIC 和其他胆汁淤积性肝病儿童的胆汁淤积性瘙痒的患者体验。PRUCISION 工具的内容效度已建立。
