Desai Archita P, Orman Eric S, Aridi Tarek G, Stump Timothy, Nephew Lauren, Ghabril Marwan S, Fallon Michael, Chalasani Naga, Monahan Patrick
Division of Gastroenterology and Hepatology, Indiana University School of Medicine, Indianapolis, Indiana, USA.
Department of Medicine, Indiana University School of Medicine, Indianapolis, Indiana, USA.
Hepatol Commun. 2025 May 29;9(6). doi: 10.1097/HC9.0000000000000727. eCollection 2025 Jun 1.
Patient-reported outcome measures (PROMs) are being used more often in chronic liver disease (CLD) clinical care and research. Their interpretability can be greatly enhanced by establishing the smallest meaningful score difference (MSD). We report scores of commonly used PROMs and their MSDs in patients at different stages of liver disease.
Patient-Reported Outcomes Measurement Information System (PROMIS)-29 Profile, Chronic Liver Disease Questionnaire (CLDQ), and Short Form-36 (SF-36) v1.0 scores were aggregated from 2442 adults with CLD at 4 different stages: inpatients with decompensated cirrhosis (n=1146) and outpatients with cirrhosis (n=677) or CLD (n=128) or recipients of liver transplant (LT, n=490) between June 2014 and April 2023 from 3 academic centers. MSDs were estimated using distribution and anchor-based methods.
The study sample's median age was 60.0 (IQR: 51.0-66.0); 55% were male, 17% Hispanic, 84% White, and 49% college educated. The etiology of CLD was alcohol in 36%, metabolic dysfunction-associated steatohepatitis (MASH) in 31%, and viral hepatitis B/C in 26%. Median PROMIS domain scores were generally lowest in inpatients and highest after transplant. For PROMIS, distribution-based and anchor-based MSDs ranged from 3 to 4 for individual domains and 4 to 6 for summary scores. Distribution-based MSDs were 1 for CLDQ and ranged from 7 to 11 for individual SF-36 domains, except role limitations domains, which ranged from 15 to 18, and component scores, which were 3. When compared across stages of liver disease, PROMIS MSDs were generally similar, although they tended to be 0.5-1.0 points smaller in the decompensated population compared to the stable populations.
This study provides data-driven recommendations for MSDs, enhancing the interpretability of commonly used PROMs in liver disease and facilitating the integration of PROMs in various clinical and research settings.
患者报告结局指标(PROMs)在慢性肝病(CLD)的临床护理和研究中使用得越来越频繁。通过确定最小有意义得分差异(MSD),可以大大提高其可解释性。我们报告了肝病不同阶段患者常用PROMs的得分及其MSD。
从2014年6月至2023年4月期间来自3个学术中心的2442名成年CLD患者中汇总了患者报告结局测量信息系统(PROMIS)-29简表、慢性肝病问卷(CLDQ)和简明健康状况调查量表(SF-36)v1.0的得分,这些患者处于4个不同阶段:失代偿期肝硬化住院患者(n = 1146)、肝硬化门诊患者(n = 677)或CLD门诊患者(n = 128)或肝移植(LT)受者(n = 490)。使用分布法和基于锚定的方法估计MSD。
研究样本的中位年龄为60.0(四分位间距:51.0 - 66.0);55%为男性,17%为西班牙裔,84%为白人,49%接受过大学教育。CLD的病因中,36%为酒精性,31%为代谢功能障碍相关脂肪性肝炎(MASH),26%为乙型/丙型病毒性肝炎。PROMIS领域得分中位数通常在住院患者中最低,移植后最高。对于PROMIS,基于分布法和基于锚定的MSD,单个领域为3至4分,汇总得分为4至6分。CLDQ基于分布法的MSD为1分,SF - 36单个领域的MSD为7至11分,但角色限制领域为15至18分,分量得分为3分。在比较肝病不同阶段时,PROMIS的MSD总体相似,尽管与稳定人群相比,失代偿期人群的MSD往往小0.5 - 1.0分。
本研究为MSD提供了数据驱动的建议,提高了肝病中常用PROMs的可解释性,并促进了PROMs在各种临床和研究环境中的整合。