Center For Liver Disease, Department of Medicine, Inova Fairfax Hospital, Falls Church, Virginia; Betty and Guy Beatty Center for Integrated Research, Inova Health System, Falls Church, Virginia.
Center for Outcomes Research in Liver Diseases, Washington, District of Columbia.
Clin Gastroenterol Hepatol. 2019 Sep;17(10):2093-2100.e3. doi: 10.1016/j.cgh.2019.01.001. Epub 2019 Jan 11.
BACKGROUND & AIMS: The chronic liver disease questionnaire for nonalcoholic steatohepatitis (CLDQ-NASH) was developed in a systematic manner for assessment of patient-reported outcomes. This instrument collects data on 36 items grouped into 6 domains: abdominal symptoms, activity/energy, emotional health, fatigue, systemic symptoms, and worry. We aimed to validate the CLDQ-NASH in a large group of patients with NASH.
We collected data from patients with biopsy-proven NASH enrolled in 2 international phase 3 trials of selonsertib (NCT03053050 and NCT03053063). Our final analysis comprised 1667 patients who completed the CLDQ-NASH (age, 58 ± 9 y; 40% male; 52% with cirrhosis; and 69% with type 2 diabetes). The CLDQ-NASH was administered before treatment initiation. A standard patient-reported outcome instrument validation pipeline with internal consistency and validity assessment was applied.
The domains of CLDQ-NASH showed good to excellent internal consistency: the Cronbach's α values were 0.80 to 0.94 and item-to-own-domain correlations were greater than 0.50 for 33 of 36 items. All items correlated to the greatest extent with their own domains (discriminant validity). Known-group validity tests indicated that the instrument consistently discriminated between patients with NASH based on the presence of cirrhosis (vs bridging fibrosis; all but 1 P value < .02), obesity (all but 1 P value < .001), psychiatric comorbidities (all P values < .0001), fatigue (all P values < .001), and type 2 diabetes (all but 1 P value < .01). Of the CLDQ-NASH domains, the highest correlated domains with the Short Form-36 were as follows: physical functioning for activity (rho = 0.70), mental health for emotional (rho = 0.72), vitality for fatigue (rho = 0.75), and body pain for systemic (rho = 0.72) (all P values < .0001). In contrast, the domains of abdominal and worry, which are disease-specific, did not correlate with the domains in the Short Form-36 (all rho ≤ 0.50).
We validated the CLDQ-NASH by an analysis of data from 1667 patients with biopsy-proven NASH enrolled in phase 3 trials, observing excellent psychometric characteristics of the instrument.
非酒精性脂肪性肝炎（NASH）慢性肝病问卷（CLDQ-NASH）是通过系统方法开发的，用于评估患者报告的结局。该工具收集了 36 个项目的数据，分为 6 个领域：腹部症状、活动/能量、情绪健康、疲劳、全身症状和担忧。我们的目的是在大量 NASH 患者中验证 CLDQ-NASH。
我们从参加塞隆昔布两项国际 3 期试验（NCT03053050 和 NCT03053063）的经活检证实的 NASH 患者中收集数据。我们的最终分析包括 1667 名完成 CLDQ-NASH 问卷的患者（年龄 58 ± 9 岁；40%为男性；52%有肝硬化；69%患有 2 型糖尿病）。CLDQ-NASH 在治疗开始前进行评估。应用了一个具有内部一致性和有效性评估的标准患者报告结局工具验证流程。
CLDQ-NASH 的各领域表现出良好至优秀的内部一致性：36 个项目中有 33 个项目的克朗巴赫's α 值为 0.80 至 0.94，项目与自身领域的相关性大于 0.50。所有项目与自身领域的相关性最大（区分效度）。基于肝硬化（桥接纤维化除外；除 1 项外均 P 值<0.02）、肥胖（除 1 项外均 P 值<0.001）、精神共病（均 P 值<0.0001）、疲劳（均 P 值<0.001）和 2 型糖尿病（除 1 项外均 P 值<0.01），该工具可一致区分 NASH 患者的不同组别。在 CLDQ-NASH 各领域中，与简明健康状况调查问卷 36 项（Short Form-36）相关性最高的领域如下：活动领域的躯体功能（rho=0.70）、情绪领域的心理健康（rho=0.72）、疲劳领域的活力（rho=0.75）和全身症状领域的身体疼痛（rho=0.72）（均 P 值<0.0001）。相比之下，与腹部症状和担忧相关的领域，与 Short Form-36 中的领域没有相关性（均 rho≤0.50）。
我们通过对参加 3 期试验的 1667 名经活检证实的 NASH 患者的数据进行分析，验证了 CLDQ-NASH 的有效性，观察到该工具具有优异的心理测量学特征。
