Development and Validation of a Symptom-Based Patient-Reported Outcome Measure for MASH: The CLDQ-MASH Symptom...

Area of Science:

• Hepatology
• Patient-Reported Outcomes
• Clinical Assessment

Background:

• Metabolic dysfunction-associated steatohepatitis (MASH) presents a significant symptom burden for patients.
• Currently, no validated patient-reported outcome (PRO) instrument specifically targets MASH symptoms.
• This study aimed to develop and validate a novel MASH Symptom Questionnaire.

Purpose of the Study:

• To create and validate a disease-specific PRO instrument for MASH symptoms.
• To establish a reliable tool for assessing symptom burden in MASH patients.
• To provide a standardized measure for clinical research and practice.

Main Methods:

• Developed a questionnaire using items from existing instruments and expert input (n=2,066).
• Utilized exploratory factor analysis to refine the instrument to 36 items across 8 domains.
• Validated the questionnaire using an independent sample (n=2,541) and assessed psychometric properties.

Main Results:

• The 36-item, 8-domain MASH Symptom Questionnaire demonstrated high internal consistency (Cronbach's alpha >0.80 for 7 domains).
• Scores significantly correlated with disease severity (histology, liver stiffness) and comorbidities (diabetes, hypertension, fatigue).
• The instrument showed strong validation properties in both development and independent validation samples.

Conclusions:

• The CLDQ-MASH Symptom Questionnaire (CLDQ-MASH SQ) is the first validated, disease-specific tool for MASH symptoms.
• It exhibits good psychometric properties and potential for comprehensive assessment of disease burden.
• Further external validation may establish the CLDQ-MASH SQ as a standard in MASH research and clinical care.