Meaningful differences in patient-reported outcome measurement scores in liver disease

Abstract

Background: 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.

Methods: 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.

Results: 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.

Conclusions: 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.

Keywords: chronic liver disease; health-related quality of life; meaningful score difference; minimal important difference; patient-reported outcome measurement.

Graphical abstract

FIGURE 1

PROMIS-29 Profile domains scores by clinical setting and stage of liver disease. HRQOL for each domain was poorer in inpatients versus outpatients with cirrhosis (all p-values <0.001). When comparing those with and without cirrhosis, HQROL was similar except for more depression in those with cirrhosis (p<0.05). Finally, compared to those pre-transplant patients (inpatients and outpatients with cirrhosis), post-transplant participants had improved HRQOL in all domains measured (p<0.001). *APSRA, ability to participate in social roles and activities; SPDSA, satisfaction with participation in discretionary social activities. Abbreviations: HRQOL, health-related quality of life; PROMIS, Patient-Reported Outcomes Measurement Information System.

FIGURE 2

PROMIS-29 Profile domain and summary score meaningful score differences by clinical setting and stage of liver disease using (A) distribution-based and (B) anchor-based methodology. Abbreviations: MSD, meaningful score difference; PROMIS, Patient-Reported Outcomes Measurement Information System.

FIGURE 3

Triangulation of PROMIS-29 Profile domain and summary score meaningful score differences. MSDs using the distribution-based method are displayed alongside anchor-based MSDs. Abbreviations: MSD, meaningful score difference; PROMIS, Patient-Reported Outcomes Measurement Information System.