A comprehensive review of patient-reported outcomes in metabolic dysfunction-associated steatotic liver disease
Aybuke Sapmaz , Annette Paik , Linda Henry , Zobair M. Younossi
Metabolism and Target Organ Damage ›› 2024, Vol. 4 ›› Issue (4) : 42
A comprehensive review of patient-reported outcomes in metabolic dysfunction-associated steatotic liver disease
The global prevalence of obesity and type 2 diabetes has increased, contributing to an increased worldwide prevalence of metabolic dysfunction-associated steatotic liver disease (MASLD). Currently, one in three adults is affected by MASLD and/or its progressive form, metabolic dysfunction-associated steatohepatitis (MASH), making this liver disease a significant public health challenge. Along with MASH-related cirrhosis, these conditions are poised to become the leading causes of chronic liver disease and liver transplants in the near future. Given the growing burden of MASLD and MASH, it is crucial to understand their impact from the patients’ perspective. One way to do this is by assessing patient-reported outcomes (PROs), including health-related quality of life (HRQL). HRQL can be assessed using generic instruments like the short form 36 version (SF-36) and the European quality of life-5 dimensions questionnaire (EQ-5D), or disease-specific tools such as the chronic liver disease questionnaire for nonalcoholic steatohepatitis (CLDQ-NASH). Given the limitations of each instrument, the best approach generally involves using both generic and disease-specific instruments. Evidence indicates that HRQL scores are significantly lower in individuals with MASLD, especially in areas assessing physical activity and the ability to perform daily living tasks. Fatigue and impaired work productivity are also important PROs for those with MASLD/MASH. These decrements in PROs worsen with disease progression but appear to improve with disease regression, including improvements linked to treatment. In this context, measuring PROs enhances the assessment of other patient-centric outcomes and provides insights for the healthcare community to develop interventions that could improve both clinical and humanistic outcomes for individuals living with MASLD/MASH.
NAFLD / MAFLD / patient-reported outcome (PRO) / quality of life
| [1] |
|
| [2] |
|
| [3] |
|
| [4] |
|
| [5] |
Sanyal AJ, Van Natta ML, Clark J, et al; NASH Clinical Research Network (CRN). Prospective study of outcomes in adults with nonalcoholic fatty liver disease. N Engl J Med 2021;385:1559-69. PMCID:PMC8881985 |
| [6] |
|
| [7] |
|
| [8] |
|
| [9] |
|
| [10] |
|
| [11] |
|
| [12] |
|
| [13] |
|
| [14] |
|
| [15] |
|
| [16] |
|
| [17] |
Younossi ZM, Yilmaz Y, Yu ML, et al; Global NASH Council. Clinical and patient-reported outcomes from patients with nonalcoholic fatty liver disease across the world: data from the global non-alcoholic steatohepatitis (NASH)/non-alcoholic fatty liver disease (NAFLD) registry. Clin Gastroenterol Hepatol 2022;20:2296-306.e6. |
| [18] |
|
| [19] |
|
| [20] |
US Food and Drug Administration. Guidance for industry: patient-reported outcome measures: use in medical product development to support labelling claims. Available from: https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM193282.pdf. [Last accessed on 19 Nov 2024]. |
| [21] |
|
| [22] |
|
| [23] |
|
| [24] |
|
| [25] |
|
| [26] |
|
| [27] |
|
| [28] |
US Food and Drug Administration. Incorporating clinical outcome assessments into endpoints for regulatory decision-making. Available from: https://www.fda.gov/media/132505/download. [Last accessed on 19 Nov 2024]. |
| [29] |
|
| [30] |
|
| [31] |
|
| [32] |
|
| [33] |
EUROQQOL. EQ-5D-5L. Available from: https://euroqol.org/information-and-support/euroqol-instruments/eq-5d-5l/. [Last accessed on 20 Nov 2024]. |
| [34] |
|
| [35] |
|
| [36] |
|
| [37] |
|
| [38] |
|
| [39] |
|
| [40] |
|
| [41] |
|
| [42] |
|
| [43] |
|
| [44] |
FACIT.org. Functional assessment of chronic illness therapy - fatigue scale (FACIT-fatigue). Available from: https://www.facit.org/measures/facit-fatigue. [Last accessed on 19 Nov 2024]. |
| [45] |
|
| [46] |
|
| [47] |
|
| [48] |
|
| [49] |
|
| [50] |
|
| [51] |
|
| [52] |
|
| [53] |
|
| [54] |
|
| [55] |
|
| [56] |
|
| [57] |
|
| [58] |
|
| [59] |
|
| [60] |
|
| [61] |
|
| [62] |
|
| [63] |
|
| [64] |
|
| [65] |
|
| [66] |
|
| [67] |
Batchelder AW, Heo M, Foley JD, et al; HERO Study Group. Shame and stigma in association with the HCV cascade to cure among people who inject drugs. Drug Alcohol Depend 2023;253:111013. |
| [68] |
|
| [69] |
Younossi ZM, Alqahtani SA, Alswat K, et al; Global NASH Council. Global survey of stigma among physicians and patients with nonalcoholic fatty liver disease. J Hepatol 2024;80:419-30. |
| [70] |
Younossi ZM, AlQahtani SA, Funuyet-Salas J, et al; Global NASH Council. The impact of stigma on quality of life and liver disease burden among patients with nonalcoholic fatty liver disease. JHEP Rep 2024;6:101066. PMCID:PMC11252535 |
| [71] |
UNOS. Data and trends. Available from: https://unos.org/data/. [Last accessed on 19 Nov 2024]. |
| [72] |
|
| [73] |
Vitale A, Trapani S, Russo FP, et al; Associazione Italiana per lo Studio del Fegato (AISF).; Società Italiana Trapianti d’Organo (SITO).; Centro Nazionale Trapianti (CNT). Waiting list mortality and 5-year transplant survival benefit of patients with MASLD: an Italian liver transplant registry study. JHEP Rep 2024;6:101147. PMCID:PMC11399673 |
| [74] |
|
| [75] |
|
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|
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