Resmetirom-eligible population among US adults: An estimation analysis based on NHANES 2017–March 2020

Abstract

Background: Resmetirom received FDA approval for treating adults with noncirrhotic metabolic dysfunction–associated steatohepatitis (MASH) and moderate-to-advanced liver fibrosis (stages F2–F3). Here, we sought to estimate the eligible U.S. adult population for resmetirom therapy, with secondary analysis focusing on individuals with type 2 diabetes mellitus (T2DM). Methods: A cross-sectional analysis was conducted using data from the National Health and Nutrition Examination Survey (NHANES) 2017–March 2020 cycle. Two eligibility scenarios were examined: a liberal scenario requiring ALT > 17 U/L for women or > 20 U/L for men, controlled attenuation parameter (CAP) > 280 dB/m, and liver stiffness measurement (LSM) > 8 kPa; and a restrictive scenario requiring ALT > 30 U/L for both sexes, CAP > 280 dB/m, and LSM > 10 kPa. The analysis incorporated sampling weights to generate nationally representative estimates. Results: The study cohort included 7244 adults (mean age 49.08 y, 49.9% male) with a mean BMI of 29.61 kg/m2, mean CAP 263.35 dB/m, and mean LSM 5.8 kPa. An estimated 8.3 million (95% CI: 6.6–9.9 million) adults met the liberal eligibility criteria, while 2.3 million (95% CI: 1.4–3.2 million) met the restrictive criteria. Patients meeting restrictive criteria were predominantly male (76.2% vs. 59.9%) and younger (mean age 46.7 vs. 48.3 y), with similar BMI (38.6 vs. 38.1 kg/m2). Among adults with T2DM, 3.5 million (95% CI: 2.4–4.5 million; 12.2%) met liberal, whereas 0.85 million (95% CI: 0.5–1.2 million; 3.0%) restrictive criteria. Conclusions: A substantial proportion of U.S. adults meet eligibility criteria for resmetirom treatment, with estimates varying significantly based on the stringency of selection criteria.