Real-World Data Show Resmetirom Mirrors Clinical Trial Success in MASH Patients
Key Insights
Real-world data from 72 MASH (search) patients prescribed resmetirom show clinical characteristics and noninvasive test results consistent with phase 3 trial data.
The study, conducted across two US tertiary care centers, suggests resmetirom's effectiveness in clinical practice mirrors its performance in controlled trials.
Findings indicate appropriate patient selection for resmetirom can be achieved using noninvasive testing, potentially negating the need for liver biopsies.
New data presented at the American College of Gastroenterology (ACG) 2024 Annual Scientific Meeting offers the first real-world insights into the use of resmetirom (Rezdiffra) in patients with metabolic dysfunction-associated steatohepatitis (search) (MASH (search)). The study, involving 72 patients prescribed resmetirom at two US tertiary care centers between March and May 2024, suggests that the drug's effectiveness in clinical practice aligns with results from phase 3 clinical trials.
The research, presented by Naim Alkhouri, MD, from Arizona Liver Health (search) and Indiana University, examined patient characteristics and noninvasive test (NIT) results following resmetirom's FDA approval on March 14, 2024. Resmetirom, a thyroid hormone receptor-β (search) selective agonist, received accelerated approval for noncirrhotic MASH (search) with moderate to advanced fibrosis based on 18 clinical studies.
Patient Demographics and Treatment
The cohort's median age was 56 years (range, 20–80), with 40.3% being male. Resmetirom was administered at doses of 60 mg (1.4%), 80 mg (47.2%), and 100 mg (51.4%). A significant proportion of patients had obesity (79%) and type 2 diabetes (41.7%). Concomitant medications included insulin (5.6%), GLP-1 agonists (23.6%), statins (41.7%), and aspirin (20.8%).
Noninvasive Testing Results
Fibroscan data indicated that 45.8% of patients had a liver stiffness measurement between 10–20 kPa, and 80.6% had a CAP ≥ 280 dB/m, suggesting advanced fibrosis and steatosis, respectively. These findings are consistent with the patient profiles in the MAESTRO-NASH trial.
Implications for Clinical Practice
"What we are showing here is that in the real world, we are able to identify appropriate patients to be prescribed resmetirom based on noninvasive testing without the need for liver biopsy, and that the patient characteristics and the NIT data are very consistent with MAESTRO-NASH, implicating that we can really replicate the results from the phase 3 program, hopefully, in the real world," said Alkhouri. He added, "This is the first glimpse. We continue to collect more data… Our goal is to demonstrate that resmetirom was equally as effective in the real world as in the phase 3 clinical trials."
The ongoing data collection aims to further validate resmetirom's efficacy in routine clinical settings, potentially solidifying its role as a key therapeutic option for MASH (search) patients.