Effect of Essential Phospholipids in Metabolic Dysfunction-Associated Steatotic Liver Disease: A Randomised Phase 4 Clinical Trial

必需磷脂对代谢功能障碍相关脂肪肝疾病的影响:一项随机4期临床试验

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Abstract

BACKGROUND AND AIMS: Metabolic dysfunction-associated steatotic liver disease (MASLD) poses a significant health burden and impacts quality of life (QoL). This study evaluates the effects of essential phospholipids (EPL) on liver steatosis, QoL, and other liver and metabolic parameters in patients with MASLD and associated comorbidities. METHODS: In this multicenter, double-blind, randomised, placebo-controlled phase 4 clinical trial, patients with MASLD and type 2 diabetes, hyperlipidemia, or obesity received either EPL or placebo, alongside standard of care. PRIMARY ENDPOINT: change in hepatic steatosis from baseline to 6 months (measured by Controlled Attenuation Parameter [CAP] score); secondary endpoints: changes in QoL (measured by the Chronic Liver Disease Questionnaire [CLDQ-MASLD]), symptom changes; other endpoints: other liver, metabolic, and lipid parameters, and safety. RESULTS: Of 193 randomised patients, 165 constituted the modified intention-to-treat population (median age: 56.5 years [EPL arm], 55.0 years [placebo arm]). More than ¾ of patients were obese and had CAP score ≥ 280 dB/m. At 6 months, EPL treatment significantly reduced CAP (p = 0.0269) versus placebo. This effect was evident at 3 months (p = 0.0049) and sustained until 3 months post treatment (p = 0.0234). QoL total score showed numerical improvement, with statistically significant improvement in fatigue subscore (p = 0.0229) with EPL versus placebo at 6 months. EPL significantly improved HbA1c levels (p = 0.0069) over 6 months. No safety concerns arose. CONCLUSIONS: The beneficial effects of EPL on hepatic steatosis, QoL and glycemic control, and its favourable safety profile make it a promising candidate for managing steatosis and enhancing overall liver health in MASLD patients with cardiometabolic risk. TRIAL REGISTRATION: The trial was registered in the EudraCT (2021-006069-39).

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