Plozasiran for Managing Persistent Chylomicronemia and Pancreatitis Risk

医学 急性胰腺炎 优势比 胃肠病学 高甘油三酯血症 安慰剂 内科学 临床终点 甘油三酯 胰腺炎 入射(几何) 载脂蛋白B 置信区间 随机对照试验 内分泌学 胆固醇 病理 光学 物理 替代医学
作者
Gerald F. Watts,Robert S. Rosenson,Robert A. Hegele,Ira J. Goldberg,Antonio Gallo,Ann C. Mertens,Alexis Baass,Rong Zhou,Ma'An Muhsin,Jennifer Hellawell,Nicholas J. Leeper,Daniel Gaudet
出处
期刊:The New England Journal of Medicine [Massachusetts Medical Society]
被引量:29
标识
DOI:10.1056/nejmoa2409368
摘要

BackgroundPersistent chylomicronemia is a genetic recessive disorder that is classically caused by familial chylomicronemia syndrome (FCS), but it also has multifactorial causes. The disorder is associated with the risk of recurrent acute pancreatitis. Plozasiran is a small interfering RNA that reduces hepatic production of apolipoprotein C-III and circulating triglycerides.MethodsIn a phase 3 trial, we randomly assigned 75 patients with persistent chylomicronemia (with or without a genetic diagnosis) to receive subcutaneous plozasiran (25 mg or 50 mg) or placebo every 3 months for 12 months. The primary end point was the median percent change from baseline in the fasting triglyceride level at 10 months. Key secondary end points were the percent change in the fasting triglyceride level from baseline to the mean of values at 10 months and 12 months, changes in the fasting apolipoprotein C-III level from baseline to 10 months and 12 months, and the incidence of acute pancreatitis.ResultsAt baseline, the median triglyceride level was 2044 mg per deciliter. At 10 months, the median change from baseline in the fasting triglyceride level (the primary end point) was −80% in the 25-mg plozasiran group, −78% in the 50-mg plozasiran group, and −17% in the placebo group (P<0.001). The key secondary end points showed better results in the plozasiran groups than in the placebo group, including the incidence of acute pancreatitis (odds ratio, 0.17; 95% confidence interval, 0.03 to 0.94; P=0.03). The risk of adverse events was similar across groups; the most common adverse events were abdominal pain, nasopharyngitis, headache, and nausea. Severe and serious adverse events were less common with plozasiran than with placebo. Hyperglycemia with plozasiran occurred in some patients with prediabetes or diabetes at baseline.ConclusionsPatients with persistent chylomicronemia who received plozasiran had significantly lower triglyceride levels and a lower incidence of pancreatitis than those who received placebo. (Funded by Arrowhead Pharmaceuticals; PALISADE ClinicalTrials.gov number, NCT05089084.)
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