Abstract
INTRODUCTION: Pemafibrate was well tolerated and effective in clinical trials that led to its approval to treat hyperlipidemia. However, these clinical trials typically have limitations and may not sufficiently assess the performance of pemafibrate in the real-world setting. Therefore, the aim of this study was to evaluate the long-term safety and efficacy of pemafibrate in patients with hyperlipidemia in Japan. METHODS: This was a prospective, multicenter, open-label, observational post-marketing surveillance (PMS) study. Patients with hyperlipidemia were observed for 24 months from the start of pemafibrate treatment. Safety evaluations included the incidence of adverse drug reactions (ADRs), subgroup analysis of ADRs, and changes in laboratory test values. Efficacy was determined by the changes in lipid test values from baseline. RESULTS: A total of 3672 cases were registered from 612 facilities in Japan. In the safety analysis, 68.1% of patients were male, mean age was 60.8 years, and 37.8% were switched to pemafibrate from other dyslipidemia agents. In total, 147 patients (4.07%) experienced ADRs; serious ADRs were reported in 8 patients (0.22%). The most common ADRs included abnormalities in laboratory tests (n = 60; 1.66%), hepatobiliary disorders (n = 24, 0.66%), musculoskeletal and connective tissue disorders (n = 14, 0.39%), and metabolic and nutritional disorders (n = 14, 0.39%). No incidents of rhabdomyolysis occurred, and 37 (1.02%) patients experienced rhabdomyolysis-related ADRs. Renal-related ADRs occurred in 13 patients (0.36%). When subdivided by patient background, the incidence of ADRs was significantly higher in patients aged ≥ 75 years than < 75 years (P = 0.028). There were no significant differences in other subgroups. The mean percent change in mixed fasting and non-fasting triglyceride levels was - 35.2% (P < 0.001). High- and low-density lipoprotein cholesterol levels both increased significantly. CONCLUSION: This PMS study demonstrated the safety and efficacy of pemafibrate after long-term administration in patients with hyperlipidemia.