THE SAFETY AND EFFICACY OF A NOVEL ANTI HYPERLIPIDEMIC MEDICATION, SEBELIPASE ALFA: A SYSTEMATIC REVIEW AND META-ANALYSIS

Abstract

Lysosomal acid lipase deficiency (LALD), a rare disorder of lipid metabolism, is treated with sebelipase alfa, a novel anti hyperlipidemia drug that is a recombinant human lysosomal acid lipase enzyme. This thorough review and meta-analysis evaluate the safety and efficacy of sebelipase alfa in the treatment of LALD. Methodology: A comprehensive search was conducted in PubMed, Cochrane, and other databases using keywords including safety, effectiveness, LALD, and sebelipase alfa. These included research published from 2013 to 2024. We considered both planned and retrospective clinical studies of sebelipase alfa versus standard of care (SOC) or placebo. The statistical analysis was conducted using RevMan 5.4.1, and the quality was evaluated using the Newcastle-Ottawa scale. Result: A total of 12 studies involving 755 individuals met the inclusion criteria. Lipid markers such as LDL-C (-39.46; 95% Cl: -43.57 to -35.35), total cholesterol (-23.75; 95% Cl: -28.38 to -19.11), triglycerides (-21.80; 95% Cl: -27.30 to 16.30), and ApoB (-20.96; 95% Cl: -25.32 to -16.61) are all significantly impacted by sebelipase alfa. There were also noticeable drops in ALT and AST values. HDL values marginally rise. On the other hand, weight increase (OR: 66.36; 95% Cl: 1.80 to 2444.55) and gastrointestinal side effects (OR: 19.38; 95% Cl: 5.49 to 68.33) were more common in individuals using Sebelipase alfa. Conclusion: Sebelipase alfa’s efficacy is supported by the significant improvements in lipid profiles and liver enzyme levels observed in LALD patients. Furthermore, cautious monitoring is necessary for potential side effects such as weight gain and gastrointestinal problems. To avoid adverse effects and improve dosage, more research is required.