Abstract
BACKGROUND: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, life-threatening disorder characterized by complement-mediated hemolysis. Crovalimab, a novel anti-C5 monoclonal antibody, may offer a more convenient alternative to current therapies, highlighting the need for a comprehensive analysis of its efficacy and safety. METHODS: A comprehensive search was conducted through PubMed, Embase, Cochrane, and clinicaltrials.gov using relevant keywords from inception until January 2025. Statistical calculation was conducted using MetaXL 5.3 by employing a random effects model to estimate the pooled event rates with 95% CI. Heterogeneity was assessed using the I (2) and χ(2) statistics. RESULTS: This meta-analysis included four studies involving 275 patients with a mean age of 41.87 ± 13.34 years with rate of hemolysis control of 86% (95% CI: 77%-94%, I (2) = 73%) and transfusion avoidance in 77% of patients (95% CI: 56%-93%, I (2) = 90%), breakthrough hemolysis of 10% (95% CI: 6%-13%, I (2) (=) 0%) and hemoglobin stabilization in 69% patients (95% CI: 43%-90%, I (2) = 98%). The incidence rate of any adverse event was 89% (95% CI: 76%-98%, I (2) = 87%) and serious adverse events 15% (95% CI: 7%-25%, I (2) = 74%). CONCLUSION: We observed high rates of hemolysis control and transfusion avoidance but significant adverse events. Study heterogeneity and publication bias limit generalizability, highlighting the need for larger, more robust trials. Trial Registration: The authors have confirmed clinical trial registration is not needed for this submission.