Efficacy and Safety of Crovalimab in Paroxysmal Nocturnal Hemoglobinuria (PNH): A Systematic Review and Meta-Analysis.

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 χ2 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.

UNLABELLED: Trial Registration: The authors have confirmed clinical trial registration is not needed for this submission.