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ABSTRACT Objective To evaluate the safety and efficacy of the C5‐inhibitor crovalimab in patients with paroxysmal nocturnal hemoglobinuria (PNH). Methods PubMed, Scopus, Web of Science, and Cochrane Library were searched for studies on the use of the C5 inhibitor crovalimab in patients with PNH. Outcomes included hemolysis control (HC: LDH ≤ 1.5 × ULN), stabilized hemoglobin (SH), transfusion avoidance (TA), and safety endpoints. Pooled estimates were calculated using random‐effects models with 95% confidence intervals (CIs), and heterogeneity was assessed via the I 2 statistic. Results Four studies involving 275 patients were included. Pooled prevalence of HC was 83% (95% CI: 0.75–0.91), SH in 61% (95% CI: 0.48–0.75), and TA in 65% (95% CI: 0.56–0.74). At least one any‐grade Adverse Event (AEs) occurred in 90% (95% CI: 0.78–0.99), while Serious Adverse Events (SAEs) occurred in 14% (95% CI: 0.05–0.23). Treatment‐related AEs affected 47%, and infections occurred in 50%. Grade 3–5 AEs were seen in 19%, dose modifications in 2%, and treatment discontinuation in 0.4%. Conclusion Crovalimab showed encouraging efficacy and an acceptable safety profile in patients with PNH, supporting its role as a promising treatment option in PNH.
Martins et al. (Wed,) studied this question.