Efficacy and Safety of the C3 Inhibitor Pegcetacoplan in Paroxysmal Nocturnal Hemoglobinuria: A Systematic Review and Meta-Analysis.
Eur J HaematolJun 19, 2026 (epub)
Kleuber Arias Meireles Martins, Mariana Menezes Corcinio, Bruno Arias Meireles Martins et al.
✦ AI-curated · Sources linked
In 30 seconds
This systematic review and meta-analysis evaluated the efficacy and safety of pegcetacoplan, a complement C3 inhibitor, in 271 patients with paroxysmal nocturnal hemoglobinuria (PNH). The study found that pegcetacoplan led to transfusion avoidance in 80.6% of patients and LDH normalization in 68.5%, indicating its potential as a therapeutic option for PNH.
Key findings
- Transfusion avoidance was achieved in 80.6% of patients.
- LDH normalization occurred in 68.5% of patients.
- Hemoglobin normalization was observed in 42.9% of patients.
- Any-grade adverse events occurred in 83.5% of patients.
Why it matters
Understanding the efficacy and safety of pegcetacoplan in PNH can help clinicians make informed decisions about treatment options, particularly for patients who do not respond to existing therapies.