Pegcetacoplan Delivers Real-World Therapeutic Benefits and Reduces Disease Burden for Patients With Paroxysmal Nocturnal Haemoglobinuria: A Systematic Literature Review of Pegcetacoplan Real-World Clinical and Patient-Reported Outcomes.
Eur J HaematolMay 25, 2026 (epub)
Juan Carlos Vallejo Llamas, Koo Wilson, Zalmai Hakimi et al.
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In 30 seconds
This systematic literature review evaluated real-world evidence for pegcetacoplan in adults with paroxysmal nocturnal haemoglobinuria (PNH). The review included 39 studies and found that pegcetacoplan treatment led to a median hemoglobin increase from 8.1-9.6 g/dL at baseline to 12.0 g/dL at 12 months, demonstrating significant therapeutic benefits and reduced disease burden.
Key findings
- Median hemoglobin increased from 8.1-9.6 g/dL at baseline to 12.0 g/dL at 12 months.
- Lactate dehydrogenase levels decreased from a median of 543.0 to 161.7 U/L after 3 months.
- Red blood cell transfusion dependence was reduced after pegcetacoplan treatment.
- FACIT-Fatigue scores improved from a mean of 28.4 to 38.6 at 3 months.
Why it matters
The findings highlight pegcetacoplan's potential to improve hematological parameters and quality of life in patients with PNH, addressing significant morbidity associated with the disease. Clinicians should consider these outcomes when evaluating treatment options.