Summary

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• Empaveli (pegcetacoplan) is indicated for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH).
• This summary is based on the review of four systematic review(s)/meta-analysis(es). ^[1-4]
• Hemoglobin Levels and LDH Reduction: Pegcetacoplan treatment in PNH patients resulted in an average hemoglobin increase of 3.68 g/L (PADDOCK study) and 2.37 g/L (Hillmen et al.), with LDH levels showing a mean reduction of -15 ± 43 U/L (Hillmen et al.) and stability within <1.5× upper limit of normal (De Castro et al.).
• Transfusion Independence and Comparative Effectiveness: Pegcetacoplan improved transfusion independence in PNH patients. When compared with other complement inhibitors (eculizumab, ravulizumab), pooled decreases in LDH were -1462.0 U/L for ≤26 weeks and -1696.5 U/L for >26 weeks of treatment, with hemoglobin increases of 1.4 g/dL and 1.9 g/dL, respectively. At least 50% of patients achieved transfusion avoidance across all durations.
• Quality of Life: Complement inhibitors, including pegcetacoplan, led to clinically meaningful improvements in FACIT-F scores, with pooled increases from 6.8 before to 9.5 after 26 weeks of treatment.
• Pegcetacoplan Adverse Events: Adverse events were reported in patients treated with pegcetacoplan across studies: 2 out of 6 patients (De Castro et al.), 7 out of 23 patients (PADDOCK study), and 7 out of 41 patients (Hillmen et al.).
• Safety Comparison: Specific safety outcomes comparing pegcetacoplan to eculizumab and ravulizumab were not detailed in the reviewed studies.
• Population Types and Subgroup Considerations: This section focuses on adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH), with 59.83% of participants being female in pegcetacoplan studies. There is also an emphasis on patients unresponsive to eculizumab, with individualized treatment plans recommended based on the mechanism of refractoriness.