Summary

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• This summary is based on the review of four systematic review(s)/meta-analysis(es). ^[1-4]
• Pegcetacoplan (Empaveli) demonstrated significant reductions in lactate dehydrogenase (LDH) levels in treatment-naive patients with paroxysmal nocturnal hemoglobinuria (PNH), with a pooled decrease of -1462.0 U/L for treatment durations ≤26 weeks and -1696.5 U/L for >26 weeks. Hemoglobin concentrations increased by 1.4 g/dL and 1.9 g/dL for the same treatment durations, respectively.
• Pegcetacoplan improved transfusion independence, preventing the need for transfusions in at least 50% of PNH patients during treatment. Clinically meaningful improvements in fatigue, measured by FACIT-F scores, were also observed, with pooled increases of 6.8 for ≤26 weeks and 9.5 for >26 weeks.
• No significant differences in effectiveness across specific population types or subgroups were highlighted, though pegcetacoplan is noted as an option for patients refractory to eculizumab.
• In studies involving pegcetacoplan, the proportion of patients reporting adverse events ranged from 33.3% (2 of 6 patients) to 17.1% (7 of 41 patients). Specific adverse events were not detailed.
• No significant safety concerns or adverse effects were noted, and no comparative safety data were provided for pegcetacoplan relative to other treatments such as eculizumab or ravulizumab.
• The population types mentioned include patients with paroxysmal nocturnal hemoglobinuria (PNH) who are refractory to eculizumab, as well as those who may benefit from switching from C5 inhibitors to C3 inhibitors like pegcetacoplan. Pegcetacoplan is identified as a therapeutic option for patients who do not respond to eculizumab.