Pegcetacoplan superior to eculizumab in paroxysmal nocturnal hemoglobinuria

Patients with paroxysmal nocturnal hemoglobinuria (PNH) treated with pegcetacoplan had greater improvements in hemoglobin and clinical and hematologic outcomes compared to those treated with eculizumab, according to a study published in the New England Journal Medicine.

In this phase 3 open-label, controlled trial, after a 4-week run-in phase in which all patients received pegcetacoplan plus eculizumab, 41 patients with PNH were randomly assigned to receive subcutaneous pegcetacoplan monotherapy and 39 patients to intravenous eculizumab. The primary endpoint was the mean change in hemoglobin level from baseline to week 16.

From baseline to week 16, pegcetacoplan was superior to eculizumab in change in hemoglobin level. Transfusions were no longer needed in 85% and 15% of patients receiving pegcetacoplan and eculizumab, respectively. Noninferiority of pegcetacoplan to eculizumab was shown for the change in absolute reticulocyte count but not for the change in lactate dehydrogenase level. There was an improvement from baseline in Functional Assessment of Chronic Illness Therapy–Fatigue scores in patients receiving pegcetacoplan.

Injection site reactions, diarrhea, breakthrough hemolysis, headache, and fatigue were the most common adverse events.

Reference
Hillmen P, Szer J, Weitz I, et al. Pegcetacoplan versus eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2021; 384:1028-1037. DOI: 10.1056/NEJMoa2029073