Study Compares Effectiveness of Pegcetacoplan Versus Ravulizumab for PNH

Researchers compared the effectiveness of pegcetacoplan versus ravulizumab in patients with paroxysmal nocturnal hemoglobinuria (PNH) and found that pegcetacolplan resulted in improvements in transfusion avoidance, hemoglobin stabilization, and fatigue, as well as a reduction in the total number of units of packed red blood cells transfused. The results were presented at the 2020 ASH Annual Meeting.

Using a matching-adjusted indirect comparison (MAIC), researchers assessed data from the phase III PEGASUS study, which analyzed pegcetacoplan versus eculizumab among patients with PNH, and the phase III 302 study, which compared ravulizumab and eculizumab among patients with PNH. Propensity score weighting was used to balance demographic and clinical characteristics and to adjust for cross-study differences in baseline characteristics.

The MAIC study included 68 patients from PEGASUS (36 treated with pegcetacoplan; 32 treated with eculizumab) and 195 from the 302 study (97 treated with ravulizumab; 98 treated with eculizumab).

In the PEGASUS trial, 51.5% of patients received four or more transfusions in the 12 months prior to screening. Pegcetacoplan was better associated with transfusion avoidance than ravulizumab (difference, 71.4%; 95% confidence interval [CI], 53.5-89.3; P<0.0001). Patients treated with pegcetacoplan required 5.7 fewer mean units of packed red blood cells transfused during treatment than ravulizumab (95% CI, –7.2 to –4.2; P<0.0001). Pegcetacoplan was also associated with 75.5% more patients achieving hemoglobin stabilization than ravulizumab (95% CI, 56.4-94.6; P<0.0001).

Mean change from baseline in Functional Assessment of Chronic Illness Therapy-Fatigue score was 8.8 points (95% CI, 4.2-13.3; P<0.0001), “suggesting that pegcetacoplan is associated with an improvement approximately three times greater than the clinically meaningful improvement of three points than ravulizumab,” the researchers noted.

The study is limited by its MAIC design.


Bhak RY, Mody-Patel N, Baver SB, et al. Comparative Effectiveness of Pegcetacoplan Versus Ravulizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria Previously Treated with Eculizumab: A Matching-Adjusted Indirect Comparison. Abstract 2581. Presented at the 62nd American Society of Hematology Annual Meeting & Exposition, Dec. 2-11, 2020.