2.2. Hematologic Response Categories

Methods for analyzing treatment responses during the randomized period of PEGASUS have been published [21]. In summary, PNH experts reviewed the PEGASUS trial data to classify patients into the following EBMT response categories: complete response (i.e., patients require no transfusions, have stable hemoglobin levels in the normal range, and have no evidence of hemolysis), major response (i.e., patients require no transfusions, have normal hemoglobin levels, and have evidence of residual intravascular or extravascular hemolysis), good response (i.e., patients require no transfusions and have evidence of chronic mild anemia or hemolysis), partial response (i.e., patients have chronic moderate anemia and/or require occasional transfusions of <3 units/6 months), minor response (i.e., patients require regular transfusions of 3–6 units/6 months), and no response (i.e., patients require regular and frequent transfusions of >6 units/6 months) [3,21]. Response category inputs from the previous analysis of the 16-week randomized controlled period of PEGASUS are shown in Table 1 [21].

EBMT hematologic responses to pegcetacoplan and eculizumab in patients with a suboptimal response to prior eculizumab treatment.

EBMT, The European Society for Blood and Marrow Transplantation. ^a Eligibility for the PEGASUS trial required patients to have a suboptimal response to ≥3 months of stable dosing and consistent eculizumab treatment, with suboptimal response defined as hemoglobin levels < 105 g/L. For the results for other patient populations, see Table 6 and the Supplementary Materials. ^b Source: Risitano et al. [21].