Bone Marrow Failure Disorders

Clinically Meaningful and Long-Term Improvements in Fatigue With the C3 Inhibitor Pegcetacoplan in Paroxysmal Nocturnal Hemoglobinuria: Post-hoc Analyses From the PEGASUS Study Week 48

At the ISTH 2021 Congress, held virtually from July 17–21, David Cella, PhD, of Northwestern University’s Feinberg School of Medicine, and coinvestigators reported on changes in fatigue at week 48 in the phase 3 PEGASUS trial (abstract LPB0118). This was a randomized, open-label, active-comparator-controlled study in 80 adults with PNH who had hemoglobin levels less than 10.5 g/dL while on treatment with eculizumab (ECU). After 16 weeks, pegcetacoplan (PEG) was superior to eculizumab in improving both hemoglobin levels and fatigue, and patients were switched to PEG after a randomized controlled period. In the current post-hoc analysis, the Functional Assessment of Chronic Illness Therapy–Fatigue (FACIT-F) scale was used to assess fatigue, with changes by treatment assessed from baseline to week 16, and from week 16 to week 48. The investigators reported that there were “clinically meaningful improvements in fatigue among patients taking PEG, continuously through Week 48 in PEGASUS, and in those who switched from ECU.” They noted that this effect “was consistent across all the items of FACIT-F, demonstrating that PEG meaningfully reduces fatigue levels and maintains this effect over time by increasing Hb levels.”

International Society on Thrombosis and Haemostasis 2021