MDS-278 Mixed-Methods Study Assessing the Impact of C3 Inhibition on the Emotional and Physical Burden of Living With Paroxysmal Nocturnal Hemoglobinuria

Context: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired hematologic condition associated with significant morbidity, mortality, and impact on health-related quality of life (HRQoL). The burden of disease and experience of PNH patients on treatment is not well characterized in published scientific literature. Objective: The objective was to characterize the emotional and physical burden of illness of PNH and assess the real-world impact of pegcetacoplan, a recently approved C3 inhibitor, in addressing this disease burden. Design: This mixed-methods study enrolled consenting adult PNH patients (>21 years old) in the US receiving treatment with pegcetacoplan for at least 3 months for in-depth telephone interviews. All respondents had received treatment with a C5 inhibitor prior to switching to pegcetacoplan. Results: Interim analysis results from 14 respondents (of a total 16 planned) are reported. Enrolled patients had a median age of 41.5 years (range 24–77) and an average of 5.8 months of pegcetacoplan experience. Patients described life with PNH as frustrating and emotionally exhausting, significantly impacting their HRQoL despite C5-inhibitor treatment. Fatigue was reported as the Most Bothersome Symptom by 86% of patients, as its persistence limits their ability to carry out daily commitments and activities, such as work/school and hobbies. After switching from C5 inhibitors to pegcetacoplan, patients reported a reduction in fatigue persistence (23% improvement on a 7-point Likert scale compared to C5 inhibitors), supporting a significant improvement in their physical ability, including ability to walk or jog (+60%), stress levels (+58%), and ability to carry out daily commitments, such as work or school (+44%). Patients also reported an overall improvement in quality of life on pegcetacoplan (+28%). Patients (93%) described feeling better or energized since initiating treatment with pegcetacoplan and believed that the therapy has given them the ability to regain a more normal life. Conclusions: This study documents the high emotional and physical burden PNH has on patients' lives reported using the Voice of the Patient. Study participants reported improvement in daily life quality measures, such as reduction in fatigue and stress and increases in ability to conduct daily activities.