OBJECTIVES: Paroxysmal nocturnal haemoglobinuria (PNH) is a rare blood condition, caused by complement-mediated haemolysis with persistent anaemia and fatigue. Currently approved treatments include complement 5 inhibitors (C5i). The aim of this study was to assess the impact of PNH despite C5i treatment on adult patients’ employment, ability to work, and overall level of activity impairment.

METHODS: Data were drawn from the Adelphi PNH Disease Specific Programme™, a cross-sectional survey of PNH-treating physicians and their patients from Canada, France, Germany, Italy, Japan, Spain, and the USA (collected Jan-July 2022). Patients completed a survey comprising demographics, employment status, and the Work Productivity and Activity Impairment (WPAI) questionnaire (7-day recall, four scores calculated % absenteeism, presenteeism, overall work impairment and activity impairment; higher scores mean higher impairment). Physicians reported patient’s current treatment, dates of diagnosis and treatment initiation. Descriptive analyses were conducted on data from C5i-treated PNH patients.

RESULTS: A total of 143 patients receiving C5i were included in the analysis; mean (SD) age of 48.2 (14.8) years (median 47.0); 52% were male, and 50% were employed. Mean (SD) disease duration was 2.5 (2.8) years and duration on C5i therapy was 1.8 (2.1) years. Of these patients, 15% (n=19) had stopped working, 21% (n=27) had changed to flexible working hours, and 11% (n=14) had accepted reduced working responsibilities due to PNH (currently or in the past). For employed patients, mean (SD) absenteeism was 9% (16.3), presenteeism 26% (20.0), and overall work impairment 32% (23.6). Activity impairment for all patients was 34% (23.2).

CONCLUSIONS: Despite receiving C5i treatment, PNH impacts patients’ ability to work and perform daily activities. Better therapies are required to provide the comprehensive control of PNH. This may positively impact employment, productivity, and overall activity of PNH patients.