Physician and Patient Perception of Disease, Symptomatology, and Treatment Administration Preference in Paroxysmal Nocturnal Haemoglobinuria: Results from a Real-World Survey

INTRODUCTION: Paroxysmal nocturnal haemoglobinuria (PNH) is an ultra-rare condition, characterised by complement-mediated red blood cell breakdown, resulting in haemolytic anaemia. Associated symptoms, such as fatigue, impact on patient quality of life. Little is known about the comparative perceptions of symptom burden and treatment preferences among patients and physicians. METHODS: This study analysed secondary data from the Adelphi Real World PNH Disease Specific Programme™, a cross-sectional survey of physicians and patients with PNH in France, Germany, Italy, Spain, China, and Japan conducted January-November 2022. Haematologists and haematologist-oncologists reported demographic and clinical data for ≤10 consecutive consulting adult patients with PNH. Only patients with both physician-reported and patient-reported data were included in the study. Patients and physicians reported on clinical signs and symptom burden, treatment satisfaction, and preferred route of administration. Bivariate analyses were used to compare physician and patient responses. RESULTS: Forty-two physicians reported data for 225 patients. Median (interquartile range) patient age was 44.0 (35.0-56.0), 56.9% were male and 96.0% received treatment at survey. Most common clinical signs and symptoms were fatigue, anaemia, haemoglobinuria, dyspnoea, and headaches. All common signs/symptoms, except haemoglobinuria, were reported as significantly more frequent and severe by patients than physicians (p < 0.05). Most bothersome signs/symptoms reported were anaemia (physicians: 31.3%, patients: 21.3%) and fatigue (physicians: 29.9%, patients: 45.5%). Physicians were significantly more satisfied with treatment than patients (p < 0.001), regardless of treatment. Although oral administration was preferred by both physicians (60.2%) and patients (74.2%), there was significant difference in overall preference (p < 0.001), with 30.8% of physicians preferring intravenous administration, compared to 16.3% of patients. CONCLUSION: There was significant patient-physician disconnect in perception of PNH symptoms burden, treatment satisfaction, and administration preferences. Improved communication and patient involvement in disease management may contribute to optimised patient outcomes and quality of life.