Insights from the European Nontuberculous mycobacterial pulmonary disease PAtient Disease Experience (ENPADE) survey- exploring disease burden and impact.

Abstract

Background: Nontuberculous mycobacterial pulmonary disease (NTM-PD) poses substantial diagnostic and management challenges, particularly among individuals with pre-existing lung conditions and/ or immunodeficiencies. NTM-PD can severely impair lung function and quality of life, potentially leading to both increased healthcare costs and mortality. There is a lack of comprehensive understanding of the disease burden and healthcare gaps from the patients' perspective. The European NTM-PD Patient Disease Experience (ENPADE) survey aimed to collect insights into these aspects.

Methods: The survey aim was addressed by several methods. First, an online questionnaire was carried out from July 2021 to February 2022 across eight European countries for quantitative data collection. Additionally, semi-structured qualitative patient interviews were conducted with a subset of patients, eliciting their insights on the aspects surveyed. Descriptive statistics were used for quantitative analysis and interview outcomes were categorised along the online questionnaire for qualitative analysis.

Results: A total of 543 patients participated in the survey and 23 patients were interviewed. Satisfaction with care received before and after diagnosis was scored, on average, moderate with 32% "highly satisfied" patients and 25% "highly dissatisfied" patients across the aspects surveyed. Dissatisfaction was expressed particularly regarding referral and access to expert care, and information received on their disease and its management. Patients reported high restrictions in daily life (49%), work (31%), and social activities (43%), often leading to substantial emotional distress, such as experiencing an increase in feeling depressed or anxious (82%). Interviews with patients highlighted a need for improved disease information, faster diagnosis, and enhanced physician-patient relationships.

Conclusions: The ENPADE survey outcomes revealed dissatisfaction among patients with care and restrictions in daily life, work, and social activities, often leading to emotional distress. These findings underscore the need for improved disease information, standardised care, and enhanced physician-patient relationships with appropriate support measures.