Evaluating the treatment of patients with paroxysmal nocturnal hemoglobinuria in Brazil: disparities in access and treatment persistence

BACKGROUND: Paroxysmal nocturnal hemoglobinuria is a rare, life-threatening disease that significantly affects quality of life. Despite therapeutic advances, challenges like treatment burden and limited care access persist. This study aimed to evaluate the geographic distribution of paroxysmal nocturnal hemoglobinuria patients in Brazil and explore how travel distance to treatment centers may influence treatment persistence. METHODS: A mixed-methods study was conducted combining quantitative analysis of administrative claims data from the Brazilian Unified Health System and qualitative insights from patient focus groups. The quantitative component included patients with at least one ICD-10 D59.5 code between January 2010 and December 2023. The qualitative component involved a focus group of adult paroxysmal nocturnal hemoglobinuria patients registered with the Brazilian Lymphoma and Leukemia Association (ABRALE). Descriptive statistics were used to summarize demographic, geographic, and treatment-related data. RESULTS: A total of 769 patients were identified, with 42.4% receiving eculizumab. The mean time to treatment initiation was 172.9 days. Spatial analysis revealed substantial regional variations, with eculizumab-treated patients traveling an average of 87.5 km for care. Persistence analysis showed that 21.2% and 61.0% of patients were non-persistent based using two different methodological approaches. Focus group participants reported limited disease awareness, long travel distances, and extended waiting times, all of which negatively impacted their daily lives and treatment adherence. CONCLUSION: This study highlights geographic disparities in access to paroxysmal nocturnal hemoglobinuria treatment in Brazil and suggests that the travel burden may contribute to treatment non-persistence. These findings underscore the need for more accessible and patient-centered therapeutic options to promote equitable healthcare delivery.