Tracking Symptoms and Treatment of Paroxysmal Nocturnal Hemoglobinuria Using a Mobile App in the US A 6-Month Observational Study on Quality of Life and Treatment Patterns

What this Trial Is About

Researchers are studying Paroxysmal Nocturnal Hemoglobinuria PNH to understand the range of symptoms, treatment use, and overall health-related quality of life experienced by patients with this condition. The study focuses on collecting real-world data, especially from home-reported outcomes, to capture symptom changes and treatment patterns, including for those using orally administered iptacopan. Participants will use the Folia mobile app to enroll, consent, and report their symptoms, treatments, and quality of life over six months. The study is observational, meaning treatments such as eculizumab, ravulizumab, pegcetacoplan, or iptacopan will be used based on clinical decisions and not assigned by the study. Monthly surveys and possible integration of health records will supplement data collection. During the study, participants will regularly track their symptoms and treatment changes through the app. Researchers will measure PNH symptom burden at the start and after six months, as well as monitor treatment use, management of flare events, treatment switching, and quality of life. The study aims to provide a comprehensive view of living with PNH over this half-year period.

Home Reported Outcomes in PNH