Home Reported Outcomes in PNH

The study aims to longitudinally capture the full spectrum of symptoms, treatment
utilization, and overall Health-Related Quality of Life (HRQoL) experienced by PNH
patients. By primarily utilizing home reported outcomes (HRO) data on symptom burden and
treatment usage, supplemented with patient-reported outcome (PRO) measures, the study
seeks to establish a new real-world data (RWD) source to understand symptom variability
and HRQoL among PNH patients, including those receiving orally administered iptacopan. The study will be prospective and observational, conducted over an initial period of six
months per individual from the point of study enrollment. Participants will utilize the
Folia mobile app to enroll, consent, and complete all study activities. A hybrid
recruitment method of clinic referrals and community referrals will be employed to
identify participants, who will be asked to track routine treatment, symptoms, changes in
treatment plans, and HRQoL using the Folia Health mobile app. Monthly survey check-ins
will be conducted to capture additional data inputs, with the possibility of integrating
electronic health record (EHR) and/or claims data.