Presentation (CTI)

OBJECTIVES: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematological condition involving recurring symptom flares. Historically, data on flares in PNH have been captured via point-in-time patient-reported outcome measures, which may not capture full information about flare incidence/characteristics due to recall bias. This analysis compared Folia Health (FH) app patient reported, real-time data on flares with data reported retrospectively at discrete timepoints.
METHODS: This abstract reports findings from 37 FH users with PNH over a 5 month period who recorded real-time information on flares. FH users tracked real-time detail on flare incidence, flare triggers, and effects on daily life. Additionally, the instances of flares were quantitatively assessed retrospectively once per month.
RESULTS: Retrospective responses about flares were compared to data obtained via ongoing tracking of flares. Sensitivity and specificity of the retrospective responses collapsed across months 1-5 (as compared to the real-time tracking approach) were found to be 93% and 46%, respectively. Real-time flare tracking captured additional data about the flare, including: the average (SD) flare duration was 2 (3.94) days, that the top known flare triggers were “Daily routine change” and “Stress”, and that the top reported effects of flares on daily life were “Feeling frustrated” and “Having trouble starting tasks”.
CONCLUSIONS: Findings illustrate the value of capturing real-time patient-reported information on flares in PNH, which allows for capture of detailed data on flare characteristics. These data may enable future analyses of treatment impact on core aspects of disease burden that are not captured by point-in-time surveys. Interestingly, real-time reporting and once-per-month reporting that relies on recall yield divergent data on flare incidence; future research should examine reasons for differences in these two measurement approaches.