Presentation (CTI)

OBJECTIVES: Sickle cell disease (SCD) and cystic fibrosis (CF) are both complex, rare hereditary conditions. This research aims to compare self-reported tracking of symptom severity and treatment usage as proxies for real-world disease burden and treatment impact in SCD and CF, using data collected by users of the Folia Health mobile app who have these conditions.
METHODS: Three months of data were analyzed from 62 individuals with CF and 31 individuals with SCD who enrolled and consented from 2019 to 2023. The number and types of treatments used, frequency of symptom tracking, including instances of pain manifestations and highest recorded symptom severity, were analyzed in each condition. Additionally, a comparison of coughing severity (on a scale of 0-10) was conducted for pre- vs post- adoption of elexacaftor/tezacaftor/ivacaftor, for 17 CF participants.
RESULTS: SCD users tracked 42 unique treatments and 65 unique symptoms, of which 20% were marked as severe at least once. SCD users reported an average of 38 pain flares during this period. Over the same period, CF users tracked 158 unique treatments and 50 unique symptoms, of which 20% were marked as severe at least once, and reported an average of 26 tracks (days) with cough present during this period. Additionally, after initiating elexacaftor/tezacaftor/ivacaftor, CF users reported a significant (p < 0.05) reduction in average cough severity of 28%.
CONCLUSIONS: Findings confirmed that both CF and SCD are complex conditions involving a high number of potential symptoms and treatments-in-use. Findings also indicate that Folia app users with CF experienced a notable decrease in coughing severity after starting on elexacaftor/tezacaftor/ivacaftor. This result highlights the urgent need for equitable investment in SCD research and the development of effective, targeted treatments to address this disparity and improve outcomes for patients living with similarly complex and rare conditions.