Enhancing the Understanding of Phenotypic Outcomes in Sickle Cell Disease (SCD) Through a Comparison Between Data Collected Through App-Based Home-Reported Outcomes (HROs) Versus Qualitative Methodologies

Author(s)

Zhang C1, Healey A2, Anwar H2
1Folia Health, Kansas City, MO, USA, 2Folia Health, Boston, MA, USA

OBJECTIVES: To compare a novel app-based data capture framework with qualitative data collection in the sickle cell disease (SCD) population.

METHODS: A literature search identified unique concepts and themes in qualitative data previously collected in SCD. These concepts were compared against an outcome inventory created from a group of SCD participants using the app-based Folia home-reported outcomes (HROs) platform. The outcome inventory was further categorized using patient-led concept grouping and mapped to relevant domains based on clinical judgment and subject matter expertise.

RESULTS: Qualitative data collection methods, such as interviews, diaries, and surveys using patient-reported outcome (PRO) measures, provide insight into the subjective burden of SCD. In contrast, direct app-based tracking allows participants to identify and track their experiences flexibly and at an individual level. Aggregated over a period of time, this app-based data collection method creates an outcome inventory - a granular dataset of symptomatology for a specific condition-of-interest.

A comparative analysis between the outcome inventory captured in Folia and themes from qualitative SCD data will be presented. Semi-structured interviews, patient-reported diaries, and PRO surveys produced a variety of concepts ranging from 8 domains to 30 domains.The Folia outcome inventory applied to tracking data of 82 SCD participants resulted in 92 unique symptoms across 8 categories. Compared to qualitative data capture, the outcome inventory revealed over 20 different manifestations of pain, and 12 types of behavioral/mood symptoms. This outcome inventory methodology captured similar concepts to semi-structured interviews but with higher volume and greater detail, including real-time symptom experiences and healthcare-seeking behavior.

CONCLUSIONS: In designing a study to understand the nuances of SCD, individualized and participant-driven research methods - such as utilizing an app-based outcome inventory - may enhance existing knowledge. The outcome inventory methodology provides a detailed understanding of SCD burden, contributing to a comprehensive assessment of the disease.