Home-based Assessment of PRO Measures in SCD Using A Smartphone App Platform: A Feasibility Study
Condition: Sickle Cell Disease; Sickle Cell Hemoglobin C; Sickle Beta Zero Thalassemia; Sickle B+ Thalassemia
MeSH terms: Anemia, Sickle Cell; Thalassemia; Hemoglobin SC Disease
Study type: Interventional

Summary: 
      The overarching goal of this proposal is to identify modifiable behavioral strategies based
      on patient-reported outcomes (PROs) and health-related quality of life (HRQOL) that will
      improve hydroxyurea (HU) adherence among adolescents and young adults with sickle cell
      disease (SCD). In this proposed study, we intend to test the functionality of a PROs-toolbox
      feature, which will be integrated into our existing smartphone application platform
      (SCD-app), over a 24-week period in a cohort of SCD patients and their caregivers.
    

Detailed description: 
      Adherence to hydroxyurea is a multi-factorial dynamic process, and, to date, predictors for
      changes in adherence levels over time remain unclear. Changes in HRQOL data could serve as
      surrogate markers for changes in hydroxyurea adherence over time, and could suggest when
      patients might or might not need or benefit from an intervention based on worse or better
      HRQOL scores, respectively.

      The main hypothesis of this study is that the assessment of patients' HRQOL at home using a
      SCD smartphone application platform (app) is feasible and acceptable, and that less frequent
      assessments of HRQOL at home will have an overall higher completion rate when compared to
      more frequent ones. We plan to assess HRQOL using the NIH-developed patient reported outcomes
      measurement information system (PROMIS®) with computerized adaptive testing (CAT) approach.
      PROMIS®-CAT approach is a reliable and valid PROs assessment platform that has been used in
      limited studies in SCD.

      The study will include the following specific aims:

        1. To evaluate the feasibility and acceptability of the assessment of patients HRQOL at
           home using smartphones with PROMIS®-CAT measures integrated into a SCD-app.

        2. To examine the effect of the frequency of required HRQOL assessments on participants'
           completion rate over 24-week period with HRQOL evaluated every 2 weeks (Group 1) versus
           every 4 weeks (Group 2).

        3. To explore participants' experience and preferences with the process and the frequency
           of HRQOL assessment at home using their smartphones with PROMIS®-CAT measures integrated
           into a SCD-app.
    

Intervention name(s): PROs assessment using sickle cell disease mobile app (SCD-app)
Intervention type(s): Behavioral
Intervention description(s): A smartphone app platform designed for patients with sickle cell disease. The SCD-app is programmed to send notification to patients to complete PROs assessment. The SCD-app is able to collect PROs data as patients complete the assessment using the app platform.
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Primary purpose: Supportive Care