Adherence to HU and HRQOL in Patients With Sickle Cell Disease: An Intervention Study Using HU-Go App

Study Overview
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Brief Summary
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This project addresses three important research questions. First, adolescents and young adults (AYA) with sickle cell disease (SCD) and their parents/caregivers will be engaged to inform the (1) domains of health-related quality of life (HRQOL) most important to them, (2) frequency at which they are willing to complete them, and (3) other procedures related to the use, uptake and effect of the HU-Go app as a tool to improve hydroxyurea (HU) adherence. Second, this study seeks to utilize novel modern mobile technology using a multi-functional personalized platform to improve adherence to HU and measure HRQOL in youth with SCD, using NIH-endorsed PROMIS® measures, based on a conceptual model with predefined behavioral targets and mediators. Third, we plan to assess HRQOL changes and identify modifiable behavioral strategies that could serve as surrogates or predictors for HU adherence. This real-time feedback might empower self-directed changes in behavior that could improve adherence to HU.

Detailed Description
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Aim 1: Identify the needs, desires, concerns, and expectations of AYA patients with SCD and their parents/caregivers that will guide implementation of a mHealth tool, HU-Go, designed to improve adherence to HU. We will conduct semi-structured interviews with patients and their parents/caregivers. Transcripts will be independently coded and thematic analysis will be conducted. Hypothesis 1: AYA patients with SCD and their parents/caregivers will identify specific features, content, and usability requirements of HU-Go. Aim 2: Assess the longitudinal relationship of HU adherence to HRQoL domains, including fatigue and depression. HU adherence will be measured using electronic pill bottles, self-report measures, laboratory markers, and medication possession ratio (MPR). HRQOL will be assessed using PROMIS® measures. Hypothesis 2: Low HU adherence is associated with impairment of HRQoL domains. Aim 3: Conduct a single-arm, pilot study to determine the effect size associated with improvement in HU adherence after using HU-Go for 12 weeks. Adherence will be measured using self-report measures, laboratory markers and MPR. Hypothesis 3: HU-Go will improve HU adherence by at least 20%.

Official Title
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Adherence to Hydroxyurea and Health-related Quality of Life in Patients With Sickle Cell Disease: An Intervention Study Using a Smartphone App (HU-Go)

Conditions
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Sickle Cell Disease, Sickle B+ Thalassemia, Sickle Beta Zero Thalassemia, Sickle Cell Hemoglobin C

Intervention / Treatment
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* Other: HU-Go app


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: At least 12 years old at the time of study enrollment Diagnosis of sickle cell disease (hemoglobin SS or SC or S/B 0 thalassemia) confirmed by hemoglobin electrophoresis On hydroxyurea Own or have access to a smartphone Be able to speak and read English Exclusion Criteria: Chronic monthly transfusion support Any hemoglobinopathy other than sickle cell disease

Ages Eligible for Study
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Minimum Age: 12 Years
Maximum Age: 25 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Supportive Care
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: HU-Go app intervention arm<br>Participants will use HU-Go app intervention arm for a total of 12 weeks. | Other: HU-Go app<br>* A novel multifunctional mobile app (HU-Go) to improve adherence to hydroxyurea in patients with sickle cell disease<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Achieving feasibility based on the number of participants completed all study procedures within 6 months of study enrollment | Feasibility is defined as having 80% or more of study participants complete the study within 6 months of enrollment | 3 months of study enrollment | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Adherence to Hydroxyurea using Modified Morisky Adherence Scale 8-items | Numerical value on a scale 0-8 (higher score indicating higher adherence to hydroxyurea) | 3 months of study enrollment | 
| Adherence to Hydroxyurea using Visual Analogue Scale | Numerical value on a scale 0-100% (higher score indicating higher adherence to hydroxyurea) | 3 months of study enrollment | 
| Patient satisfaction with the smartphone app intervention (HU-Go) | Customized patient satisfaction questionnaire, numerical value on a scale 0-10 (higher score indicating higher satisfaction with the app) | 3 months of study enrollment | 
| HRQOL outcomes | Patient reported outcomes measurement information system (PROMIS) measures, numerical value on a scale of 0-100 points, normal average for the general population is 50 with standard deviation of 10 points. Each HRQOL outcome domain score will be reported separately (higher T scores indicating worse pain, fatigue, depression and anxiety, and lower scores indicating worse physical functioning and peer relationships). | 3 months |