The Longitudinal Relationship of HU Adherence to HRQOL, Barriers to Adherence and Habit in SCD.

Study Overview
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Brief Summary
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The primary objective of this study is to better understand factors contributing to variations in hydroxyurea (HU) adherence behavior in adolescents and young adults (AYA) with sickle cell disease (SCD). To meet this objective, the researchers will conduct a prospective cohort study to determine the longitudinal relationship between HU adherence and health-related quality of life (HRQOL) overtime among AYA with SCD. The long-term goal of this research is to promote medication adherence behavior and improve health outcomes in AYA with SCD.

Detailed Description
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Sickle cell disease is the most common genetic disorder in the US, affecting about 100,000 Americans, and about 1 in 400 African American live births, incurring annual health care costs of $335 million. SCD can lead to serious complications including unpredictable, debilitating pain episodes, cardiopulmonary disease, stroke, and long-term end organ damage.These complications lead to significant declines in health-related quality of life (HRQOL) and other patient-reported outcomes (PROs), culminating in early mortality, particularly among AYA. Hydroxyurea, at present, is the main FDA approved medication for SCD that reduces morbidity and mortality, improves HRQoL and lowers healthcare utilization.However, adherence to HU remains suboptimal with only 35-50% of patients achieving high adherence (≥90%), particularly among AYA with SCD. Low HU adherence has been associated with worse health outcomes, poor HRQOL and increased healthcare utilization. Low HU adherence is multifactorial, especially in AYA with other competing priorities and vulnerability in developmental and psychological factors contributing to adherence behavior. The specific aim for this study is to determine the longitudinal relationship of HU adherence behavior to health-related quality of life, barriers to adherence and habit formation among AYA with SCD.

Official Title
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The Longitudinal Relationship of Hydroxyurea Adherence Behavior to Health-related Quality of Life, Barriers to Adherence and Habit Formation in Patients With Sickle Cell Disease.

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: Age 12-21 years old Any sickle cell disease genotype On steady state of hydroxyurea for 2 months Own or have access to a smartphone Parents of patients that meet the eligibility criteria and are enrolled in the study will be included Exclusion Criteria: Patients with recent hospitalizations within the past 7 days

Ages Eligible for Study
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Minimum Age: 12 Years
Maximum Age: 21 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 the HU-Go app intervention arm for 12 months. | 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 |
| --- | --- | --- |
| Hydroxyurea adherence rate | Adherence rate is defined as number of given HU doses given, as captured by HU-Go app, divided by total number of doses expected during study period. | 12 months | 

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) | 12 months | 
| Adherence to Hydroxyurea using Visual Analogue Scale | Numerical value on a scale 0-100% (higher score indicating higher adherence to hydroxyurea) | 12 months | 
| Pain score using Patient reported outcomes measurement information system (PROMIS) measure | Patient reported outcomes measurement information system (PROMIS) measure for pain intensity, numerical value on a scale of 0-100 points, normal average for the general population is 50 with standard deviation of 10 points (higher T scores indicating worse pain). | 12 months | 
| Fatigue score using Patient reported outcomes measurement information system (PROMIS) measure | Patient reported outcomes measurement information system (PROMIS) measure for fatigue, numerical value on a scale of 0-100 points, normal average for the general population is 50 with standard deviation of 10 points (higher T scores indicating worse fatigue). | 12 months | 
| Physical function score using Patient reported outcomes measurement information system (PROMIS) measure | Patient reported outcomes measurement information system (PROMIS) measure for physical function, numerical value on a scale of 0-100 points, normal average for the general population is 50 with standard deviation of 10 points (lower T scores indicating worse physical function). | 12 months | 
| Depression score using Patient reported outcomes measurement information system (PROMIS) measure | Patient reported outcomes measurement information system (PROMIS) measure for depression, numerical value on a scale of 0-100 points, normal average for the general population is 50 with standard deviation of 10 points (higher T scores indicating worse depression). | 12 months | 
| Anxiety score using Patient reported outcomes measurement information system (PROMIS) measure | Patient reported outcomes measurement information system (PROMIS) measure for anxiety, numerical value on a scale of 0-100 points, normal average for the general population is 50 with standard deviation of 10 points (higher T scores indicating worse anxiety). | 12 months | 
| Peer relationships score using Patient reported outcomes measurement information system (PROMIS) measure | Patient reported outcomes measurement information system (PROMIS) measure for peer relationships, numerical value on a scale of 0-100 points, normal average for the general population is 50 with standard deviation of 10 points (lower T scores indicating worse peer relationships). | 12 months | 
| Codes and themes from qualitative data analysis | Semi-structured interviews focused on HU taking behavior, barriers to adherence and habit formation | 12 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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sickle cell disease, hydroxyurea