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

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

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.

Study Overview

• Status: Completed
• Conditions: Sickle Cell Disease; Sickle B+ Thalassemia; Sickle Beta Zero Thalassemia; Sickle Cell Hemoglobin C
• Intervention / Treatment: Other: HU-Go app

Detailed Description

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%.

• Study Type: Interventional
• Enrollment (Actual): 33
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Ann & Robert H Lurie Children's Hospital of Chicago

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 10 years to 23 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Supportive Care
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

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

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Ann & Robert H Lurie Children's Hospital of Chicago
• Investigators: Principal Investigator: Sherif M. Badawy, MD, MS, Ann & Robert H Lurie Children's Hospital of Chicago

Study record dates

Study Major Dates

• Study Start (Actual): May 15, 2018
• Primary Completion (Actual): July 11, 2020
• Study Completion (Actual): July 11, 2020

Study Registration Dates

• First Submitted: August 17, 2016
• First Submitted That Met QC Criteria: December 15, 2020
• First Posted (Actual): December 19, 2020

Study Record Updates

• Last Update Posted (Actual): July 29, 2021
• Last Update Submitted That Met QC Criteria: July 27, 2021
• Last Verified: July 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Anemia, Sickle Cell; Thalassemia; Hemoglobin SC Disease
• Other Study ID Numbers: IRB 2015-761

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No
• IPD Plan Description: All patients data will be deidentified.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No