Clinical trial NCT04675645: Adherence to HU and HRQOL in Patients With Sickle Cell Disease: An Intervention Study Using HU-Go App
Official title: Adherence to Hydroxyurea and Health-related Quality of Life in Patients With Sickle Cell Disease: An Intervention Study Using a Smartphone App (HU-Go)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Sickle Cell Disease
Sickle B+ Thalassemia
Sickle Beta Zero Thalassemia
Sickle Cell Hemoglobin C
Lead sponsor: Ann & Robert H Lurie Children's Hospital of Chicago
Sponsor class: OTHER
First posted: 2020-12-19
Enrollment: 33
Sex: ALL
Minimum age (years): 12
Maximum age (years): 25
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: SUPPORTIVE_CARE

Brief summary: 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: 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%.

Eligibility criteria: 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

Primary outcomes:
measure: Achieving feasibility based on the number of participants completed all study procedures within 6 months of study enrollment
timeFrame: 3 months of study enrollment
Secondary outcomes:
measure: Adherence to Hydroxyurea using Modified Morisky Adherence Scale 8-items
timeFrame: 3 months of study enrollment
measure: Adherence to Hydroxyurea using Visual Analogue Scale
timeFrame: 3 months of study enrollment
measure: Patient satisfaction with the smartphone app intervention (HU-Go)
timeFrame: 3 months of study enrollment
measure: HRQOL outcomes
timeFrame: 3 months
Other outcomes:
Locations:
city: Chicago
country: United States
facility: Ann & Robert H Lurie Children's Hospital of Chicago
state: Illinois
Study officials:
affiliation: Ann & Robert H Lurie Children's Hospital of Chicago
name: Sherif M. Badawy, MD, MS
role: PRINCIPAL_INVESTIGATOR
References: