Clinical trial NCT04691323: The Longitudinal Relationship of HU Adherence to HRQOL, Barriers to Adherence and Habit in SCD.
Official title: 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.
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: NA
Conditions: Sickle Cell Disease
Sickle B+ Thalassemia
Sickle Beta Zero Thalassemia
Sickle Cell Hemoglobin C
Keywords: sickle cell disease
hydroxyurea
Lead sponsor: Ann & Robert H Lurie Children's Hospital of Chicago
Sponsor class: OTHER
Collaborators: name: National Heart, Lung, and Blood Institute (NHLBI)
First posted: 2020-12-31
Enrollment: 68
Sex: ALL
Minimum age (years): 12
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: SUPPORTIVE_CARE

Brief summary: 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: 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.

Eligibility criteria: 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

Primary outcomes:
measure: Hydroxyurea adherence rate
timeFrame: 12 months
Secondary outcomes:
measure: Adherence to Hydroxyurea using PROMIS Medication Adherence Scale (PMAS)
timeFrame: 12 months
measure: Adherence to Hydroxyurea using Visual Analogue Scale
timeFrame: 12 months
measure: Pain score using Patient reported outcomes measurement information system (PROMIS) measure
timeFrame: 12 months
measure: Fatigue score using Patient reported outcomes measurement information system (PROMIS) measure
timeFrame: 12 months
measure: Physical function score using Patient reported outcomes measurement information system (PROMIS) measure
timeFrame: 12 months
measure: Depression score using Patient reported outcomes measurement information system (PROMIS) measure
timeFrame: 12 months
measure: Anxiety score using Patient reported outcomes measurement information system (PROMIS) measure
timeFrame: 12 months
measure: Peer relationships score using Patient reported outcomes measurement information system (PROMIS) measure
timeFrame: 12 months
measure: Codes and themes from qualitative data analysis
timeFrame: 12 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: