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NCT Number: NCT04691323

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

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.

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Key information

Conditions

Sickle Cell Disease Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobin SC Disease Hemoglobinopathies Sickle B+ Thalassemia Sickle Beta Zero Thalassemia Sickle Cell Hemoglobin C

Age range

12 year–21 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Ann & Robert H Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

HU-Go app

Other

A novel multifunctional mobile app (HU-Go) to improve adherence to hydroxyurea in patients with sickle cell disease

Primary outcomes

  1. Hydroxyurea adherence rate

    Time frame: 12 months

    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.

Secondary outcomes

  1. Adherence to Hydroxyurea using PROMIS Medication Adherence Scale (PMAS)

    Time frame: 12 months

    Higher score indicating higher adherence to hydroxyurea

  2. Adherence to Hydroxyurea using Visual Analogue Scale

    Time frame: 12 months

    Numerical value on a scale 0-100% (higher score indicating higher adherence to hydroxyurea)

  3. Pain score using Patient reported outcomes measurement information system (PROMIS) measure

    Time frame: 12 months

    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).

  4. Fatigue score using Patient reported outcomes measurement information system (PROMIS) measure

    Time frame: 12 months

    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).

  5. Physical function score using Patient reported outcomes measurement information system (PROMIS) measure

    Time frame: 12 months

    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).

  6. Depression score using Patient reported outcomes measurement information system (PROMIS) measure

    Time frame: 12 months

    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).

  7. Anxiety score using Patient reported outcomes measurement information system (PROMIS) measure

    Time frame: 12 months

    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).

  8. Peer relationships score using Patient reported outcomes measurement information system (PROMIS) measure

    Time frame: 12 months

    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).

  9. Codes and themes from qualitative data analysis

    Time frame: 12 months

    Semi-structured interviews focused on HU taking behavior, barriers to adherence and habit formation

Sponsors and collaborators

Lead sponsor

Ann & Robert H Lurie Children's Hospital of Chicago

Other

Collaborators

  • National Heart, Lung, and Blood Institute (NHLBI)

Registry information

Official study 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.

Important dates

Study start
2021
Primary completion
2025
Study completion
2025
First posted
Dec 31, 2020
Registry last updated
Dec 24, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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