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

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ClinicalTrials.gov Identifier: NCT04691323
Recruitment Status : Recruiting
First Posted : December 31, 2020
Last Update Posted : July 27, 2022
Sponsor:
Collaborator:
National Heart, Lung, and Blood Institute (NHLBI)
Information provided by (Responsible Party):
Sherif Badawy, MD, Ann & Robert H Lurie Children's Hospital of Chicago

Study Description
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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.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Sickle B+ Thalassemia Sickle Beta Zero Thalassemia Sickle Cell Hemoglobin C Other: HU-Go app Not Applicable

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.
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 72 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Supportive Care
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.
Actual Study Start Date : March 2, 2021
Estimated Primary Completion Date : November 2023
Estimated Study Completion Date : February 2025

Resource links provided by the National Library of Medicine

Drug Information available for: Hydroxyurea

Arms and Interventions
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Arm Intervention/treatment
HU-Go app intervention arm
Participants will use the HU-Go app intervention arm for 12 months.
 Other: HU-Go app
A novel multifunctional mobile app (HU-Go) to improve adherence to hydroxyurea in patients with sickle cell disease


Outcome Measures
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Primary Outcome Measures :
  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 Outcome Measures :
  1. Adherence to Hydroxyurea using Modified Morisky Adherence Scale 8-items [ Time Frame: 12 months ]
    Numerical value on a scale 0-8 (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


Eligibility Criteria
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Ages Eligible for Study:   12 Years to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04691323


Contacts
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Contact: Sherif M. Badawy, MD, MS (312) 227-4836 sbadawy@luriechildrens.org
Contact: Kathryn King, BS (312) 227-4825 katking@luriechildrens.org

Locations
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United States, Illinois
Ann & Robert H Lurie Children's Hospital of Chicago Recruiting
Chicago, Illinois, United States, 60611
Contact: Sherif M. Badawy, MD, MS    312-227-4836    sbadawy@luriechildrens.org   
Contact: Kathryn King, BS    (312) 227-4825    katking@luriechildrens.org   
Principal Investigator: Sherif M. Badawy, MD, MS         
Sponsors and Collaborators
Ann & Robert H Lurie Children's Hospital of Chicago
National Heart, Lung, and Blood Institute (NHLBI)
Investigators
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Principal Investigator: Sherif M. Badawy, MD, MS Ann & Robert H Lurie Children's Hospital of Chicago
More Information
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Responsible Party: Sherif Badawy, MD, Assistant Professor, Department of Pediatrics, Division of Hematology, Oncology and Stem Cell Transplant, Ann & Robert H Lurie Children's Hospital of Chicago
ClinicalTrials.gov Identifier: NCT04691323    
Other Study ID Numbers: IRB 2020-3366
1K23HL150232 ( U.S. NIH Grant/Contract )
First Posted: December 31, 2020    Key Record Dates
Last Update Posted: July 27, 2022
Last Verified: July 2022

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Sherif Badawy, MD, Ann & Robert H Lurie Children's Hospital of Chicago:
sickle cell disease
hydroxyurea
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Thalassemia
Hemoglobin SC Disease
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
 Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn