Hydroxyurea Adherence for Personal Best in Sickle Cell Disease (HABIT): Efficacy Trial

NCT03462511 | Completed Results DMC

• 2 other identifiers: AAAR29081R01NR017206-01
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 4

Brief Summary

Many youth with chronic disease have difficulty taking medication every day and therefore do not receive full benefit from treatment. Sickle Cell Disease (SCD) is an inherited blood disease that affects African Americans and other underserved communities. Hydroxyurea (HU) is the sole FDA-approved drug therapy for SCD and is highly effective and improves quality of life. The proposed study, a 5-site four-year randomized control trial (RCT), builds upon the investigators' recent feasibility study of the same title. Overall goals are reducing barriers to HU use and improving adherence for youth 10-18 years through creation of a daily medication habit. The goal of the proposed multi-site study is to test the efficacy of the HABIT intervention at 6 months and sustainability of the effect at 12 months.

Conditions

Sickle Cell Disease

Keywords

Hydroxyurea; Community health worker (CHW); Text messages; Medication adherence

Outcome Measures

Primary Outcomes (2)

• Mean Change in Biomarker Fetal Hemoglobin (HbF)

  A serum biomarker obtained from youth used to measure adherence to hydroxyurea

  Baseline, 6 months, 12 months

• Mean Change in Proportion of Days Covered (PDC) by Hydroxyurea

  The days covered by hydroxyurea was assessed using youth prescription refill data and was used to measure hydroxyurea adherence. The baseline measure is the proportion of days covered by hydroxyurea in the year prior to study enrollment, using prescription refill data.

  Baseline, 6 months, 12 months

Secondary Outcomes (3)

• Mean Change in Youth Score on Peds Quality of Life (Generic Quality of Life)

  Baseline, 4 months, 9 months and 12 months

• Mean Change in Youth Score on PedsQL Sickle Cell Disease Module (Disease Specific Quality of Life)

  Baseline, 9 months and 12 months

• Mean Change in Parent Youth Concordance Regarding Self-management Responsibility

  Baseline, 6 months and 12 months

Study Arms (2)

Control Group

ACTIVE COMPARATOR

Dyads randomized to the control group will receive: Standard care and Education handouts.

Other: Standard of Care; Other: Education materials

Intervention Group

EXPERIMENTAL

In addition to standard care and education handouts, dyads randomized to the intervention group will receive the HABIT intervention, which includes CHW support and tailored text messages.

Behavioral: HABIT Intervention; Other: Standard of Care; Other: Education materials

Interventions

HABIT Intervention BEHAVIORAL

Dyads randomized to the intervention group will receive the HABIT intervention, which includes CHW support and tailored text messages.

Intervention Group

Standard of Care OTHER

Standard of care used to treat patients with SCD

Control Group; Intervention Group

Education materials OTHER

Education materials provided to all patients enrolled in the trial

Control Group; Intervention Group

Eligibility Criteria

• Age: 10 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• One of the two most common sickle cell disease variants (HbSS or HbS-B0 thalassemia)
• Age 10 through18 years (inclusive)
• Currently prescribed hydroxyurea (HU) ≥18 months (for identifying historical Personal best HbF)
• Current HU dose is within 5% of dose at Personal Best HbF
• Pre-enrollment HbF ≥15% below historical Personal best, based on mean of ≥2 HbF assessments over preceding 12 months
• Youth able to speak/read English or Spanish
• Parent/guardian speaks/reads English or Spanish
• Parent/ legal guardian willing to participate
• Family expects to reside in community for ≥ 1.5 years

You may not qualify if:

• Youth not prescribed HU
• \<2 HbF assessments over past 12 months
• Transfusion within 3 months preceding enrollment
• Final screen HbF (visit 0) of ≤15% decrease below Personal best HbF
• Sexually active female 10 years or older and not using reliable contraception (due to HU teratogenic risk)
• Pregnancy
• Cognitive impairment (\>2 levels below expected grade)
• Youth not residing with parent/legal guardian
• Parent/legal guardian does not reside with youth

Sponsors & Collaborators

• Columbia University: lead
• National Institute of Nursing Research (NINR): collaborator

Study Sites (4)

Feinstein Institute for Medical Research

Manhasset, New York, 11030, United States

Location

Columbia University Irving Medical Center

New York, New York, 10032, United States

Location

Albert Einstein College of Medicine

The Bronx, New York, 10461, United States

Location

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Related Publications (6)

• Bruzzese JM, Usseglio J, Iannacci-Manasia L, Diggs KA, Smaldone AM, Green NS. Mental and Emotional Health of Caregivers of Youth with Sickle Cell Disease: A Systematic Review. J Health Care Poor Underserved. 2023;34(3):1070-1104.

  PMID: 38015138 BACKGROUND

• Askew MA, Smaldone AM, Gold MA, Smith-Whitley K, Strouse JJ, Jin Z, Green NS. Pediatric hematology providers' contraceptive practices for female adolescents and young adults with sickle cell disease: A national survey. Pediatr Blood Cancer. 2022 Oct;69(10):e29877. doi: 10.1002/pbc.29877. Epub 2022 Jul 20.

  PMID: 35856776 BACKGROUND

• Smaldone A, Manwani D, Aygun B, Smith-Whitley K, Jia H, Bruzzese JM, Findley S, Massei J, Green NS. HABIT efficacy and sustainability trial, a multi-center randomized controlled trial to improve hydroxyurea adherence in youth with sickle cell disease: a study protocol. BMC Pediatr. 2019 Oct 15;19(1):354. doi: 10.1186/s12887-019-1746-6.

  PMID: 31615480 BACKGROUND

• Green NS, Manwani D, Aygun B, Appiah-Kubi A, Smith-Whitley K, Castillo Y, Soriano L, Jia H, Smaldone AM. Hydroxyurea Adherence for Personal Best in Sickle Cell Treatment (HABIT) efficacy trial: Community health worker support may increase hydroxyurea adherence of youth with sickle cell disease. Pediatr Blood Cancer. 2024 Apr;71(4):e30878. doi: 10.1002/pbc.30878. Epub 2024 Feb 6.

  PMID: 38321562 RESULT

• Green NS, Manwani D, Smith-Whitley K, Aygun B, Appiah-Kubi A, Smaldone AM. Mental health assessment of youth with sickle cell disease and their primary caregivers during the COVID-19 pandemic. Pediatr Blood Cancer. 2022 Sep;69(9):e29797. doi: 10.1002/pbc.29797. Epub 2022 May 25.

  PMID: 35614571 RESULT

• Lalji R, Koh L, Francis A, Khalid R, Guha C, Johnson DW, Wong G. Patient navigator programmes for children and adolescents with chronic diseases. Cochrane Database Syst Rev. 2024 Oct 9;10(10):CD014688. doi: 10.1002/14651858.CD014688.pub2.

  PMID: 39382077 DERIVED

MeSH Terms

Conditions

Anemia, Sickle Cell; Medication Adherence

Interventions

Standard of Care

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Patient Compliance; Patient Acceptance of Health Care; Treatment Adherence and Compliance; Health Behavior; Behavior

Intervention Hierarchy (Ancestors)

Quality Indicators, Health Care; Quality of Health Care; Health Services Administration; Health Care Quality, Access, and Evaluation

Results Point of Contact

• Title: Arlene Smaldone, PhD, RN
• Organization: Columbia University School of Nursing

ams130@columbia.edu

212-342-3048

Study Officials

• Arlene Smaldone, PhD, CPNP-PC

  Columbia University School of Nursing

  PRINCIPAL INVESTIGATOR

• Nancy S Green, MD

  Columbia University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: HEALTH SERVICES RESEARCH
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Nursing

Study Record Dates

• First Submitted: March 6, 2018
• First Posted: March 12, 2018
• Study Start: August 15, 2018
• Primary Completion: December 31, 2021
• Study Completion: December 31, 2021
• Last Updated: June 26, 2024
• Results First Posted: June 26, 2024

Record last verified: 2024-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (4)