Clinical trial NCT03462511: Hydroxyurea Adherence for Personal Best in Sickle Cell Disease (HABIT): Efficacy Trial
Official title: Hydroxyurea Adherence for Personal Best in Sickle Cell Disease (HABIT): Efficacy Trial
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Sickle Cell Disease
Keywords: Hydroxyurea
Community health worker (CHW)
Text messages
Medication adherence
Lead sponsor: Columbia University
Sponsor class: OTHER
Collaborators: name: National Institute of Nursing Research (NINR)
First posted: 2018-03-12
Enrollment: 50
Sex: ALL
Minimum age (years): 10
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: HEALTH_SERVICES_RESEARCH

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.

Detailed description: Barriers to medication adherence are common in youth with chronic illness and are a source of racial/ethnic disparities in underserved communities. An inherited blood disease, Sickle Cell Disease (SCD) is characterized by chronic and acute illness and reduced quality of life (QOL). It affects African Americans and other underserved communities. Hydroxyurea (HU) is the sole FDA-approved drug therapy for SCD and is highly effective and improves QOL. Poor adherence is common among youth and young adults with SCD.

The importance of poor medication adherence, use of community-based health workers (CHWs) to bridge the gap between health services and underserved parent-youth dyads affected by SCD, the strength of the science, the success of the investigators' multi-ethnic feasibility study, and the potential application of study findings to youth with other serious chronic illnesses speak to the importance of this trial.

Eligibility criteria: Inclusion Criteria - Youth:

* 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

Inclusion Criteria - Parent:

* Parent/guardian speaks/reads English or Spanish
* Parent/ legal guardian willing to participate
* Family expects to reside in community for ≥ 1.5 years

Exclusion Criteria - Youth:

* 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

Exclusion Criteria - Parent:

* Parent/legal guardian does not reside with youth

Primary outcomes:
measure: Mean Change in Biomarker Fetal Hemoglobin (HbF)
timeFrame: Baseline, 6 months, 12 months
measure: Mean Change in Proportion of Days Covered (PDC) by Hydroxyurea
timeFrame: Baseline, 6 months, 12 months
Secondary outcomes:
measure: Mean Change in Youth Score on Peds Quality of Life (Generic Quality of Life)
timeFrame: Baseline, 4 months, 9 months and 12 months
measure: Mean Change in Youth Score on PedsQL Sickle Cell Disease Module (Disease Specific Quality of Life)
timeFrame: Baseline, 9 months and 12 months
measure: Mean Change in Parent Youth Concordance Regarding Self-management Responsibility
timeFrame: Baseline, 6 months and 12 months
Other outcomes:
Locations:
city: Bronx
country: United States
facility: Albert Einstein College of Medicine
state: New York
city: Manhasset
country: United States
facility: Feinstein Institute for Medical Research
state: New York
city: New York
country: United States
facility: Columbia University Irving Medical Center
state: New York
city: Philadelphia
country: United States
facility: The Children's Hospital of Philadelphia
state: Pennsylvania
Study officials:
affiliation: Columbia University School of Nursing
name: Arlene Smaldone, PhD, CPNP-PC
role: PRINCIPAL_INVESTIGATOR
affiliation: Columbia University
name: Nancy S Green, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.
citation: 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.
citation: 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.
citation: 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.
citation: 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.