Hydroxyurea Adherence for Personal Best in Sickle Cell Disease (HABIT): Efficacy Trial
Trial Snapshot
- Phase
- Not Applicable
- Status
- Completed
- Sponsor
- Columbia University
- Enrollment
- 50
- Locations
- 4
- Primary Endpoint
- Mean Change in Biomarker Fetal Hemoglobin (HbF)
Study Overview
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.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Health Services Research
- Masking
- Single (Outcomes Assessor)
Eligibility Criteria
- Ages
- 10 Years to 18 Years (Child, Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •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 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
Arms & Interventions
Control Group
Dyads randomized to the control group will receive:
Standard care and Education handouts.
Intervention: Standard of Care (Other)
Control Group
Dyads randomized to the control group will receive:
Standard care and Education handouts.
Intervention: Education materials (Other)
Intervention Group
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.
Intervention: HABIT Intervention (Behavioral)
Intervention Group
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.
Intervention: Standard of Care (Other)
Intervention Group
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.
Intervention: Education materials (Other)
Outcomes
Primary Outcomes
Mean Change in Biomarker Fetal Hemoglobin (HbF)
Time Frame: Baseline, 6 months, 12 months
A serum biomarker obtained from youth used to measure adherence to hydroxyurea
Mean Change in Proportion of Days Covered (PDC) by Hydroxyurea
Time Frame: Baseline, 6 months, 12 months
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.
Secondary Outcomes
- Mean Change in Parent Youth Concordance Regarding Self-management Responsibility(Baseline, 6 months and 12 months)
- 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)
Investigators
Arlene Smaldone
Professor of Nursing
Columbia University