Peer Reviewed Study

Study protocol for ADHERE (Applying Directly observed therapy to HydroxyurEa to Realize Effectiveness): Using small business partnerships to deliver a scalable and novel hydroxyurea adherence solution to youth with sickle cell disease

PLOS ONE
Joseph Walden, Lauren Brown, Sebastian Seiguer, Katie Munshaw, Joseph Rausch, Sherif Badawy, Patrick McGann, Savannah Winkler, Lisbel Gonzalez, Susan Creary

Written By Joseph Walden, Lauren Brown, Sebastian Seiguer, Katie Munshaw, Joseph Rausch, Sherif Badawy, Patrick McGann, Savannah Winkler, Lisbel Gonzalez, Susan Creary

Jun 25, 2024

Background: Sickle cell disease (SCD) is an inherited blood disorder that affects approximately 100,000 Americans, primarily from underrepresented racial minority populations, and results in costly, multi-organ complications. Hydroxyurea, the primary disease-modifying therapy for SCD, is effective at reducing most complications; however, adherence to hydroxyurea remains suboptimal and is the primary barrier to clinical effectiveness.

Objective: Given prior challenges and the need for a successful and scalable hydroxyurea adherence solution to improve outcomes, the primary aim of this study is to assess retention and sustained engagement during a pilot randomized controlled trial (RCT) comparing VDOT for hydroxyurea delivered by Scene Health to attention control among youth with SCD. The secondary aim of this study is to use a novel adherence monitoring device to measure hydroxyurea adherence and explore needs and preferences for longer-term adherence monitoring and intervention.

Methods: This study was initially approved by a single Institutional Review Board on 08/15/2023 (STUDY00003303) and is registered with the National Institutes of Health

(NCT06264700). All participants will provide consent/assent. Relevant data will be made available at the conclusion of this study upon request. This trial includes all items on the Standard Protocol Items: Recommendations for Interventional Trials Checklist.

Anticipated outcomes and future directions: This study's anticipated outcomes are that VDOT will be acceptable and result in sustained retention and engagement with a centrally delivered adherence intervention. These results will be used to inform the definitive efficacy RCT of VDOT for hydroxyurea.

Funding: This research is supported by funding awarded from the National Heart, Lung, and Blood Institute of the National Institutes of Health (1 R44 HL169085) (https://www.nhlbi.nih.gov/). The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.

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