SBIR-STTR Award

Direct to Phase II

Sickle cell disease (SCD) is a severe inherited blood disorder that leads to frequent acute and costly long-term complications. Hydroxyurea is the primary SCD modifying medication in childhood because studies show that it can reduce acute complications and mitigate costly long-term comorbidities. Hydroxyurea non-adherence is common, however, and a key reason for the limited impact that it has had on the outcomes of youth in clinical practice. Despite the impact that improving hydroxyurea adherence among children and adolescents could have on reducing SCD burden, studies testing adherence-promoting interventions for youth are lacking. Video directly observed therapy (VDOT) is an electronic hydroxyurea adherence-promoting strategy delivered using smartphones and includes reminder alerts, video directly observed therapy by trained observers, encouraging adherence feedback from the observers, and monetary incentives for achieving adherence goals. VDOT has been tested in a single-arm study and showed promising results. Yet, attrition was higher than anticipated due to some dissatisfaction with the previous email platform that was used to deliver VDOT and many participants having inconsistent personal smartphone and cellular data access. Engagement with VDOT was not thoroughly assessed, even though the degree of engagement with VDOT has potential to moderate the impact of the intervention. Aspects of the previous study, including the method used to measure adherence and the non-randomized design also limited the ability to definitively conclude that VDOT improves adherence. Finally, the adherence decline that was observed after VDOT was discontinued without any additional adherence support suggested that additional monitoring and intervention after intensive VDOT may be needed to promote lasting adherence behavior change. This feedback and experience prompted us to partner with emocha Health. This is a small business that has a user-friendly VDOT platform, experience successfully providing VDOT to other chronically ill populations, and an established system to provide intermittent ongoing adherence to support after VDOT to limit attrition, optimize participant engagement, and sustain adherence. Therefore, the primary objective of this proposal is to assess retention and sustained engagement during a pilot RCT comparing VDOT via the emocha Health platform to attention control. We will also explore adolescents' and caregivers' needs and preferences for longer-term adherence monitoring and intervention. This application aligns with NHLBI's goal to optimize therapeutic strategies for SCD as it improving adherence is critically relevant to support life-long healthy behaviors and improve outcomes of these youth.

Public Health Relevance Statement:
Narrative/

Public Health Relevance Statement:
This research is highly relevant to public health as youth with sickle cell disease (SCD) are underserved and remain at high risk for adverse acute and long-term health outcomes. Hydroxyurea is the primary disease-modifying medication for these youth, but hydroxyurea non-adherence is common and a key reason for its limited impact in clinical practice. Relevant to the NHLBI's goal to optimize therapeutic strategies for SCD, this proposal will answer the critical questions to conduct a successful efficacy trial of video directly observed therapy, a promising adherence-promoting intervention, to improve hydroxyurea adherence and outcomes among youth with SCD. Terms: <12-20 years old; adolescence (12-20); Adolescence; Affect; Hb SS disease; HbSS disease; Hemoglobin S Disease; Hemoglobin sickle cell disease; Hemoglobin sickle cell disorder; sickle cell disease; sickle cell disorder; sickle disease; sicklemia; Sickle Cell Anemia; Behavior; Child; 0-11 years old; Child Youth; Children (0-21); kids; youngster; Chronically Ill; Clinical Trials; comorbidity; co-morbid; co-morbidity; Disease; Disorder; Pharmaceutical Preparations; Drugs; Medication; Pharmaceutic Preparations; drug/agent; Electronic Mail; E-Mail; Email; electronic communication; Electronics; electronic; electronic device; Feedback; Goals; Health; Hematological Disease; Blood Diseases; Hematologic Diseases; Hematological Disorder; blood disorder; Hospitalization; Hospital Admission; hydroxyurea; Hydroxycarbamid; Hydroxycarbamide; hydroxy-urea; Intervention Studies; intervention research; interventional research; interventional study; interventions research; Life Cycle Stages; Life Cycle; life course; Methods; Morbidity - disease rate; Morbidity; Patients; Public Health; Records; Research; Research Personnel; Investigators; Researchers; Telephone; Phone; Testing; Translating; Measures; Care Givers; Caregivers; Health Costs; Healthcare Costs; Health Care Costs; Businesses; Youth 10-21; Youth; Blinded; Caring; improved; Procedures; Site; Acute; Chronic; Adolescent Youth; juvenile; juvenile human; Adolescent; Training; pediatric; Childhood; adult youth; young adulthood; young adult; Funding; randomized control trial; Randomized, Controlled Trials; Therapeutic; Attenuated; attenuate; attenuates; Inherited; Hereditary; System; behavior change; preference; American; experience; Participant; Admission activity; Admission; Directly Observed Therapy; Intervention; Intervention Strategies; interventional strategy; Cell Phone; Cellular Telephone; Mobile Phones; iPhone; smart phone; smartphone; Cellular Phone; Effectiveness; Adherence; Data; Qualifying; Randomized; randomisation; randomization; randomly assigned; Patient-Focused Outcomes; Patient outcome; Patient-Centered Outcomes; patient oriented outcomes; Monitor; National Heart, Lung, and Blood Institute; NHLBI; cost; burden of disease; disease burden; years of life lost to disability; years of life lost to disease; burden of illness; designing; design; efficacy trial; Outcome; Population; aged; user-friendly; trial comparing; high risk; public health relevance; effective treatment; effective therapy; clinical practice; financial reward; monetary incentive; financial incentive; arm; m-Health; mobile health; mHealth; ED visit; ER visit; Emergency care visit; Emergency hospital visit; Emergency room visit; Emergency department visit; behavior outcome; behavioral outcome; attentional control; improved outcome; data access; recruit; care costs; participant engagement; patient engagement; behavior adherence; behavioral adherence; video delivery; video delivered; electronic adherence monitoring