Empowering adolescents and young adults with sickle cell disease as partners in treatment decision making (EMPOWER-AYA)

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PROJECT SUMMARY While the evolving landscape of sickle cell disease (SCD) treatments is a welcome advancement, pervasive research-to-practice gaps limit the reach of these therapies. Despite decades of evidence supporting hydroxyurea (HU), a medication that improves red blood cell function and decreases sickling, uptake has been slow in SCD care. Only 40-65% of youth with SCD receive HU and half show poor adherence, with adolescents and young adults (AYAs) exhibiting the worst adherence. Barriers to HU uptake are multifactorial. AYAs report lack of involvement in decision making, inadequate knowledge, and doubts about efficacy and safety. Providers may not offer HU due to concerns about patient adherence. These challenges are compounded by sociocultural obstacles to care. It is likely that these barriers will similarly restrict use of recently-approved SCD therapies (L-glutamine, voxelotor, crizanlizumab). Evidence-based interventions are urgently needed to optimize reach and uptake of effective therapies during this high-risk developmental period. Shared decision making (SDM) interventions enhance patient knowledge, engagement, satisfaction with care, and adherence and are particularly beneficial for marginalized groups. However, no such interventions exist for AYAs with SCD. To address this gap, we will develop a technology-enhanced SDM intervention for AYAs with SCD ages 15-25 years by expanding and adapting a decision support intervention designed for parents of children with SCD, the HU Shared Decision Making (H-SDM) Toolkit. This toolkit targets multi-level barriers by providing technology-enhanced tools for: 1) clinicians (motivational interviewing training, including virtual reality simulation); 2) parents (multimedia decision aids); and 3) implementation (audit and feedback strategies). It enhances knowledge, reduces decisional conflict, and increases the proportion of patients prescribed HU. This study will achieve the following aims: 1) Expand the H-SDM Toolkit to include decision supports for new SCD therapies; 2) Adapt and refine the H-SDM Toolkit for AYAs with SCD; and, 3) Evaluate acceptability, feasibility, and preliminary efficacy of the adapted toolkit. To expand the H-SDM Toolkit, we will conduct online crowdsourcing with SCD providers nationally to assess factors influencing uptake of recently-approved therapies. To adapt the H-SDM Toolkit for AYAs, we will conduct qualitative interviews with AYAs and their caregivers and focus groups with providers. Our team of diverse stakeholders will then participate in co-design workshops to develop an initial prototype of the adapted toolkit and an implementation blueprint to integrate it into routine clinical practice; iterative cycles of usability testing will be conducted with AYAs, caregivers, and providers to establish technical and functional reliability and utility of the toolkit and implementation plan. Finally, we will conduct a single-arm pilot trial to evaluate acceptability, feasibility, and preliminary efficacy of the adapted toolkit. This study will produce an evidence-based, technology-enhanced SDM intervention to enhance reach and uptake of disease-modifying therapies and thereby improve health outcomes among AYAs with SCD.