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

项目总结 虽然镰状细胞病(SCD)治疗的不断发展是一个可喜的进步，但普遍存在 研究到实践的差距限制了这些疗法的覆盖范围。尽管几十年来的证据支持 羟基尿素(HU)，一种改善红细胞功能和减少镰刀伤的药物，已经被 慢性阻塞性肺疾病治疗缓慢。只有40%-65%的患有SCD的年轻人接受了HU，一半的人表现出较差的依从性， 青少年和青壮年(AYA)的依从性最差。HU吸收的障碍是多方面的。 Ayas报告说，缺乏对决策的参与，知识不足，对疗效和 安全。由于担心患者的依从性，提供者可能不会提供HU。这些挑战是 再加上护理方面的社会文化障碍。这些障碍很可能同样会限制使用 最近批准的SCD疗法(L-谷氨酰胺、Voxelotor、Crizanlizumab)。循证干预措施包括 在这一高风险发展时期，迫切需要优化有效治疗的覆盖范围和吸收。 共享决策(SDM)干预提高了患者的知识、参与度、护理满意度 和坚持，对边缘化群体特别有益。然而，不存在这样的干预 阿亚斯患有SCD。为了弥补这一差距，我们将为AYA开发技术增强型SDM干预措施， 通过扩展和调整专为儿童父母设计的决策支持干预，SCD年龄在15-25岁之间 患有SCD的儿童，HU共享决策(H-SDM)工具包。该工具包通过以下方式针对多层障碍 为以下方面提供技术增强的工具：1)临床医生(激励性访谈培训，包括虚拟现实 模拟)；2)父母(多媒体决策辅助工具)；3)实施(审计和反馈策略)。它 增加知识，减少决策冲突，增加患者开HU的比例。 本研究将达到以下目的：1)扩展H-SDM工具包，使其包含对新的 SCD疗法；2)改编和改进用于患有SCD的AYAS的H-SDM工具包；以及3)评估可接受性， 调整后的工具包的可行性和初步效果。为了扩展H-SDM工具包，我们将进行在线 与全国SCD供应商进行众包，以评估影响采用最近批准的疗法的因素。 为了使H-SDM工具包适用于AYA，我们将对AYA及其照顾者和 有提供者的焦点小组。我们由不同利益相关者组成的团队将参加联合设计研讨会，以 开发经过调整的工具包的初始原型和实施蓝图，以将其集成到例程中 临床实践；将与AYA、照顾者和提供者一起进行可用性测试的迭代周期，以 建立工具包和实施计划的技术和功能可靠性和实用性。最后，我们会 进行单臂试点试验，以评估改编后的 工具包。这项研究将产生一项以证据为基础、技术增强的SDM干预措施，以增强REACH 以及接受疾病修正疗法，从而改善患有SCD的AYA的健康结果。

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.