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

使患有镰状细胞病的青少年和年轻人成为治疗决策的合作伙伴 (EMPOWER-AYA)

• 批准号: 10271044
• 负责人: Aimee K Hildenbrand
• 金额: $ 10.67万
• 依托单位: ALFRED I. DU PONT HOSP FOR CHILDREN
• 依托单位国家: 美国
• 财政年份: 2014
• 资助国家: 美国
• 起止时间: 2014-08-01 至 2024-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10271044
• 关键词: Address; Adherence; Adolescent; Adolescent and Young Adult; Adult; Age; American Society of Hematology; Caregivers; Caring; Cell physiology; Child; Clinic; Complex; Conflict (Psychology); Data; Decision Aid; Decision Making; Delaware; Development; Disease; Educational workshop; Enhancement Technology; Erythrocytes; Evidence based intervention; Exhibits; FDA approved; Feedback; Focus Groups; Foundations; Funding; Glutamine; Goals; Gold; Growth; Health; Health Personnel; Intervention; Interview; Knowledge; Link; Methods; Modification; Multimedia; Outcome; Parents; Patient Care; Patients; Pharmaceutical Preparations; Population; Positioning Attribute; Provider; Quality of life; Reporting; Research; Safety; Secure; Sickle Cell; Sickle Cell Anemia; Testing; Training; Triad Acrylic Resin; Uncertainty; United States National Institutes of Health; Wood material; Youth; adolescent health outcomes; arm; barrier to care; clinical practice; compliance behavior; crowdsourcing; design; effective therapy; empowered; evidence base; experience; health care service utilization; high risk; hydroxyurea; improved; literacy; motivational enhancement therapy; multi-site trial; novel therapeutics; pilot trial; prototype; provider-level barriers; research to practice; routine care; satisfaction; shared decision making; sickling; social culture; standard care; system-level barriers; therapy design; tool; uptake; usability; virtual reality simulation

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.

项目摘要 虽然镰状细胞病（SCD）治疗的不断发展的景观是一个受欢迎的进步，普遍存在的 研究与实践之间的差距限制了这些疗法的普及。尽管数十年来的证据表明 羟基脲（HU）是一种改善红细胞功能和减少镰状化的药物， 在SCD护理中缓慢。只有40-65%的SCD青年接受HU，一半的人表现出很差的依从性， 青少年和年轻人（AYAs）表现出最差的依从性。HU吸收的障碍是多因素的。 AYAs报告说，他们缺乏参与决策，知识不足，对疗效和 安全为代价的供应商可能不会提供HU由于对患者依从性的担忧。这些挑战 由于社会文化障碍，护理工作更加困难。这些障碍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 迫切需要在这一高风险发展时期优化有效疗法的普及和使用。 共同决策（SDM）干预措施提高了患者的知识，参与度，对护理的满意度， 和遵守，并特别有利于边缘化群体。然而，没有这样的干预措施， AYA与SCD。为了弥补这一差距，我们将为AYA开发一种技术增强型SDM干预措施， SCD年龄15-25岁，通过扩大和调整为父母设计的决策支持干预， SCD儿童，HU共享决策（H-SDM）工具包。该工具包针对多层次障碍， 提供技术增强的工具：1）临床医生（动机访谈培训，包括虚拟现实 模拟）; 2）父母（多媒体决策辅助）;和3）执行（审计和反馈策略）。它 增强知识，减少决策冲突，并增加患者处方HU的比例。 本研究将实现以下目标：1）扩展H-SDM工具包，以包括新的决策支持 SCD治疗; 2）调整和完善H-SDM工具包，用于患有SCD的AYA; 3）评估可接受性， 可行性和经调整的工具包的初步功效。为了扩展H-SDM工具包，我们将在线 在全国范围内与SCD提供者进行众包，以评估影响最近批准的治疗的因素。 为了使H-SDM工具包适用于AYA，我们将对AYA及其护理人员进行定性访谈， 与供应商的焦点小组。然后，我们由不同利益相关者组成的团队将参加共同设计研讨会， 制定经调整的工具包的初始原型和实施蓝图，将其纳入日常工作 临床实践;将与AYA、护理人员和提供者一起进行可用性测试的迭代周期， 确定工具包和实施计划的技术和功能可靠性和实用性。最后我们将 进行单组试点试验，以评估适应的可接受性，可行性和初步疗效 工具箱。这项研究将产生一个以证据为基础的，技术增强的SDM干预，以提高覆盖面 并接受疾病缓解疗法，从而改善患有SCD的AYA的健康结果。