ADHERE (Applying Directly observed therapy to HydroxyurEa to Realize Effectiveness)

摘要/摘要： 镰状细胞病(SCD)是一种严重的遗传性血液疾病，会导致频繁的急性和代价高昂的长期 并发症。羟基脲是儿童时期治疗SCD的主要药物，因为研究表明，它 可以减少急性并发症，减轻代价高昂的长期并存。羟基脲不粘连是 然而，这是常见的，也是它对临床上年轻人的结果影响有限的一个关键原因 练习一下。尽管改善儿童和青少年中羟基尿素的依从性可能会产生影响 在减少慢性阻塞性肺病负担方面，缺乏对促进青少年坚持治疗干预措施进行测试的研究。视频 直接观察疗法(VDOT)是一种电子羟基脲黏附促进策略，使用 智能手机，包括提醒提醒，训练有素的观察者直接观察治疗的视频，鼓励 来自观察员的遵守反馈，以及实现遵守目标的货币激励。VDOT有 在一项单臂研究中进行了测试，并显示了令人振奋的结果。然而，流失率比预期的要高 对以前用于提供VDOT和许多参与者的电子邮件平台表示一些不满 对个人智能手机和蜂窝数据的访问不一致。与VDOT的合作不是 经过彻底评估，即使与VDOT的接触程度有可能缓解影响 干预的结果。前一项研究的方面，包括用于衡量依从性的方法和 非随机化设计也限制了明确得出VDOT改善依从性的能力。最后， 在VDOT停止后观察到的遵从性下降，没有任何额外的遵从性 支持建议在密集VDOT后可能需要额外的监测和干预以促进 持久的坚持行为改变。这种反馈和体验促使我们与emocha合作 健康。这是一家拥有用户友好的VDOT平台的小型企业，体验成功提供 向其他慢性疾病人群提供VDOT，以及提供间歇性持续依从性的既定系统 在VDOT之后提供支持，以限制人员流失、优化参与者参与度并保持忠诚度。因此， 该建议的主要目标是评估在试点RCT期间的保留和持续参与 通过emocha Health平台将VDOT与注意力控制进行比较。我们还将探索青少年和 照顾者对长期依从性监测和干预的需求和偏好。此应用程序 与NHLBI优化SCD治疗策略的目标一致，因为它提高依从性至关重要 与支持这些青年的终身健康行为和改善结果有关。

Summary/Abstract: 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.