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

总结/摘要： 镰状细胞病（SCD）是一种严重的遗传性血液病，导致频繁的急性和昂贵的长期治疗。 并发症羟基脲是儿童时期主要的SCD修饰药物，因为研究表明， 可以减少急性并发症和减轻昂贵的长期合并症。羟基脲不粘附是 然而，这是一个普遍的问题，也是它对临床上年轻人的结果影响有限的一个关键原因。 实践尽管提高儿童和青少年对羟基脲的依从性可能会产生影响， 尽管对减少SCD负担有重要作用，但缺乏测试促进青年依从性干预措施的研究。视频 直接观察治疗（VDOT）是一种电子羟基脲粘附促进策略， 智能手机，包括提醒警报，视频直接观察治疗由训练有素的观察员，鼓励 来自观察者的遵守反馈，以及实现遵守目标的货币激励。VDOT拥有 在一项单臂研究中进行了测试，并显示出有希望的结果。然而，减员率高于预期， 由于对以前用于交付VDOT的电子邮件平台和许多参与者的不满， 具有不一致的个人智能电话和蜂窝数据访问。与VDOT的接触并不是 彻底评估，即使与VDOT的接触程度有可能减轻影响 的干预。先前研究的方面，包括用于测量依从性的方法和 非随机设计也限制了明确得出VDOT改善依从性的结论的能力。最后， VDOT停止后观察到的依从性下降，无任何额外依从性 支持者建议，在强化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 健康这是一个小企业，有一个用户友好的VDOT平台，经验成功地提供 其他慢性病人群的VDOT，以及提供间歇性持续依从性的既定系统 在VDOT后提供支持，以限制人员流失、优化参与者参与度并维持依从性。因此 本提案的主要目的是评估在试点RCT期间的保留率和持续参与度 通过emocha Health平台将VDOT与注意力控制进行比较。我们还将探讨青少年和 护理人员对长期依从性监测和干预的需求和偏好。本申请 与NHLBI的目标一致，即优化SCD的治疗策略，因为它提高了依从性， 与支持终身健康行为和改善这些年轻人的结果有关。

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.