A multidimensional strategy to improve hydroxyurea adherence in children with sickle cell

 描述(申请人提供)：镰状细胞病是最常见的遗传性血液疾病，它会导致严重的发病率和死亡率。患有这种疾病的青少年死亡风险特别高，因为他们在过渡到成人护理提供者时往往没有准备好管理自己的药物。羟基脲是镰状细胞病患者唯一可用的疾病修改药物。虽然多项临床试验表明羟基尿素可以减少并发症，但再灌装记录表明，很少有患者坚持使用羟基尿素达到临床试验所达到的水平。依从性差的患者比依从性高的患者健康结果更差。尽管如此，依从性干预措施仍未得到检验。我的长期目标是成为镰状细胞疾病和用药依从性方面的独立临床研究员。我提出的研究的目的是确定一种创新的电子工具，称为移动直接观察疗法，或“移动DOT”的影响。Mobile DOT使用警报信息、视频、反馈和激励措施来鼓励患有镰状细胞疾病的儿童和青少年坚持使用羟基尿素。具体地说，这项研究的目的是确定：(1)移动DOT是否改善了通过重新填充记录衡量的羟基尿素依从性；(2)羟基尿素生物标记物、自我报告依从性和重新填充依从性是否是有效的羟基尿素依从性措施；以及(3)移动DOT是否能够提高青少年参与者的自我管理技能。我的职业发展计划包括三个重点领域的指导、正式课程和研讨会：行为理论、青少年和年轻人的过渡，以及临床试验和成本效益研究。全国儿童医院 它为这项研究提供了一个理想的环境，因为它有大量患有镰状细胞疾病的儿童和青少年，而且有强大的机构支持来完成卫生服务研究项目。这项研究的结果将为最终的多中心研究的设计提供参考，该研究将寻求确定羟基尿素的电子黏附工具是否具有成本效益，并导致改善患有镰状细胞疾病的儿童和青少年的结果。

 DESCRIPTION (provided by applicant): Sickle cell disease is the most common inherited blood disorder and it leads to significant morbidity and mortality. Adolescents with this disease are at a particularly high risk of death because they are frequently unprepared to manage their medications at the time of transition to adult care providers. Hydroxyurea is the only disease-modifying medication available for patients with sickle cell disease. While multiple clinical trial show that hydroxyurea can reduce complications, refill records indicate that few patients adhere to hydroxyurea at the levels achieved in clinical trials. Poorly adherent patients have worse health outcomes than those with high adherence. Despite this, adherence interventions remain untested. My long-term goal is to become an independent clinical investigator in sickle cell disease and medication adherence. The objective of my proposed study is to determine the impact of an innovative electronic tool, called Mobile Directly Observed Therapy, or "Mobile DOT." Mobile DOT uses alert messages, videos, feedback, and incentives to encourage hydroxyurea adherence in children and adolescents with sickle cell disease. Specifically, this study aims to determine: (1) if Mobile DOT improves hydroxyurea adherence as measured by refill records (2) if hydroxyurea biomarkers, self-reported adherence, and refill adherence are valid hydroxyurea adherence measures, and (3) if Mobile DOT leads to improved self-management skills in adolescent participants. My career development plan includes mentorship, formal coursework, and seminars in three focus areas: behavioral theory, adolescent and young adult transition, and clinical trial and cost-effectiveness studies. Nationwide Children's Hospital provides an ideal setting for this research because it has a large population of children and adolescents with sickle cell disease and the strong institutional support to complete health services research projects. Results from this study will inform the design of the definitive multi-center study that will seek to determine if an electronic adherence tool for hydroxyurea is cost-effective and leads to improved outcomes for children and adolescents with sickle cell disease.