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

提高镰状细胞病儿童羟基脲依从性的多维策略

• 批准号: 8869788
• 负责人: Susan Creary
• 金额: $ 12.76万
• 依托单位: RESEARCH INST NATIONWIDE CHILDREN'S HOSP
• 依托单位国家: 美国
• 财政年份: 2015
• 资助国家: 美国
• 起止时间: 2015-09-01 至 2020-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8869788
• 关键词: Address; Adherence; Adolescent; Adolescent and Young Adult; Adult; Affect; Age; Area; Behavioral; Biological Markers; Caring; Cessation of life; Child; Childhood; Chronic; Clinical Investigator; Clinical Trials; Complex; Data; Development Plans; Directly Observed Therapy; Disease; Electronics; Erythrocytes; Face; Feedback; Funding; Goals; Health; Health Care Costs; Health Services Research; Hematological Disease; Incentives; Incidence; Inherited; Institution; Intervention; Measures; Mentorship; Morbidity - disease rate; Multicenter Studies; National Heart, Lung, and Blood Institute; Outcome; Pain; Participant; Patient Self-Report; Patients; Pediatric Hospitals; Pharmaceutical Preparations; Population; Premature Mortality; Provider; Records; Reporting; Research; Research Project Grants; Risk; Self Management; Sickle Cell; Sickle Cell Anemia; Subgroup; Surveys; Testing; Time; United States; acute chest syndrome; career development; clinical practice; contingency management; cost effective; cost effectiveness; design; health disparity; high risk; hydroxyurea; improved; innovation; medication compliance; mortality; patient population; population health; public health relevance; skills; success; successful intervention; theories; tool

 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.

 描述（由申请人提供）：镰状细胞病是最常见的遗传性血液疾病，它会导致显着的发病率和死亡率。患有这种疾病的青少年面临特别高的死亡风险，因为他们当时常常没有准备好管理他们的药物。羟基脲是镰状细胞病患者唯一可用的疾病缓解药物，虽然多项临床试验表明羟基脲可以减少并发症，但补充记录表明很少有患者坚持使用。尽管如此，依从性干预措施仍然未经检验，我的长期目标是成为镰状细胞病和药物依从性的独立临床研究者。我提出的研究的目的是确定一种称为“移动直接观察治疗”或“移动 DOT”的创新电子工具的影响，该工具使用警报消息、视频、反馈和激励措施来鼓励患有羟基脲的儿童和青少年坚持使用羟基脲。镰刀具体而言，本研究旨在确定：(1) 通过补充记录测量，Mobile DOT 是否可以改善羟基脲依从性；(2) 羟基脲生物标志物、自我报告的依从性和补充依从性是否是有效的羟基脲依从性衡量标准；(3)如果移动 DOT 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。全国儿童医院研究。 为这项研究提供了理想的环境，因为它有大量患有镰状细胞病的儿童和青少年，并且为完成卫生服务研究项目提供了强有力的机构支持，这项研究的结果将为最终的多中心研究的设计提供信息。寻求确定羟基脲的电子依从工具是否具有成本效益，并能改善患有镰状细胞病的儿童和青少年的治疗结果。