Overcoming Adherence Problems in Children with Iron Deficiency Anemia

 描述（由申请人提供）：全世界有超过10亿人患有缺铁性贫血（IDA）。在美国，估计有3%的幼儿（1至4岁）患有IDA，这是一个快速生长的时期，在此期间铁对发育至关重要。口服铁剂已用于初级治疗数百年，然而，不依从性导致大量患者的治疗失败。口服铁有两种有前途的替代方法：（1）通过行为依从性干预优化口服铁递送，以及（2）一次性静脉（IV）输注铁的直接观察治疗（DOT）。如果任何一种方案显示出改善的结果，其结果将导致IDA患者治疗的范式转变。该提案描述了一个职业发展计划，将准备候选人成为一个成功的独立研究者，并实现她的长期职业目标，成为IDA患者管理的国际领导者。PI的近期目标是确定成功治疗缺铁性贫血（IDA）儿童的最有效方法。为了实现这一目标，她提出了一个职业发展计划，整合了教学课程，参加地方和国家会议和密集的研讨会，并在德克萨斯州达拉斯的UT西南部的杰出和合作研究环境中指导研究。该环境包括转化医学中心内的NIH资助的CTSA和AHRQ资助的以患者为中心的结局研究中心。该项目的研究目的是：1）确定口服铁剂药物依从性的比率，并描述营养性IDA幼儿铁剂治疗的障碍和促进因素; 2）为IDA儿童的照顾者制定依从性干预措施; 3）对标准口服铁剂和一次性IV铁剂的DOT方案进行干预。这些目标将作为PI职业发展计划和培训目标的基础：1）定性研究方法; 2）干预开发; 3）比较有效性研究。总之，本K23提案的研究和培训目标将为RCT的R 01申请提供培训、经验和初步数据，该RCT足以比较“优化”口服铁剂方案与标准口服铁剂和静脉铁剂DOT方案的临床疗效。检查的结果将包括临床影响以及以患者为中心的结果，如与健康相关的 生活拟议的研究和培训目标将使PI发展混合方法，干预措施的发展和比较有效性研究的专业知识，同时成为IDA管理的领导者。评估一种新的依从性干预与口服铁剂常规治疗以及替代IV铁剂治疗的创新方法挑战了IDA儿童口服铁剂治疗的范式。它有可能对IDA儿童的临床护理产生重大影响。

 DESCRIPTION (provided by applicant): Over 1 billion persons worldwide have iron deficiency anemia (IDA). In the United States, an estimated 3% of young children (age 1 to 4 years) have IDA, a time of rapid growth during which iron is critical for development. Oral iron has been used for centuries for primary treatment, however, non-adherence results in treatment failure in a substantial number of patients. Two promising alternative approaches exist to oral iron: (1) optimization of oral iron delivery via a behavioral adherence intervention, and (2) directly observed therapy (DOT) of a one-time infusion of intravenous (IV) iron. Should either regimen demonstrate improved outcomes, the result would lead to a paradigm shift in the treatment of patients with IDA. This proposal describes a career development plan that will prepare the candidate to become a successful independent investigator and attain her long-term career goal of becoming an international leader in the management of patients with IDA. The PI's immediate goal is to identify the most effective approach for successful treatment of children with iron deficiency anemia (IDA). To meet this goal, she has proposed a career development plan that integrates didactic coursework, participation in local and national conferences and intensive workshops, and mentored research studies within the distinguished and collaborative research environment at UT Southwestern in Dallas, Texas. This environment includes an NIH-funded CTSA within the Center for Translational Medicine and an AHRQ-funded Center for Patient-Centered Outcomes Research. The research aims of this project are to: 1) determine rates of oral iron medication adherence and characterize barriers to and facilitators of iron therapy in young children with nutritional IDA; 2) develop an adherence intervention for caregivers of children with IDA; and 3) pilot the intervention against standard oral iron and a DOT regimen with a one-time dose of IV iron. These aims will serve as the foundation for the PI's career development plan and training aims: 1) qualitative research methods; 2) intervention development; and 3) comparative effectiveness research. Together, the research and training aims of this K23 proposal will provide the training, experience, and preliminary data for an R01 application for an RCT sufficiently powered to compare the clinical efficacy of the "optimized" oral iron regimen with standard oral iron and the DOT regimen of IV iron. Outcomes examined will include clinical impact as well as patient-centered outcomes such as health-related quality of life. The proposed research and training aims will allow the PI to develop expertise in mixed-methods, intervention development and comparative effectiveness research while becoming a leader in the management of IDA. The innovative approach of assessing a novel adherence intervention against usual care with oral iron as well as the alternative of IV iron therapy challenges the paradigm of oral iron therapy for children with IDA. It has the potential for high impact on clinical care in children with IDA.