Overcoming Adherence Problems in Children with Iron Deficiency Anemia

克服缺铁性贫血儿童的依从性问题

基本信息

批准号：
9751929
负责人：
Jacquelyn M Powers
金额：
$ 18万
依托单位：
BAYLOR COLLEGE OF MEDICINE
依托单位国家：
美国
项目类别：
财政年份：
2016
资助国家：
美国
起止时间：
2016-08-01 至 2022-07-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/9751929
关键词：
Acute Address Adherence Adolescent Adverse effects Affect Age Attitude Award Belief Bypass Caregivers Chelation Therapy Child Chronic Clinical Clinical Trials DNA biosynthesis Data Development Development Plans Directly Observed Therapy Dose Educational workshop Ensure Environment Erythropoiesis Etiology Female of child bearing age Foundations Funding Goals Health Healthy People 2020 Heart failure Hemoglobin Inferior Infusion procedures International Intervention Interview Intravenous Intravenous infusion procedures Iron Iron deficiency anemia Knowledge Lead Logic Low income Measures Medical Mentors Methods Micronutrients Modeling Motivation Neurons Nutritional Oral Outcome Outcomes Research Outpatients Parents Patient-Focused Outcomes Patients Pediatric Hematology Persons Pharmaceutical Preparations Positioning Attribute Preparation Process Production Public Health Qualitative Research Randomized Randomized Controlled Trials Regimen Research Research Methodology Research Personnel Research Training Resistance Safety Sickle Cell Stroke Structure Testing Texas Therapeutic Intervention Time Training Transfusion Treatment Failure Treatment Protocols United States United States Agency for Healthcare Research and Quality United States National Institutes of Health arm auditory processing base behavioral adherence career career development caregiver interventions clinical care clinical efficacy comparative effectiveness comparative efficacy cost effective therapy effectiveness research executive function experience ferryl iron health related quality of life hydroxyurea improved improved outcome infection risk innovation iron deficiency medication compliance neurodevelopment novel patient oriented peer public health relevance rapid growth research study response satisfaction success symposium theories therapy development translational medicine treatment as usual visual processing

项目摘要

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.

描述（由适用提供）：全球超过10亿人患有铁缺乏贫血（IDA）。在美国，估计有3％的幼儿（1至4岁）患有IDA，这是一个快速增长的时期，在此期间，铁对于发育至关重要。但是，口服铁已使用了几个世纪来进行初级治疗，但是，不遵守导致大量患者的治疗失败。口服铁的两种有希望的替代方法：（1）通过行为依从性干预优化口服铁的递送，以及（2）一次性静脉输注（IV）铁的直接观察到治疗（DOT）。如果要么表现出改善的预后，结果将导致IDA患者治疗的范式转移。该提案描述了一项职业发展计划，该计划将使候选人成为成功的独立调查员，并实现她成为IDA患者管理国际领导者的长期职业目标。 PI的近期目标是确定成功治疗铁缺乏贫血（IDA）的最有效方法。为了实现这一目标，她提出了一项职业发展计划，该计划将教学课程，参与地方和民族会议以及密集的研讨会，并在德克萨斯州达拉斯西南部的杰出和协作研究环境中进行了研究。这种环境包括转化医学中心内的NIH资助的CTSA，以及以AHRQ资助的以患者为中心的结果研究中心。该项目的研究目的是：1）确定口服铁药物的依从性率，并表征了营养IDA幼儿铁治疗的障碍和促进剂； 2）为IDA儿童的护理人员开发依从性干预； 3）试行对标准口服铁和一剂静脉输铁的DOT方案进行干预。这些目标将成为PI职业发展计划和培训目标的基础：1）定性研究方法； 2）干预发展； 3）比较有效性研究。该K23提案的研究和培训目的共同提供了R01应用程序的培训，经验和初步数据，用于将“优化”口服铁治疗方案与标准口服铁和IV铁的DOT方案的临床效率进行比较。检查的结果将包括临床影响以及以患者为中心的结果，例如与健康相关的质量生活。拟议的研究和培训目标将使PI能够在混合方法，干预开发和比较有效性研究中发展专业知识，同时成为IDA管理的领导者。评估针对口腔铁的通常护理的新型依从性干预的创新方法以及IV铁疗法的替代方法挑战了IDA儿童口服铁治疗的范式。它有可能对IDA儿童的临床护理产生高影响。