Overcoming Adherence Problems in Children with Iron Deficiency Anemia

克服缺铁性贫血儿童的依从性问题

• 批准号: 9087768
• 负责人: Jacquelyn M Powers
• 金额: $ 18.18万
• 依托单位: BAYLOR COLLEGE OF MEDICINE
• 依托单位国家: 美国
• 财政年份: 2016
• 资助国家: 美国
• 起止时间: 2016-08-01 至 2020-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9087768
• 关键词: Acute; Address; Adherence; Adolescent; Adverse effects; Affect; Age; Attitude; Auditory; Award; Behavioral; 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; Infection; 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; Risk; Safety; Sickle Cell; Structure; Testing; Texas; Time; Training; Transfusion; Treatment Failure; Treatment Protocols; United States; United States Agency for Healthcare Research and Quality; United States National Institutes of Health; Visual; arm; base; career; career development; caregiver interventions; clinical care; clinical efficacy; comparative effectiveness; comparative efficacy; cost; effective therapy; effectiveness research; executive function; experience; health related quality of life; hydroxyurea; improved; improved outcome; innovation; iron deficiency; medication compliance; meetings; neurodevelopment; novel; public health relevance; rapid growth; research study; response; satisfaction; success; symposium; theories; therapy development; translational medicine; treatment as usual

 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(申请人提供)：全球超过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)患有缺铁性贫血，这是一个快速生长的时期，在此期间，铁对发育至关重要。几个世纪以来，口服铁一直被用于初级治疗，然而，在相当数量的患者中，不坚持治疗会导致治疗失败。口服铁有两种有希望的替代方法：(1)通过行为依从性干预来优化口服铁的输送，以及(2)一次性静脉注射铁的直接观察疗法(DOT)。如果任何一种方案都显示出改善的结果，结果将导致IDA患者治疗模式的转变。这份提案描述了一项职业发展计划，该计划将使候选人为成为一名成功的独立调查员做好准备，并实现她成为IDA患者管理方面的国际领导者的长期职业目标。该协会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，她提出了一项职业发展计划，其中包括授课、参加地方和全国会议和密集研讨会，并在德克萨斯州达拉斯的德克萨斯大学西南分校卓越和协作的研究环境中指导研究学习。这种环境包括转化医学中心内由NIH资助的CTSA和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)确定营养性缺铁性贫血儿童口服铁质药物依从性的比率，并确定铁治疗的障碍和促进者；2)为IDA儿童的照顾者开发铁依从性干预措施；3)试行针对标准口服铁质的干预措施和一次性静脉注射铁的DOT方案。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。总而言之，K23提案的研究和培训目标将为R01应用RCT提供培训、经验和初步数据，该RCT的功率足以比较标准口服铁剂和静脉铁剂的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，如与健康相关的质量 生活。拟议的研究和培训目标将使国际开发协会能够发展混合方法、干预措施发展和比较有效性研究方面的专门知识，同时成为国际开发协会管理方面的领导者。评估一种新的依从性干预措施与常规的口服铁质护理以及静脉铁质疗法替代方案的创新方法，挑战了IDA儿童口服铁质疗法的范式。它有可能对IDA儿童的临床护理产生很大影响。