Sickle Cell Disease Functional Assessment Validation and Exercise Pilot Intervention

1个项目摘要/摘要 2尽管镰状细胞病的平均预期寿命从1973年的14岁提高到 3 61年来，来自学术中心的队列中，患有镰状细胞病(SCD)的成年人 4出现功能障碍。他们会在身体、社交和认知方面出现缺陷 5由于年龄相关的变化和SCD并发症，如缺血性坏死， 6中风、心肺疾病和感觉障碍。没有经过验证的评估 7改善老年SCD患者身体功能的工具或干预。未能认识到 8和管理脆弱性和功能衰退可能导致残疾和过早死亡。在……里面 9一项初步研究，我们评估了镰刀细胞疾病功能评估(SCD-SCD)的可行性 10 FA)，证明是可行的、可接受的、安全的。在这项研究中，我们将评估 11 SCD-FA在老年SCD(年龄≥)前瞻性队列中的效度和信度 1240年)(目标1)。我们将评估SCD-FA在预测天数方面的效用 每年住院13人为主要结局，体能变化，镰刀细胞 14日间医院就诊、急诊科就诊、对日常生活活动的依赖以及 15例死亡为次要后果。我们还将确定身体受损的风险因素 16绩效(目标2)。SCD-FA将提供一种方法，用于：1)评估 17多个健康领域，2)确定干预目标，3)衡量 18功能。为了解决功能障碍，我们将调整和试点个性化练习 19老年人(年龄≥40岁)与慢性阻塞性肺疾病(目标3)培训计划。这次干预将是一次 20从Gerofit改编的为期8周的虚拟计划，这是一个监督运动和健康的计划 21老年人推广计划，包括为每个人的健康量身定做的锻炼 22状态和潜在的功能缺陷。预期的结果是有试点数据来设计一个 23对患有SCD的老年人进行个性化运动干预的随机对照试验。 24岁的Oyedeji博士是杜克大学血液科的医学助理教授 25所大学。她的职业发展计划包括开发健康评估工具的技能 26在#年专家的指导下制定和制定和实施干预措施 27心理测量学、干预发展、实施、老年病学和镰状细胞病。 28这项研究结合了课程作业、职业发展活动和她的指导 29团队将提供必要的经验来支持她的成功，因为她正在朝着她的方向努力 30成为SCD和老龄化领域的独立调查者和领导者的目标。

1 PROJECT SUMMARY/ABSTRACT 2 Despite improvements in median life expectancy for sickle cell disease from 14 years in 1973 to 3 61 years in recent cohorts from academic centers, adults with sickle cell disease (SCD) 4 experience functional impairments. They develop deficits in physical, social, and cognitive 5 function due to both age-related changes and SCD complications, such as avascular necrosis, 6 strokes, cardiopulmonary disease, and sensory deficits. There are no validated assessment 7 tools or interventions to improve physical function in older adults with SCD. Failure to recognize 8 and manage vulnerabilities and functional decline can lead to disability and premature death. In 9 a pilot study we evaluated the feasibility if the Sickle Cell Disease Functional Assessment (SCD- 10 FA) and demonstrated that it was feasible, acceptable, and safe. In this study, we will evaluate 11 the validity and reliability of the SCD-FA in a prospective cohort of older adults with SCD (age ≥ 12 40 years) (Aim 1). We will evaluate the utility of the SCD-FA to predict number of days 13 hospitalized per year as the primary outcome, and change in physical performance, Sickle Cell 14 Day Hospital visits, emergency department visits, dependence in Activities of Daily Living, and 15 death as secondary outcomes. We will also determine risk factors for impaired physical 16 performance (Aim 2). The SCD-FA will provide a method for: 1) assessing vulnerabilities in 17 multiple health domains, 2) identifying targets for interventions, and 3) measuring change in 18 function. To address functional impairments, we will adapt and pilot a personalized exercise 19 training program in older adults (age ≥ 40 years) with SCD (Aim 3). The intervention will be an 20 8-week virtual program adapted from Gerofit, which is a supervised exercise and health 21 promotion program for older adults that includes exercises customized to each person’s heath 22 status and underlying functional deficits. The expected outcome is to have pilot data to design a 23 randomized control trial of a personalized exercise intervention for older adults with SCD. 24 Dr. Oyedeji is an Assistant Professor of Medicine in the Division of Hematology at Duke 25 University. Her career development plan includes developing skills in health assessment tool 26 development and developing and implementing interventions with guidance from experts in 27 psychometrics, intervention development, implementation, geriatrics, and sickle cell disease. 28 This research coupled with coursework, professional development activities, and her mentorship 29 team will provide the necessary experience to support her success as she works towards her 30 goal of becoming an independent investigator and leader in SCD and aging.