Sickle Cell Disease Functional Assessment Validation and Exercise Pilot Intervention

1项目概要/摘要 2尽管镰状细胞病患者的平均预期寿命从1973年的14岁提高到1997年的14岁， 3来自学术中心的最近队列中的61年，患有镰状细胞病（SCD）的成人 四是功能障碍。他们在身体、社交和认知方面都有缺陷， 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，和感觉缺陷。没有经过验证的评估 7种工具或干预措施，以改善患有SCD的老年人的身体功能。不承认 8和管理脆弱性和功能下降可能导致残疾和过早死亡。在 9.一项初步研究，我们评估了镰状细胞病功能评估（SCD-1）的可行性。 10 FA），并证明其可行、可接受和安全。在这项研究中，我们将评估 11在老年SCD患者（年龄≥ 10岁）的前瞻性队列中，SCD-FA的有效性和可靠性 12 40年）（目标1）。我们将评估SCD-FA预测天数的实用性 每年13例住院患者作为主要结局，以及体能变化、镰状细胞 14天医院访视、急诊科访视、日常生活活动依赖性，以及 15例死亡为次要结局。我们还将确定身体受损的风险因素， 16业绩（目标2）。SCD-FA将提供一种方法，用于：1）评估 17多个卫生领域，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的演习 19项老年SCD患者（年龄≥ 40岁）的培训计划（目标3）。干预将是一个 20 8周的虚拟程序改编自Gerofit，这是一个监督锻炼和健康 21个针对老年人的推广计划，包括根据每个人的健康状况定制的锻炼 22状态和潜在功能缺陷。预期的结果是获得试点数据， 23项针对老年SCD患者的个性化运动干预的随机对照试验。 Oyedeji博士是杜克大学血液学系的医学助理教授 25大学她的职业发展计划包括发展健康评估工具的技能 在专家的指导下， 27心理测量学，干预措施的制定，实施，老年医学和镰状细胞病。 28这项研究加上课程，专业发展活动，和她的导师 29团队将提供必要的经验，以支持她的成功，因为她对她的工作 30目标是成为SCD和老龄化领域的独立研究者和领导者。

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.