Longitudinal study of cognitive remediation to prepare for transition of care in youth with sickle cell disease

认知补救的纵向研究，为患有镰状细胞病的青少年的护理过渡做好准备

• 批准号: 10661515
• 负责人: Donna Murdaugh
• 金额: $ 11.46万
• 依托单位: UNIVERSITY OF ALABAMA AT BIRMINGHAM
• 依托单位国家: 美国
• 财政年份: 2022
• 资助国家: 美国
• 起止时间: 2022-07-07 至 2027-03-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10661515
• 关键词: Admission activity; Adolescent; Adopted; Adult; Advocacy; African American; African American population; Age; Antibiotic Prophylaxis; Area; Behavioral; Brain; Brain Injuries; Brain region; Caring; Cause of Death; Cessation of life; Characteristics; Child; Childhood; Chronic; Clinical; Clinical Trials Design; Cognitive; Cognitive deficits; Cognitive remediation; Conduct Clinical Trials; Coupled; Decision Making; Deep South; Deposition; Development; Development Plans; Disease; Disparity; Education; Enrollment; Erythrocytes; Evidence based intervention; Foundations; Functional Magnetic Resonance Imaging; Future; Genetic; Goals; Health; Healthcare; Hematological Disease; Impaired cognition; Infection; Intervention; Iron; K-Series Research Career Programs; Life; Life Expectancy; Longevity; Longitudinal Studies; Malignant Neoplasms; Measures; Medical; Mentors; Methodology; Minority Groups; Morbidity - disease rate; Nature; Neonatal Screening; Neurobiology; Neurocognitive; Neurologic; Neurophysiology - biologic function; Neuropsychological Tests; Outcome; Parents; Patient Self-Report; Patients; Pattern; Prevalence; Psychological reinforcement; Quality of life; Randomized, Controlled Trials; Readiness; Research; Research Methodology; Research Personnel; Research Proposals; Rest; Risk; Scientist; Self Assessment; Self Care; Sickle Cell Anemia; Stem cell transplant; Stroke; Survival Rate; Testing; Time; Training; Transfusion; Waiting Lists; Work; Youth; acute chest syndrome; aged; arm; career; career development; cognitive change; cognitive function; deprivation; early childhood; efficacy evaluation; executive function; experience; feasibility testing; follow-up; gene therapy; high risk; hydroxyurea; improved; indexing; male; minority engagement; multimodal neuroimaging; neural; neuroimaging; neuromechanism; patient oriented; premature; prevent; programs; remediation; response; satisfaction; skills; social health determinants; success; survivorship; telehealth; vaso-occlusive crisis

PROJECT SUMMARY / ABSTRACT Sickle cell disease (SCD) is the most common genetic red blood cell disorder in the African American (AA) population. Prior to 1970, only half of the children with SCD survived to adulthood. With universal newborn screening and medical advances (antibiotic prophylaxis, transfusions, hydroxyurea, stem cell transplantation and gene therapy), survival rates have improved. In patients with SCD, transition from pediatric to adult medical care is a high-risk period for death. Preventing premature death requires that patients with SCD engage in self-care as they transition into adulthood. However, cognitive impairment is a pervasive debilitating feature of SCD across the lifespan, and influences ability to engage in effective decision-making needed for self-care. Additionally, cognitive impairment is compounded by specific challenges faced by transition-aged youth with SCD due to health-related disparities and poor social determinants of health. As such, there is an unmet need for patients with SCD to adopt and assimilate decision-making skills to enhance self-care and self-advocacy, which could result in improvement in transition of care and independence in adulthood, thus preventing life-threatening complications and premature death. This study aims to utilize a cognitive remediation program (CRP) delivered via telehealth to improve decision-making skills necessary for successful transition of care. Scientific Aim 1 will determine if a 4-week CRP improves transition readiness skills in youth with SCD (10-18y). Persistence of improvement across time will also be evaluated. Scientific Aim 2 will assess brain-related changes pre- to post- CRP, in order to identify neurobiological markers related to cognitive changes in youth with SCD after intervention. Additionally, two exploratory aims will be investigated in order to 1) identify demographic and clinical characteristics associated with improvement in transition readiness skills post-CRP, and 2) to test the feasibility and preliminary efficacy of a 1-year post-CRP booster session in order to continue to maintain transition readiness skills. Overall, this study will promote my long-term career goal of improving quality of life for patients with SCD and will provide opportunities for additional training and career development. The career development plan includes formal training in research methodology specific to conducting clinical trials, engagement of minority populations in research, and neuroimaging methodology and analyses. The scientific and training plans are supported by a team of experienced mentors and advisors who are committed to the success of this project and my development as a patient-oriented scientist. The primary mentor is an expert in neurocognitive outcomes among blood disorders, an experienced mentor of young investigators, and the director of the UAB Institute for Cancer Outcomes and Survivorship. The results of this proposal will be utilized to form the foundation of a future clinical trial designed to test the refined CRP among those who are most likely to benefit from the intervention. This project will strengthen my skills as a clinical researcher, establish an independent research platform, and make a true contribution towards improving long-term health outcomes in patients with SCD.

项目概要/摘要 镰状细胞病 (SCD) 是非裔美国人 (AA) 中最常见的遗传性红细胞疾病 人口。 1970 年之前，只有一半患有 SCD 的儿童能活到成年。与通用新生儿 筛查和医学进步（抗生素预防、输血、羟基脲、干细胞移植和 基因治疗），生存率有所提高。对于 SCD 患者，从儿科医疗护理过渡到成人医疗护理 是死亡的高危期。预防过早死亡需要 SCD 患者进行自我护理 当他们过渡到成年时。然而，认知障碍是 SCD 的一个普遍的衰弱特征。 寿命，并影响自我保健所需的有效决策的能力。此外， 患有 SCD 的过渡年龄青少年面临的具体挑战加剧了认知障碍 与健康相关的差异和不良的健康社会决定因素。因此，患者的需求尚未得到满足 与 SCD 一起采用和吸收决策技能，以增强自我保健和自我倡导，这可以 改善成年后的照料过渡和独立性，从而防止危及生命的情况 并发症和过早死亡。本研究旨在利用认知矫正计划（CRP） 通过远程医疗来提高护理成功过渡所需的决策技能。科学目标 1 将 确定为期 4 周的 CRP 是否可以提高 SCD 青少年（10-18 岁）的过渡准备技能。持续存在 还将评估随时间推移的改进。科学目标 2 将评估治疗前后大脑相关的变化 CRP，以确定与 SCD 青少年认知变化相关的神经生物学标志物 干涉。此外，还将研究两个探索性目标，以便 1）确定人口统计和临床 与 CRP 后过渡准备技能改进相关的特征，以及 2) 测试可行性 以及 CRP 后 1 年加强会议的初步效果，以继续维持过渡 准备技能。总的来说，这项研究将促进我改善患者生活质量的长期职业目标 与 SCD 合作，并将提供额外培训和职业发展的机会。职业发展 计划包括针对进行临床试验的研究方法的正式培训、参与 少数群体的研究以及神经影像方法和分析。科学的培训计划 由经验丰富的导师和顾问团队提供支持，他们致力于该项目的成功 以及我作为一名以患者为中心的科学家的发展。主要导师是神经认知结果方面的专家 在血液疾病领域，年轻研究者的经验丰富的导师，UAB 研究所所长 癌症结果和生存率。该提案的结果将被用来为未来奠定基础 旨在测试最有可能从干预中受益的人群的精细 CRP 的临床试验。 这个项目将增强我作为临床研究员的技能，建立一个独立的研究平台，并 为改善 SCD 患者的长期健康结果做出真正的贡献。