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.

项目摘要 /摘要 镰状细胞病（SCD）是非裔美国人最常见的遗传红细胞疾病（AA） 人口。在1970年之前，只有一半的SCD儿童幸存到成年。与环球新生儿 筛查和医学进展（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。在SCD患者中，从小儿过渡到成人医疗 是死亡的高风险时期。防止过早死亡需要SCD患者进行自我保健 当他们过渡到成年时。但是，认知障碍是SCD的普遍衰弱特征 寿命，影响着自我保健所需的有效决策的能力。此外， 认知障碍是由SCD过渡时代青年面临的具体挑战加剧了 与健康有关的差异和健康的社会决定因素不良。因此，对患者有未满足的需求 借助SCD采用并吸收决策技巧，以增强自我保健和自我倡导，这可能 导致成年后护理和独立过渡的改善，从而防止威胁生命 并发症和过早死亡。这项研究旨在利用交付的认知补救计划（CRP） 通过远程医疗，提高成功过渡护理所需的决策技巧。科学目标1将 确定为期4周的CRP是否提高了SCD（10-18y）青年的过渡准备技能。持久性 跨时间的改进也将进行评估。科学目标2将评估与大脑相关的变化， CRP，为了确定与SCD青年认知变化有关的神经生物学标记 干涉。此外，将研究两个探索目标，以确定人口统计学和临床 与CRP后过渡准备技能提高有关的特征和2）测试可行性 和CRP后1年的助推器会议的初步疗效，以便继续保持过渡 准备技能。总体而言，这项研究将促进我改善患者生活质量的长期职业目标 使用SCD，将为额外的培训和职业发展提供机会。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 研究中的少数人群以及神经影像学方法和分析。科学和培训计划 得到了一个经验丰富的导师和顾问团队的支持，他们致力于该项目的成功 以及我作为以患者为导向的科学家的发展。主要导师是神经认知结果的专家 在血液疾病中，年轻调查人员的经验丰富的导师和UAB研究所主任 癌症的结果和生存。该提案的结果将用于构成未来的基础 临床试验旨在测试最有可能从干预措施中受益的人中的CRP。 该项目将加强我作为临床研究人员的技能，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。