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: 10427879
• 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/10427879
• 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; Education; Enrollment; Erythrocytes; Evidence based intervention; Foundations; Functional Magnetic Resonance Imaging; Future; Genetic; Goals; Health; Hematological Disease; Impaired cognition; Infection; Institutes; 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 Care; Sickle Cell Anemia; Stem cell transplant; Stroke; Survival Rate; Testing; Time; Training; Transfusion; Waiting Lists; Work; Youth; acute chest syndrome; aged; arm; base; 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; neuroimaging; neuromechanism; patient oriented; premature; prevent; programs; relating to nervous system; remediation; response; satisfaction; skills; social health determinants; success; survivorship; telehealth; transplantation therapy; 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.

项目总结/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 人口在1970年之前，只有一半的SCD儿童存活到成年。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），存活率有所提高。在SCD患者中，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 当他们过渡到成年期。然而，认知障碍是SCD的一个普遍的衰弱特征， 寿命，并影响自我护理所需的有效决策能力。此外，本发明还 患有SCD的过渡年龄青年面临的特定挑战使认知障碍变得更加复杂， 与健康有关的差距和健康的不良社会决定因素。因此，患者的需求未得到满足 与SCD采用和吸收决策技能，以加强自我保健和自我宣传，这可以 改善成年后的照料和独立过渡，从而防止危及生命的 并发症和过早死亡。本研究旨在利用认知补救计划（CRP）提供 通过远程保健提高成功过渡护理所需的决策技能。科学目标1 确定4周CRP是否改善SCD青年（10- 18岁）的过渡准备技能。持续存在 还将评估随着时间推移的改进情况。科学目标2将评估大脑相关的变化， CRP，以确定神经生物学标志物与SCD青年认知变化有关， 干预此外，将研究两个探索性目的，以1）确定人口统计学和临床 与CRP后过渡准备技能改善相关的特征，以及2）测试可行性 和CRP加强治疗后1年的初步疗效，以继续维持过渡期 准备技能。总的来说，这项研究将促进我提高患者生活质量的长期职业目标 并将提供额外的培训和职业发展的机会。职业发展 计划包括开展临床试验的研究方法的正式培训， 研究中的少数群体，以及神经成像方法和分析。科学和培训计划 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 以及我作为一个以病人为导向的科学家的发展。主要导师是神经认知结果方面的专家 在血液疾病中，年轻研究人员的经验丰富的导师，以及UAB研究所的主任， 癌症的结果和生存。这一建议的结果将被用来构成未来的基础， 一项临床试验，旨在测试最有可能从干预中获益的人群中的精制CRP。 这个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， 为改善SCD患者的长期健康结局做出真正的贡献。