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

项目摘要/摘要 镰状细胞病(SCD)是非裔美国人中最常见的遗传性红细胞疾病 人口。1970年以前，患有SCD的儿童中只有一半能活到成年。普遍出生的新生儿 筛查和医学进展(抗生素预防、输血、羟基尿素、干细胞移植和 基因治疗)，存活率有所提高。在SCD患者中，从儿科医疗过渡到成人医疗 是死亡的高危时期。防止过早死亡需要SCD患者进行自我护理 当他们过渡到成年的时候。然而，认知障碍是SCD的一个普遍的衰弱特征。 寿命，并影响进行自我护理所需的有效决策的能力。另外， 由于以下原因，患有SCD的过渡年龄青年面临的具体挑战加剧了认知障碍 与健康有关的差距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，以加强自我照顾和自我倡导，这可以 有助于改善成年后的护理过渡和独立性，从而防止危及生命 并发症和过早死亡。这项研究旨在利用认知补救计划(CRP)提供 通过远程医疗来提高成功过渡护理所必需的决策技能。科学目标1将 确定为期4周的CRP是否提高了患有SCD的青少年(10-18岁)的过渡准备技能。持之以恒 还将评估随时间推移的改进情况。科学目标2将评估与脑相关的变化 C反应蛋白，以确定与青少年SCD术后认知改变相关的神经生物学标志物 干预。此外，还将调查两个探索性目标，以便1)确定人口统计学和临床 与CRP后过渡准备技能提高相关的特征，以及2)测试可行性 和为期1年的CRP后强化疗程的初步疗效，以继续保持过渡 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 并将为其他培训和职业发展提供机会。职业生涯发展 计划包括进行临床试验的特定研究方法的正式培训，参与 少数民族人群的研究，以及神经成像方法和分析。科学和训练计划 由经验丰富的导师和顾问组成的团队提供支持，他们致力于这个项目的成功 以及我作为一名以病人为中心的科学家的发展。初级导师是神经认知结果方面的专家。 在血液疾病中，一位经验丰富的年轻研究人员的导师，以及UAB研究所的主任 癌症结局和存活率。这项提议的结果将被用来形成未来的基础 临床试验旨在测试那些最有可能从干预中受益的人中精炼的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。

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.