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.

项目摘要/摘要