Educational Attainment in Sickle Cell Disease

描述(由申请人提供)： 这项建议的目的是支持申请者成为一名独立的临床调查员，在提高镰状细胞病(SCD)儿童的教育水平方面具有专业知识。申请者将与她的导师和华盛顿大学医学院的多学科SCD中风研究团队合作。SCD卒中研究团队协调了24个地点的多中心静默脑梗塞输血试验(SITT)，申请者是当地的首席研究员。她将在现有的独立试点试验和她的调查员发起的SITT辅助研究方面获得经验。这些活动将为开展以患者为中心的研究和培养独立临床研究的技能提供丰富的环境。认知缺陷和学业成绩差是SCD学生独立的主要障碍。超过40%的SCD学生将不及格或接受特殊教育，大约15%的学生将在四年内高中毕业。这项建议将包括三项研究。第一项研究将扩大她在患有SCD和中风的学生中进行的教育康复试点试验，将患有SCD和严重记忆缺陷(&gt；1 SD低于预期平均年龄)的学生包括在内(n=30)。每个学生将随机接受两年的辅导或辅导加上记忆训练技能，以检验以下假设：通过80%的完成该计划的学生来衡量，针对SCD和认知障碍的教育康复计划是可行的。将进行二次分析，以确定康复计划在记忆测量方面的效果。申请人已在SITT启动了两项辅助研究。以下假设将在筛查无症状脑梗塞的镰状细胞性贫血(SCA)学生中进行检验：在SCA学生中，较低的SES将导致较低的教育程度低于SES和SCA较高的学生。第二项辅助研究将在随机接受输血治疗或观察三年的SCA患者中检验以下假设：接受输血治疗的SCA患者和接受输血治疗的无症状中风患者相比，没有接受输血治疗的无症状中风患者的学习成绩会有所提高。通过完成这些研究所获得的知识和技能将使申请者能够制定基于证据的策略，以提高SCD学生的教育水平。

DESCRIPTION (provided by applicant): The purpose of this proposal is to support the applicant in becoming an independent clinical investigator with expertise in improving educational attainment in children with sickle cell disease (SCD). The applicant will collaborate with her mentor and the multi-disciplinary SCD stroke research team at Washington University School of Medicine. The SCD stroke research team coordinates the 24 site multi-center Silent Infarct Transfusion Trial (SITT), and the applicant is the local principal investigator. She will gain experience with both her existing independent pilot trial and her investigator-initiated ancillary studies to SITT. These activities will provide a rich environment for conducting patient-oriented research and developing the skills for independent clinical investigation. Cognitive deficits and poor academic attainment are major barriers to independence in students with SCD. Over 40% of students with SCD will fail a grade or receive special education, and approximately 15% will graduate from high school in four years. This proposal will include three studies. The first study will expand her pilot trial for education rehabilitation in students with SCD and strokes to include students with SCD and significant memory deficits (> 1 SD below the age expected mean)(n=30). Each student will be randomized to receive two years of either tutoring or tutoring plus memory training skills to test the following hypothesis: An education rehabilitation program for students SCD and cognitive deficits is feasible, measured by > 80% of the students completing the program. A secondary analysis will be performed to determine the efficacy of the rehabilitation program on memory measures. The applicant has initiated two ancillary studies in SITT. The following hypothesis will be tested in students with sickle cell anemia (SCA) screened for silent cerebral infarcts: In students with SCA, a lower SES will result in a lower educational attainment than students with a high SES and SCA. The second ancillary study will test the following hypothesis in students with SCA who are randomized to either transfusion therapy or observation for three years: Students with SCA and silent strokes who receive blood transfusion therapy will have an improvement in their academic achievement when compared to students with silent strokes who are not transfused. The knowledge and skills acquired from completing these studies will allow the applicant to develop evidence based strategies to improve educational attainment in students with SCD.