Educational Attainment in Sickle Cell Disease

描述（由申请人提供）： 本提案的目的是支持申请人成为一名独立的临床研究者，在提高镰状细胞病（SCD）儿童的教育程度方面具有专业知识。申请人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24个中心的多中心无症状输血试验（SITT），申请人是当地的主要研究者。她将从她现有的独立试点试验和她的制药商发起的SITT辅助研究中获得经验。这些活动将提供一个丰富的环境，进行以病人为导向的研究和发展的技能，独立的临床调查。认知缺陷和学业成绩差是SCD学生独立的主要障碍。超过40%患有SCD的学生将不及格或接受特殊教育，大约15%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教育康复的试点试验，以包括患有SCD和显著记忆缺陷的学生（低于预期平均年龄> 1 SD）（n=30）。每个学生将随机接受两年的辅导或辅导加记忆训练技能，以测试以下假设：学生SCD和认知缺陷的教育康复计划是可行的，通过> 80%的学生完成计划来衡量。将进行二次分析，以确定康复计划对记忆测量的疗效。申请者在SITT开展了两项辅助研究。以下假设将在筛查无症状脑梗死的镰状细胞性贫血（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比SES和SCA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学生中测试以下假设：与未接受输血的无症状中风学生相比，接受输血治疗的SCA和无症状中风学生的学业成绩将有所改善。从完成这些研究中获得的知识和技能将使申请人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.