Prevention of Stroke after STOP, A Retrospective Chart Review

描述(由申请人提供)：STOP和STOP II研究提供了第一个由I类证据支持的针对镰状细胞病的全面中风预防策略。虽然TCD筛查和预防性输血的策略在卒中预防镰刀细胞性贫血(STOP试验)中非常有效，如果即使在输血30个月或更长时间后取消这种疗法，也可以观察到恢复到高风险或卒中的情况(STOP II)，但在试验完成后，这种方法在现实世界中的有效性尚不清楚。该项目利用为获得STOP和STOP II数据而花费的巨大财力和人力资源，以及由此产生的数据库(可供研究人员使用)，通过深入分析试验后研究参与者关于中风预防行为(TCD和输液重新筛查)和中风以及其他重要结果的医疗记录，获得有关3400多名儿童的信息。至关重要的两个结果：1)中风--在这方面，试验结束后发生的中风是由于筛查失败(没有TCD，或者时机选择不当或符合筛查计划)，或者治疗失败(不建议或接受输血，或者尽管符合充分的输血要求，仍有突破性的中风)，以及如何最好地利用这些信息来改进中风的预防实践；2)转为高危TCD。数据库包含3000多名儿童的初步(异常前)TCD数据，并根据试验期间的重新筛选发布了转换的概率。假设试验后临床实践中不同程度的重新筛选，并在试验后发现新的异常；预测异常TCD发生率的试验后模型是否确认停止或与停止时观察到的有实质性差异？在任何一种情况下，试验后的经验都将与STOP数据相结合，以制定更有力的基于证据的临床指南，用于TCD的重新筛查(如果第一次TCD没有表明需要治疗)，以确保对中风风险进行充分的监测。STOP和STOP II是在专门的儿科SCD中心进行的，将研究过渡到成人提供者系统对中风预防的影响，评估态度，并使用这些数据为进一步的研究和教育提供建议。总体而言，该项目提供了大量的新信息，将确保在Stop and Stop II中所做的主要投资被最大限度地用于现实世界的应用，以预防患有SCD的儿童中风。公共卫生相关性：这项调查的目标是获得和分析能够提高镰状细胞病儿童和年轻人的护理质量的信息，特别是在预防中风方面。对信息的分析将直接影响TCD筛查指南。这些定性数据还将提供对竞争疗法的价值以及治疗选择和中风事件的影响的洞察，并最终提供信息，帮助预防患有镰状细胞病的年轻人发生毁灭性的中风。

DESCRIPTION (provided by applicant): The STOP and STOP II studies provided the first comprehensive stroke prevention strategy in Sickle Cell Disease supported by Class I evidence. While the strategy of TCD screening and prophylactic transfusion was very efficacious in the Stroke Prevention in Sickle Cell Anemia (STOP trial), and reversion to high risk or stroke was observed if this therapy is removed even after 30 or more months of transfusion (STOP II) it is unclear how effective this approach is in real world practice after the trials were completed. This project leverages the enormous financial and human resources expended to obtain STOP and STOP II data, and their resulting data bases (available to the investigators) with information on over 3400 children, by making an in-depth analysis of the medical records of study participants in the post trial period regarding stroke prevention behaviors (re-screening with TCD and transfusion) and stroke as well as other important outcomes. Two outcomes are of paramount interest: 1) stroke - in this respect were the strokes that took place after the trial the result of screening failures (no TCD or inadequate timing or compliance with screening programs) or treatment failures (transfusion not recommended or accepted or breakthrough stroke despite compliance with adequate transfusion) and how can this information be best used to improve stroke prevention practices; 2) conversion to high risk TCD. The data bases contain preliminary (pre abnormal) TCD data on over 3000 children, and probabilities for conversion have been published from STOP based on rescreening during the trail. Assuming varying degrees of rescreening in clinical practice post trial, and new abnormals identified post trial; does the post trial model predicting the incidence of abnormal TCD confirm STOP or vary substantially from what was observed in STOP? In either case post trial experience will be combined with STOP data to formulate stronger evidence based clinical guidelines for re screening with TCD (if the first TCD does not indicate need for treatment) to insure adequate surveillance for stroke risk. STOP and STOP II were carried out in specialized pediatric SCD centers, the impact of transition to adult provider systems on stroke prevention will be studied, attitudes assessed and these data used to inform recommendations for further research and education. Overall the project provides substantial new information that will insure the major investment made in STOP and STOP II is used to best advantage in real world applications to prevent stroke in children with SCD. PUBLIC HEALTH RELEVANCE: The goal of this investigation is to obtain and analyze information that can improve the quality of care for children and young adults with sickle cell disease especially in the area of preventing stroke. The analysis of information will directly influence guidelines for TCD screening. These qualitative data will also provide insight into the value of competing therapies as well as the impact of treatment options and stroke events, and ultimately provide information to help prevent devastating stroke in young persons with Sickle Cell Disease.