Prevention of Stroke after STOP, A Retrospective Chart Review

项目摘要 STOP和STOP II研究提供了第一个全面的中风预防策略， 镰状细胞病由I类证据支持。而中药筛选和 预防性输血在镰状细胞性贫血（STOP）的卒中预防中非常有效 试验），如果在30或更长时间后取消这种治疗，则观察到高风险或中风的逆转 几个月的输血（STOP II），目前还不清楚这种方法在真实的世界实践中的有效性， 审判已经结束。这个项目利用了巨大的财政和人力资源， 用于获得STOP和STOP II数据，以及由此产生的数据库（可供 调查人员）对3400多名儿童的信息，通过深入分析医疗 研究参与者在试验后阶段关于卒中预防行为的记录（重新筛选 TCD和输血）和卒中以及其他重要结局。两个结果是 最重要的利益：1）中风-在这方面，中风发生后，审判的结果， 筛选失败（无TCD或时间安排不当或不符合筛选计划）或治疗 失败（不推荐或不接受输血，或尽管遵守 以及如何最好地利用这些信息来改善卒中预防 2）转为高危中药治疗。数据库包含初步（异常前）TCD数据 超过3000名儿童，转换的概率已经从STOP发布，基于 在审判期间重新筛选。假设试验后在临床实践中进行不同程度的重新筛选，以及 试验后发现新的异常;试验后模型是否预测TCD异常的发生率 确认STOP或与STOP中观察到的有实质性差异？无论哪种情况，审判后 经验将与STOP数据相结合，以制定更强有力的循证临床指南 再次进行TCD筛查（如果第一次TCD未显示需要治疗），以确保充分 监测中风风险。STOP和STOP II在专门的儿科SCD中心进行， 将研究向成人提供者系统过渡对中风预防的影响， 这些数据被用来为进一步的研究和教育提供建议。整体 该项目提供了大量的新信息，将确保在STOP项目上的重大投资 STOP II在真实的世界中用于预防SCD儿童中风。

Project Abstract 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.