Prevention of Stroke after STOP, A Retrospective Chart Review

停止后预防中风，回顾性图表回顾

• 批准号: 7699050
• 负责人: ROBERT J ADAMS
• 金额: $ 70.03万
• 依托单位: MEDICAL UNIVERSITY OF SOUTH CAROLINA
• 依托单位国家: 美国
• 财政年份: 2009
• 资助国家: 美国
• 起止时间: 2009-09-01 至 2013-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7699050
• 关键词: Adult; Area; Attitude; Behavior; Brain Infarction; Child; Childhood; Clinical; Clinical Trials; Data; Databases; Diagnosis; Education; Enrollment; Event; Failure; Frequencies; Goals; Guidelines; Human Resources; Incidence; Infarction; Investigation; Investments; Medical Records; Modeling; Outcome; Participant; Patients; Persons; Phase; Prevention strategy; Probability; Protocols documentation; Provider; Publishing; Quality of Care; Recommendation; Research; Research Personnel; Risk; Screening Result; Screening procedure; Sickle Cell Anemia; Stroke; Stroke prevention; Symptoms; System; Testing; Time; Transfusion; Transient Ischemic Attack; Treatment Failure; Validation; base; care systems; cerebrovascular; clinical practice; cohort; evidence base; experience; high risk; improved; insight; interest; prevent; programs; prophylactic; public health relevance; real world application; trial comparing; young adult

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.

描述（由申请人提供）：STOP 和 STOP II 研究提供了第一个由 I 类证据支持的镰状细胞病全面中风预防策略。虽然 TCD 筛查和预防性输血策略在镰状细胞性贫血中风预防（STOP 试验）中非常有效，并且即使在输血 30 个月或更长时间后取消这种治疗（STOP II），也可以观察到恢复到高风险或中风的情况，但在试验完成后，尚不清楚这种方法在现实世界实践中的有效性。该项目利用了为获取 STOP 和 STOP II 数据而花费的大量财力和人力资源，以及由此产生的数据库（可供研究人员使用），其中包含超过 3400 名儿童的信息，对研究参与者在试验后阶段有关中风预防行为（TCD 和输血重新筛查）和中风以及其他重要结果的医疗记录进行深入分析。两个结果是最重要的：1）中风——在这方面，试验后发生的中风是否是筛查失败（没有 TCD 或时机不当或不遵守筛查计划）或治疗失败（不推荐或不接受输血或尽管遵守充分输血但发生突发性中风）的结果，以及如何最好地利用这些信息来改善中风预防实践； 2) 转换为高风险 TCD。该数据库包含 3000 多名儿童的初步（异常前）TCD 数据，并且基于试验期间的重新筛查，已从 STOP 发布了转换概率。假设试验后在临床实践中进行不同程度的重新筛查，并在试验后发现新的异常情况；预测异常 TCD 发生率的试验后模型是否证实了 STOP 或与 STOP 中观察到的情况有很大差异？无论哪种情况，试验后经验都将与 STOP 数据相结合，制定更有力的基于证据的临床指南，用于 TCD 重新筛查（如果第一个 TCD 不表明需要治疗），以确保对中风风险进行充分监测。 STOP 和 STOP II 在专门的儿科 SCD 中心进行，将研究过渡到成人提供系统对中风预防的影响，评估态度，并将这些数据用于为进一步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 STOP 和 STOP II 的主要投资在现实世界的应用中得到最大的利用，以预防患有 SCD 的儿童中风。公共卫生相关性：本次调查的目的是获取和分析可以提高镰状细胞病儿童和年轻人护理质量的信息，特别是在预防中风方面。信息分析将直接影响 TCD 筛查指南。这些定性数据还将深入了解竞争疗法的价值以及治疗方案和中风事件的影响，并最终提供信息以帮助预防患有镰状细胞病的年轻人发生毁灭性中风。