1 Centre for Prevention of Stroke and Dementia, Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, UK.
2 Department of Neurosciences, Biomedicine and Movement Sciences, University of Verona, Verona, Italy.
Int J Stroke. 2017 Aug;12(6):580-588. doi: 10.1177/1747493017706189. Epub 2017 Apr 25.
Background Sickle cell disease (SCD) is one of the most common causes of stroke in children worldwide. Based on the results of the Stroke Prevention Trial in Sickle Cell Anemia (STOP), annual transcranial Doppler ultrasound (TCD) screening for affected children is standard practice. However, the need for TCD surveillance programs could override the accuracy of the screening, affecting the correct stratification of stroke risk and subsequent clinical management of the target population. Aims To shed light on this issue, a systematic review of the literature on TCD screening for children and adolescents with SCD was carried out (CRD42016050549), according to a list of clinically relevant questions, with a particular focus on screening practices in European countries. Quality of the evidence was rated using the grading of recommendations assessment, development and evaluation. Summary of review Thirty-three studies published in English or French were included (5 randomized controlled trials, 8 experimental non-randomized, and 20 observational studies). The quality of the retrieved evidence ranged between low and high, but was rated as moderate or high most of the times. TCD is effective as a screening tool for the primary prevention of stroke in SCD children. There is no high-quality evidence on the effectiveness of alternative screening methods, such as imaging-TCD with or without angle correction or magnetic resonance angiography. No evidence was found on effectiveness of the screening on children on hydroxyurea and with genotypes other than HbSS and HbS/β0. No European data were found on screening rates or adherence of screening practices to the STOP protocol. Conclusions High-quality studies on alternative screening methods that are currently used in real-world practice, and on screening applicability to specific subgroups of patients are urgently needed. Considering the low awareness of the disease in European countries and the lack of data on screening practices and adherence, clinicians need up-to-date guidelines for more uniform and evidence-based surveillance of children with SCD.
背景 镰状细胞病（SCD）是全球儿童中风的最常见原因之一。基于镰状细胞贫血症中风预防试验（STOP）的结果，对受影响的儿童进行年度经颅多普勒超声（TCD）筛查是标准做法。然而，TCD 监测计划的必要性可能会影响筛查的准确性，从而影响中风风险的正确分层以及目标人群的后续临床管理。
目的 为了解决这个问题，对 TCD 筛查 SCD 儿童和青少年的文献进行了系统评价（CRD42016050549），根据一系列临床相关问题，特别关注欧洲国家的筛查实践。使用推荐评估、制定和评估的分级来评估证据的质量。
综述总结 共纳入了 33 项以英语或法语发表的研究（5 项随机对照试验、8 项实验性非随机对照试验和 20 项观察性研究）。检索到的证据质量在低到高之间，但大多数情况下被评为中度或高度。TCD 是 SCD 儿童中风一级预防的有效筛查工具。目前没有高质量的证据表明替代筛查方法（如带或不带角度校正的 TCD 成像或磁共振血管造影）的有效性。也没有证据表明该筛查对羟基脲治疗的儿童或基因型不是 HbSS 和 HbS/β0 的儿童有效。没有发现关于欧洲国家的筛查率或筛查实践对 STOP 方案的依从性的相关数据。
结论 迫切需要关于目前在实际中使用的替代筛查方法的高质量研究，以及关于筛查对特定亚组患者适用性的研究。鉴于欧洲国家对该病的认识不足，且缺乏关于筛查实践和依从性的数据，临床医生需要最新的指南，以便对 SCD 儿童进行更统一和基于证据的监测。
