BACKGROUND Emerging evidence suggests that endovascular thrombectomy is beneficial for treatment of childhood stroke, but the safety and effectiveness of endovascular thrombectomy has not been compared with best medical treatment. We aimed to prospectively analyse functional outcomes of endovascular thrombectomy versus best medical treatment in children with intracranial arterial occlusion stroke. METHODS In this prospective registry study, 45 centres in 12 countries across Asia and Australia, Europe, North America, and South America reported functional outcomes for children aged between 28 days and 18 years presenting with arterial ischaemic stroke caused by a large-vessel or medium-vessel occlusion who received either endovascular thrombectomy plus best medical practice or best medical treatment alone. Intravenous thrombolysis was considered part of best medical treatment and therefore permitted in both groups. The primary outcome was the difference in median modified Rankin Scale (mRS) score between baseline (pre-stroke) and 90 days (±10 days) post-stroke, assessed by the Wilcoxon rank test (α=0·05). Efficacy outcomes in the endovascular thrombectomy and best medical treatment groups were compared in sensitivity analyses using propensity score matching. The Save ChildS Pro study is registered at the German Clinical Trials Registry, DRKS00018960. FINDINGS Between Jan 1, 2020, and Aug 31, 2023, of the 241 patients in the Save ChildS Pro registry, 208 were included in the analysis (115 [55%] boys and 93 [45%] girls). 117 patients underwent endovascular thrombectomy (median age 11 years [IQR 6-14]), and 91 patients received best medical treatment (6 years [3-12]; p<0·0001). The median Pediatric National Institutes of Health Stroke Scale (PedNIHSS) score on admission was 14 (IQR 10-19) in the endovascular thrombectomy group and 9 (5-13) in the best medical treatment group (p<0·0001). Both treatment groups had a median pre-stroke mRS score of 0 (IQR 0-0) at baseline. The change in median mRS score between baseline and 90 days was 1 (IQR 0-2) in the endovascular thrombectomy group and 2 (1-3) in the best medical treatment group (p=0·020). One (1%) patient developed a symptomatic intracranial haemorrhage (this patient was in the endovascular thrombectomy group). Six (5%) patients in the endovascular thrombectomy group and four (5%) patients in the best medical treatment group had died by day 90 (p=0·89). After propensity score matching for age, sex, and PedNIHSS score at hospital admission (n=79 from each group), the change in median mRS score between baseline and 90 days was 1 (IQR 0-2) in the endovascular thrombectomy group and 2 (1-3) in the best medical treatment group (p=0·029). Regarding the primary outcome for patients with suspected focal cerebral arteriopathy, endovascular thrombectomy (n=18) and best medical treatment (n=33) showed no difference in 90-day median mRS scores (2 [IQR 1-3] vs 2 [1-4]; p=0·074). INTERPRETATION Clinical centres tended to select children with more severe strokes (higher PedNIHSS score) for endovascular thrombectomy. Nevertheless, endovascular thrombectomy was associated with improved functional outcomes in paediatric patients with large-vessel or medium-vessel occlusions compared with best medical treatment. Future studies need to investigate whether the positive effect of endovascular thrombectomy is confined to older and more severely affected children. FUNDING None.

中文翻译：

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儿童中风的血管内血栓切除术 （Save ChildS Pro）：一项国际、多中心、前瞻性登记研究。


背景 新出现的证据表明，血管内血栓切除术有益于治疗儿童中风，但血管内血栓切除术的安全性和有效性尚未与最佳药物治疗进行比较。我们旨在前瞻性分析颅内动脉闭塞性卒中患儿血管内血栓切除术与最佳药物治疗的功能结局。方法 在这项前瞻性注册研究中，亚洲和澳大利亚、欧洲、北美和南美洲 12 个国家的 45 个中心报告了 28 天至 18 岁因大血管或中血管闭塞引起的动脉缺血性卒中接受血管内血栓切除术加最佳医疗实践或单独接受最佳药物治疗的儿童的功能结局。静脉溶栓被认为是最佳药物治疗的一部分，因此两组都允许。主要结局是基线 （卒中前） 和卒中后 90 天 （±10 天） 之间中位改良 Rankin 量表 （mRS） 评分的差异，通过 Wilcoxon 秩检验 （α=0·05） 评估。使用倾向评分匹配在敏感性分析中比较血管内血栓切除术组和最佳药物治疗组的疗效结果。Save ChildS Pro 研究已在 DRKS00018960 年德国临床试验注册处注册。发现 在 2020 年 1 月 1 日至 2023 年 8 月 31 日期间，在 Save ChildS Pro 登记处的 241 名患者中，有 208 名被纳入分析（115 名 [55%] 男孩和 93 名 [45%] 女孩）。117 例患者接受了血管内血栓切除术 （中位年龄 11 岁 [IQR 6-14]），91 例患者接受了最佳药物治疗 （6 年 [3-12];p<0·0001）。 入院时儿科美国国立卫生研究院卒中量表 （PedNIHSS） 评分中位为血管内血栓切除术组 14 （IQR 10-19），最佳药物治疗组 （p<0·0001） 9 （5-13）。两个治疗组基线时卒中前 mRS 评分的中位值为 0 （IQR 0-0）。基线和 90 天之间中位 mRS 评分的变化在血管内血栓切除术组中为 1 （IQR 0-2），在最佳药物治疗组中为 2 （1-3） （p=0·020）。1 例 （1%） 患者出现症状性颅内出血 （该患者为血管内血栓切除术组）。血管内血栓切除术组有 6 例 （5%） 患者和最佳药物治疗组有 4 例 （5%） 患者在 90 天时死亡 （p=0·89）。入院时年龄、性别和 PedNIHSS 评分倾向评分匹配后 （每组 n=79），基线和 90 天之间中位 mRS 评分的变化在血管内血栓切除术组中为 1 （IQR 0-2），在最佳药物治疗组中为 2 （1-3） （p=0·029）。关于疑似局灶性脑动脉病患者的主要结局，血管内血栓切除术 （n=18） 和最佳药物治疗 （n=33） 显示 90 天中位 mRS 评分无差异 （2 [IQR 1-3] vs 2 [1-4];p=0·074）。解释 临床中心倾向于选择中风更严重 （PedNIHSS 评分较高） 的儿童进行血管内血栓切除术。然而，与最佳药物治疗相比，血管内血栓切除术与大血管或中血管闭塞儿科患者功能结局的改善相关。未来的研究需要调查血管内血栓切除术的积极影响是否仅限于年龄较大和受影响更严重的儿童。资金 无。