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.

Clinic for Radiology Department for Interventional Neuroradiology University of Münster Münster Germany

Department of Diagnostic and Interventional Neuroradiology Hospital Bremen Mitte Bremen Ost Bremen Germany; 1st Department of Radiology School of Medicine National and Kapodistrian University of Athens Areteion Hospital Athens Greece

Department of Diagnostic and Interventional Neuroradiology University Medical Center Hamburg Eppendorf Hamburg Germany

Department of Interventional Neuroradiology Careggi University Hospital Florence Italy

Department of Medical Imaging Children's Hospital at Westmead Sydney NSW Australia; Children's Hospital at Westmead Clinical School Faculty of Medicine and Health University of Sydney Sydney NSW Australia

Department of Neurology and Department of Pediatrics University of California San Francisco San Francisco CA USA

Department of Neurology and Neurogeriatrics University Clinic of the Ruhr Universität Bochum Minden Germany

Department of Neurology Baylor College of Medicine Houston TX USA

Department of Neurology Comprehensive Stroke Center 2nd Faculty of Medicine Charles University and Motol University Hospital Prague Czech Republic

Department of Neurology Johns Hopkins School of Medicine Baltimore MD USA

Department of Neurology Massachusetts General Hospital Harvard Medical School Boston MA USA

Department of Neurology University Hospital Knappschaftskrankenhaus Ruhr University Bochum Bochum Germany

Department of Neurology University of Texas Rio Grande Valley Harlingen TX USA

Department of Neuroradiology Aachen University Aachen Germany

Department of Neuroradiology Alfried Krupp Krankenhaus Essen Germany

Department of Neuroradiology Heidelberg University Hospital Heidelberg Germany

Department of Neuroradiology Hospital de Pediatría J P Garrahan Buenos Aires Argentina

Department of Neuroradiology LMU University Hospital LMU Munich Munich Germany

Department of Neuroradiology Medical University of Vienna Vienna Austria

Department of Neuroradiology Saarland University Hospital Homburg Germany

Department of Neuroradiology University Hospital Basel Basel Switzerland

Department of Neuroradiology University Hospital Basel Basel Switzerland; Department of Diagnostic and Interventional Neuroradiology University Medical Center Hamburg Eppendorf Hamburg Germany; Department of Radiology and Neuroradiology Stadtspital Zürich Zürich Switzerland

Department of Neuroradiology University Medical Center of the Johannes Gutenberg University Mainz Germany

Department of Neuroradiology University of Marburg Marburg Germany

Department of Neurosurgery Barrow Neurological Institute Phoenix AZ USA; Department of Radiology Phoenix Children's Hospital Phoenix AZ USA

Department of Neurosurgery Emory University School of Medicine Atlanta GA USA

Department of Paediatric Neurology Comprehensive Stroke Center 2nd Faculty of Medicine Charles University and Motol University Hospital Prague Czech Republic; Department of Neurology Comprehensive Stroke Center 2nd Faculty of Medicine Charles University and Motol University Hospital Prague Czech Republic

Department of Paediatrics Khoo Teck Puat National University Children's Medical Institute National University Hospital Singapore

Department of Pediatrics and Adolescent Medicine Eltern Kind Zentrum Prof Hess Klinikum Bremen Mitte Bremen Germany

Department of Pediatrics and Department of Neurology Nationwide Children's Hospital and The Ohio State University Columbus OH USA

Department of Radiology and Neuroradiology Sana Kliniken Duisburg Duisburg Germany; Department of Diagnostic and Interventional Neuroradiology Medical School Hannover Hannover Germany

Department of Radiology and Neuroradiology Stadtspital Zürich Zürich Switzerland

Department of Radiology and Neuroradiology University Hospital Halle Halle Germany

Department of Radiology and Neuroradiology University Medical Center Schleswig Holstein Kiel Germany; Department of Neuroradiology University Medical Center Schleswig Holstein Lübeck Germany

Department of Radiology LMU University Hospital LMU Munich Munich Germany

Diagnostic and Interventional Neuroradiology Department University Hospital of Tours Tours France; French Center for Pediatric Stroke Paris France

Division of Child and Adolescent Neurology Department of Pediatrics The University of Texas McGovern Medical School Houston TX USA

Division of Child Neurology Department of Neurology Stanford University Stanford CA USA

Division of Critical Care Medicine and Division of Pediatric Neurology Department of Pediatrics Nationwide Children's Hospital and The Ohio State University Columbus OH USA

Division of Neuropaediatrics Development and Rehabilitation Department of Paediatrics Inselspital Bern University Hospital University of Bern Bern Switzerland

Division of Neuropaediatrics Development and Rehabilitation Department of Paediatrics Inselspital Bern University Hospital University of Bern Bern Switzerland; Graduate School for Health Sciences University of Bern Bern Switzerland

Division of Neuroradiology and Division of Neurointervention Hospital for Sick Children University of Toronto Toronto ON Canada

Division of Pediatric Neurology Department of Pediatrics University of Utah School of Medicine Salt Lake City UT USA

Division of Pediatric Neurology Oregon Health and Science University Portland OR USA

French Center for Pediatric Stroke Paris France; Department of Pediatric Radiology Faculté de Médecine Necker Children's Hospital Université Paris Descartes Sorbonne Paris Cité Paris France

Institute of Neuroradiology Medical Faculty and University Hospital Carl Gustav Carus Dresden University of Technology Dresden Germany

Institute of Radiology University Hospital Regensburg Regensburg Germany

Morozov Moscow Children Clinical Hospital Moscow Russia; Division of Neuroradiology and Division of Neurointervention Hospital for Sick Children University of Toronto Toronto ON Canada

Neuroendovascular Program Massachusetts General Hospital Harvard Medical School Boston MA USA

Neuroendovascular Program Massachusetts General Hospital Harvard Medical School Boston MA USA; Neurovascular Centre Divisions of Therapeutic Neuroradiology and Neurosurgery St Michael's Hospital University of Toronto ON Canada

Neuroradiological Clinic Katharinenhospital Klinikum Stuttgart Stuttgart Germany

Pediatric Thrombosis and Hemostasis Unit Dr von Hauner Children's Hospital LMU Munich Munich Germany

Research Center of Endovascular Neurosurgery Federal Center of Brain Research and Neuro Technologies of FMBA Moscow Russia; Morozov Moscow Children Clinical Hospital Moscow Russia

University Hospital LMU Munich Germany; Department of Radiology Texas Children's Hospital Baylor College of Medicine Houston TX USA

Comment In

Lancet Child Adolesc Health. 2024 Dec;8(12):844-845. doi: 10.1016/S2352-4642(24)00257-8. PubMed

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