Abstract Body: Background: The most common cause of arterial ischemic stroke (AIS) in previously healthy children, focal cerebral arteriopathy (FCA) can progress rapidly over days with worsening brain injury. A 2017 retrospective study of corticosteroid treatment for FCA changed practice at many pediatric stroke centers. To assess its impact, we compared FCA treatment rates and outcomes in FCA cohorts from the two Vascular effects of Infection in Pediatric Stroke studies.
Methods: The (VIPS II) study prospectively enrolled and centrally confirmed 205 children (29 days to 18 years) with AIS at 22 centers (North America, Australia), 12/2016-1/2022. Study neuroradiologist (M.W.) and pediatric vascular neurologist (H.J.F.) independently reviewed all baseline and follow-up brain and vascular imaging and clinical data to classify the AIS etiology using published definitions from the original VIPS I study and confirm recurrent strokes; disagreements were resolved through discussion. M.W. measured the FCA Severity Score (FCASS); higher scores reflect more severe disease. We compared the FCA patients in VIPS II to those in VIPS I (2010-2014), the prospective cohort study with similar methods.
Results: Of 75 children with definite arteriopathy enrolled in VIPS II, 43% had FCA, compared to 32% of 127 definite arteriopathy cases in VIPS I (Table). Treatment changed dramatically between the two studies, with 56% of VIPS II FCA cases receiving IV corticosteroids, compared to only 5% of FCA cases in VIPS I. The use of anticoagulation declined in favor of antiplatelets. Compared to VIPS I, the VIPS II FCA cases were more severe at baseline (median FCASS 6 versus 4; p=0.006). Among cases with short-term follow-up imaging, the increase in FCASS was not significantly different: median + 2 points in VIPS II (n=24) and +3 in VIPS I (n=33; p=0.76). The 12-month neurological outcomes were similar.
Conclusions: Treatment of FCA with corticosteroids increased dramatically between the VIPS I and VIPS II studies, reflecting a major shift in clinical practice on the basis of modest evidence. VIPS II cases were more severe at baseline, but we observed no significant difference in disease progression or neurological outcomes. Pediatric stroke centers should enroll FCA patients into ongoing FCA corticosteroid treatment trials—PASTA in Europe and Australia and FOCAS in North America—that will provide rigorous data regarding the effectiveness and safety of this intervention.