Background and Purpose-The Pediatric Stroke Outcome Measure (PSOM) is an objective, disease-specific outcome measure containing 115 test items suitable for newborn to adult ages. The PSOM measures neurological deficit and function across 5 subscales: right sensorimotor, left sensorimotor, language production, language comprehension, and cognitive/behavior yielding a final 10-point deficit score. The goal of this study was to examine PSOM construct validity in measuring neurological outcome in pediatric stroke survivors and interrater reliability (IRR) for both prospective and retrospective scoring. Methods-For construct validity, PSOM subscale scores were correlated with scores on standardized neuropsychological measures matched by functional domain. We assessed IRR by comparing same-day "live" PSOM scores from 2 independent raters in 10 children (prospective IRR) and by comparing PSOM scores estimated from medical dictations across 5 raters in another 10 children (retrospective IRR). Key Words: outcome measures Ⅲ pediatric stroke Ⅲ PSOM Ⅲ validation study S troke during childhood is an increasingly recognized cause of significant long-term morbidity that creates a substantial burden of illness per affected individual. 1 Childhood stroke incidence is 5 to 8 per 100 000 children annually with approximately 50% ischemic including arterial ischemic stroke (AIS) or cerebral sinovenous thrombosis (CSVT). 2,3 Neonatal incidence is higher at 1 in 3000 to 5000 live births. 4,5 Resultant neurological deficits are reported in 50% to 90% of children and include motor, language, and cognitive deficits. 1,6 -10 Outcomes research and clinical trials require a feasible, valid, and reliable outcome measure in pediatric stroke. Results-WeOutcome measures developed for diffuse cerebral pediatric disorders including cerebral palsy, HIV, adrenoleukodystrophy, and head trauma 1 may be insensitive to the focal and sometimes mild deficits that result from pediatric stroke. The Rankin Scale, Barthel Index, and other adult stroke scales 11 are not applicable to young children due to their reliance on self-reporting and independence in activities of daily living. Finally, across the adult years, expected performance is similar in contrast to children in whom abilities change considerably with maturation from infancy to teenage years.The Pediatric Stroke Outcome Measure (PSOM) is an objective disease-specific measure of neurological recovery after childhood stroke. The PSOM was developed and implemented from 1994 in a prospective outcome study in our institutional Children's Stroke Clinic in Toronto, Canada. 1,12 Currently, the PSOM is also in use in the International Pediatric Stroke Study 13 and multiple other pediatric stroke studies. 8,14,15 Received October 3, 2011; accepted February 10, 2012 Methods Subject SelectionBeginning in 1994, children diagnosed with acute AIS or CSVT at the Hospital for Sick Children, Toronto, Canada, were prospectively enrolled in a longitudinal pediatric stroke outcome study. Children with outcome assess...
Neonatal cerebral sinovenous thrombosis is a frequent contributor to neonatal mortality and morbidity. Treatment is controversial and reported clinical outcomes vary widely. Newborns with radiologically confirmed neonatal cerebral sinovenous thrombosis from 1992–2009 were prospectively followed in our Children's Stroke Clinic for standardized outcomes, including the Pediatric Stroke Outcome Measure. Outcomes were available in 90/104 (87%) neonates. Early outcomes included cerebral sinovenous thrombosis-associated death (5) and thrombus propagation [15 (6 associated with new venous infarcts)]. Lack of anticoagulation predicted propagation (RR = 13, P = .0007). Complete thrombus recanalization occurred in 90% by 3 months. Late outcomes (median, 2.5 years) were epilepsy (15) and neurological disability (50), which included moderate-severe language (43), sensorimotor (38), and cognitive/behavioral (24) deficits. Overall, 61% had poor outcome (death/any deficit). Concurrent neurological comorbidity at diagnosis (odds ratio = 2.8, P = .029) predicted poor outcome. Clinical trials are urgently needed to establish more effective treatment strategies.
Objective: Vagus nerve stimulation (VNS) is a common treatment for medically intractable epilepsy, but response rates are highly variable, with no preoperative means of identifying good candidates. This study aimed to predict VNS response using structural and functional connectomic profiling. Methods: Fifty-six children, comprising discovery (n = 38) and validation (n = 18) cohorts, were recruited from 3 separate institutions. Diffusion tensor imaging was used to identify group differences in white matter microstructure, which in turn informed beamforming of resting-state magnetoencephalography recordings. The results were used to generate a support vector machine learning classifier, which was independently validated. This algorithm was compared to a second classifier generated using 31 clinical covariates. Results: Treatment responders demonstrated greater fractional anisotropy in left thalamocortical, limbic, and association fibers, as well as greater connectivity in a functional network encompassing left thalamic, insular, and temporal nodes (p < 0.05). The resulting classifier demonstrated 89.5% accuracy and area under the receiver operating characteristic (ROC) curve of 0.93 on 10-fold cross-validation. In the external validation cohort, this model demonstrated an accuracy of 83.3%, with a sensitivity of 85.7% and specificity of 75.0%. This was significantly superior to predictions using clinical covariates alone, which exhibited an area under the ROC curve of 0.57 (p < 0.008). Interpretation: This study provides the first multi-institutional, multimodal connectomic prediction algorithm for VNS, and provides new insights into its mechanism of action. Reliable identification of VNS responders is critical to mitigate surgical risks for children who may not benefit, and to ensure cost-effective allocation of health care resources. ANN NEUROL 2019;86:743-753 N early one-third of children with epilepsy are refractory to medications. 1,2 Persistent seizures are associated with mortality, disability, psychosocial isolation, and diminished quality of life. 3-6 Vagus nerve stimulation (VNS) is an effective, safe, and well-tolerated intervention for a subset of patients with treatment-resistant epilepsy. 7-10 Although the goal of VNS is not complete resolution of seizures, many children will show a significant reduction in seizure frequency, as well as a reduction in hospitalizations and psychosocial comorbidities. 11,12 View this article online at wileyonlinelibrary.com.
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