Research Appraisals
Evidence-based critical appraisals of the latest medical research, systematically evaluated using Oxford CEBM methodology.
Showing 6 appraisals
Neurosurgical review
Development and external validation of a machine learning model for predicting postoperative hydrocephalus in 1,073 posterior fossa tumor patients
Postoperative hydrocephalus is a common complication following posterior fossa tumor resection, affecting 7-40% of patients. Although preoperative cerebrospinal fluid (CSF) diversion may be required in selected patients with hydrocephalus, decisions remain individualized in routine neurosurgical practice. We therefore developed and externally validated a model to provide supplementary preoperative risk stratification using routinely available variables. We retrospectively analyzed 1,073 patients following resection of posterior fossa tumors (PFTs) treated at five tertiary centers between 2013 and 2024, dividing them into a development cohort (n = 854) and an external validation cohort (n = 219). We initially screened 30 perioperative variables from the multicenter dataset and then selected a clinically implementable model restricted to variables available before tumor resection. Feature importance was ranked using Shapley additive explanations (SHAP), and seven distinct machine learning (ML) algorithms were evaluated. Model performance was comprehensively measured using the area under the receiver operating characteristic curve (AUC), sensitivity, specificity, precision-recall curves, and decision curve analysis. The final clinically implementable model included three preoperative variables: Evans index, tumor-fourth ventricle relationship, and preoperative cerebrospinal fluid diversion status. In the external validation cohort, the support vector machine model showed good external discrimination, with an area under the receiver operating characteristic curve of 0.877, accuracy of 81.3%, sensitivity of 80.8%, and specificity of 81.7%. Logistic regression achieved comparable discrimination. Calibration assessment suggested dataset shift between the development and external validation cohorts, indicating that absolute predicted probabilities should be interpreted cautiously in populations with different baseline risks. A concise three-variable preoperative model showed good external discrimination for clinically relevant postoperative hydrocephalus after posterior fossa tumor resection. The model may support preoperative risk communication and postoperative surveillance planning, but should not be used as a stand-alone indication for cerebrospinal fluid diversion. Prospective validation and local recalibration are warranted before routine clinical implementation.
31 July 2026
Read appraisal →Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery
Non-operative management of traumatic occipitocervical distraction injuries in children: a systematic review
PURPOSE: Existing guidelines for the management of atlanto-occipital dislocation (AOD) recommend occipitocervical fusion (OCF) for these low frequency, high-consequence injuries. Recent literature has questioned this guidance and provided case examples of successful non-operative management of a more inclusive and broadly defined spectrum of injuries to the occipitocervical junction (OCJ) termed occipitocervical distraction injuries (OCDI). A systematic review of existing literature on this topic would inform efforts to investigate the safety and efficacy of non-operative management of these injuries. METHODS: We performed a systematic review based on preferred reporting items for systematic reviews (PRISMA) guidelines of English language publications to define and map literature related to non-operative management of pediatric traumatic OCDI. RESULTS: We identified 40 patients in 21 manuscripts meeting defined inclusion and exclusion criteria. 82.5% of patients were successfully managed with non-operative management alone. No patient declined neurologically during orthotic treatment or in relation to crossover to surgical management through a minimum of 3-month follow-up. CONCLUSIONS: This review supports the contention that non-operative management is both safe and effective in the management of OCDI. Further research to develop standardized diagnostic and treatment protocols for non-operative management of OCDI through registries and multi-center research efforts is needed.
31 July 2026
Read appraisal →Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery
Genetics versus environment in the pathophysiology of sagittal synostosis
PURPOSE: This systematic review aims to provide an updated overview of the relative contribution of germline genetic and environmental factors to the pathophysiology of sagittal craniosynostosis (sCS). METHODS: Using the PubMed database in accordance with Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines, relevant studies addressing genetic and/or environmental determinants of sCS in paediatric patients were systematically reviewed. RESULTS: A total of 1238 records were identified, of which 97 studies met inclusion criteria after full-text review. Overall, 25,877 patients were included, with 11,086 diagnosed with sCS (42.8%). Germline genetic variants potentially associated with craniosynostosis were reported in 251 patients, accounting for 12.6% of genetically tested individuals with sCS. Identified variants were distributed across 125 genes, with recurrent findings in 25 genes. Environmental and perinatal factors were more consistently reported across large population-based and case-control studies. Frequently associated factors included male sex, advanced maternal age, maternal smoking, thyroid dysfunction, fertility treatments, foetal constraint, prematurity, and abnormal birth weight, although effect sizes varied across studies. CONCLUSION: Identifiable germline genetic causes appear to account for only a minority of sCS cases, whereas epidemiological evidence suggests that environmental and perinatal influences may also contribute to disease pathogenesis. Nevertheless, most cases lack identifiable germline mutations in key signalling pathways, and the available evidence does not support either genetic or environmental factors as individually deterministic drivers of disease development. Overall, the evidence synthesized in this review supports a multifactorial model for sCS, in which rare genetic susceptibility and non-genetic influences likely interact in the etiopathogenesis of the condition rather than reflecting a single dominant aetiology. However, heterogeneity across studies, differences in genetic testing methodologies, potential bias in exposure assessment, and the limited availability of integrative analyses restrict definitive conclusions regarding the relative contribution of these factors.
19 July 2026
Read appraisal →Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery
A systematic review of long-term outcomes (>10-year follow-up) of paediatric craniopharyngioma
PURPOSE: Adamantinomatous craniopharyngiomas (ACP) in children are rare benign tumours. Relatively few publications have addressed the long-term impact of this tumour and its treatment. We undertake a systemic review of the existing literature aiming to define the outcome of ACP beyond 10 years' follow-up. METHODS: Ovid MEDLINE, Embase and Google Scholar were used to search the literature. The search criteria were children (aged under 18 at diagnosis), any intervention for and outcomes of ACP. Publications with less than 10 years' follow-up and single case reports were excluded. RESULTS: Twenty-one studies were included reporting on 1152 children, with a mean age of 8.8 years at diagnosis. Mean follow-up was 14 years. The most common presenting symptom was headache. The mean overall survival at 5, 10, 15 and 20 years post-diagnosis was 91.6%, 84.4%, 77.5% and 68.0%, respectively. In children undergoing gross total resection only or radiotherapy only, survival was higher than with combined treatments. Seventy-five percent of children had endocrine dysfunction after intervention; the most prevalent was hypothyroidism (81.9%). The prevalence of endocrinopathies was similar across all treatment groups. Lower QoL was reported than the general population, predominantly related to the effects of hypothalamic injury. One in ten patients suffered a late complication related to radiotherapy with vasculopathy being the most prevalent. CONCLUSIONS: The long-term morbidity and mortality for paediatric ACP remain high. The impact from hypothalamic injury cannot be understated and is likely the cause for this finding. As surgical treatment advances, less hypothalamic injury may arise and improved outcomes may follow. Gross total resection, if felt achievable without hypothalamic injury, should form the mainstay of treatment due to the late radiotherapy-related complications that arise.
10 July 2026
Read appraisal →The New England journal of medicine
Decompression with or without Duraplasty for Chiari I and Syringomyelia
BACKGROUND: In children with Chiari type I malformation and syringomyelia, neurosurgical posterior fossa decompression (PFD) provides clinical improvement, but whether duraplasty (incising the dura and placing a dural graft) improves outcomes is unclear. METHODS: We conducted a multicenter, cluster-randomized, controlled trial of PFD with duraplasty (PFD-D) as compared with PFD alone. Persons 21 years of age or younger with cerebellar tonsillar ectopia of at least 5 mm and a maximum syrinx diameter of 3.0 to 9.9 mm were enrolled at 38 centers. Centers were cluster-randomized: all the participants within each center underwent the same intervention. The primary outcome was surgical complications within 6 months. Secondary outcomes were clinical improvement, syrinx reduction, and repeat decompression at 10 to 24 months and the change in overall health-related quality of life at 6 to 24 months. RESULTS: A total of 162 participants were included in the trial, of whom 78 were assigned to undergo PFD-D and 84 to undergo PFD alone. The percentage of participants with complications within 6 months was 14% with PFD-D and 6% with PFD (adjusted odds ratio, 2.59; 95% confidence interval [CI], 0.86 to 7.84; P = 0.11). At 24 months, the percentage of participants with clinical improvement was 58% with PFD-D and 46% with PFD; the mean (±SD) syrinx reduction was 3.08±2.33 mm and 1.22±1.79 mm, respectively; and the percentage of participants with repeat decompression was 3% and 14%. Changes in health-related quality of life were similar in the two groups. CONCLUSIONS: The percentage of participants with surgical complications did not differ significantly between those who underwent PFD-D and and those who underwent PFD alone. Larger trials are needed to determine the relative benefits and risks of these two procedures. (Funded by the Patient-Centered Outcomes Research Institute and others; ClinicalTrials.gov number, NCT02669836.).
29 May 2026
Read appraisal →Neurosurgical review
The efficacy of ventriculosubgaleal shunt in managing neonatal hydrocephalus due to intraventricular hemorrhage: a systematic review and meta-analysis
Intraventricular hemorrhage (IVH) is a severe complication in premature neonates, occurring in 25%-30% of cases and often leading to posthemorrhagic hydrocephalus (PHH). When blood clots in the cerebrospinal fluid (CSF), preventing permanent shunt placement, temporary interventions are considered. Ventriculosubgaleal shunt (VSGS) utilizes the subgaleal space to absorb and drain excess CSF, reducing infection risk and allowing hydrocephalus control until the neonate reaches an appropriate weight and CSF clarity. This systematic review and meta-analysis evaluate the safety and efficacy of VSGS in treating neonatal PHH. A systematic review was conducted using Medline, Embase, and Web of Science following Cochrane and PRISMA guidelines. Eligible studies included those with ≥ 4 neonates. The primary outcomes analyzed were VSGS-related infection, VSGS revision, VSGS catheter migration, catheter obstruction, VSGS-related CSF leakage, permanent ventriculoperitoneal shunt (VPS) placement, overall mortality, and procedure-related mortality. A total of nineteen studies, encompassing 562 neonates, were included in our analysis. The pooled VSGS-related infection rate was 9% (95%CI: 5% to 12%). The need for VSGS revision was observed in 4% of cases (95%CI: 0% to 8%). The catheter obstruction rate was 2% (95% CI: 0% to 5%), while VSGS catheter migration occurred in 1% of cases (95%CI: 0% to 5%). VSGS-related CSF leakage was reported in 6% of neonates (95%CI: 3% to 9%). Permanent VPS placement was required in 75% of patients (95%CI: 67% to 82%). The overall mortality rate was 10% (95%CI: 4% to 16%), and the procedure-related mortality rate was 1% (95%CI: 0% to 2%). This systematic review and meta-analysis identified VSGS as a safe and effective option for treating hydrocephalus caused by IVH in premature neonates.
10 May 2026
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