Research Appraisals
Evidence-based critical appraisals of the latest medical research, systematically evaluated using Oxford CEBM methodology.
Showing 7 appraisals
Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery
Primary Intracranial High Grade Congenital Glial Lesions (PIHGCL): A systematic review of case reports from 1985 to 2025
BACKGROUND: Among congenital intracranial space-occupying lesions, teratomas, gliomas, and neuroblastomas are more frequently reported. However, primary congenital glioblastoma (cGBM) and congenital anaplastic astrocytoma (cAA) are underrecognized entities. Their detection remains challenging due to nonspecific prenatal findings and limited awareness among pediatricians and obstetric care providers, contributing to delayed diagnosis and worsened outcomes. OBJECTIVE: To summarize the clinical presentations, diagnostic imaging characteristics, reported cases, histopathological features, and neuro-oncological management strategies of primary intracranial high grade congenital glial lesions (PIHGCL). MATERIALS AND METHODS: A comprehensive literature search following Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines was conducted in 2025 over PubMed and Google Scholar using keywords and Boolean operators, retrieving 35 studies (44 patients) with PIHGCL published 1985 to 2025. Quality assessment was done using Joanna Briggs Institute (JBI) tool. The data was analyzed descriptively as well as analytically to determine survival outcomes in treatment groups. RESULTS: A total of 28 studies (37 patients) discussed cGBM, and 7 studies (7 patients) described the occurrence of cAA. Most of the patients were infants i.e., 65.9% (29), presenting with macrocephaly in 27.7% (10) patients. Most of the lesions were intracerebral, 72.7% (32), lateralized equally to both sides, i.e., 46.5% (20). Surgical excision alone was done in 29.5% (13) patients, surgical excision with adjuvant chemotherapy was given to 43.2% (19) patients, chemotherapy alone in 6.8% (3) patients whereas 20.5% (9) underwent only conservative treatment. Kaplan Meir-analysis revealed a significant difference in survival in patients who received adjuvant treatment along with surgical excision in contrast to other treatment groups (χ2 = 7.83, df = 3, p = 0.05). CONCLUSION: PIHGCL presents during fetal or neonatal period with clinicopathological features distinct from adult and pediatric glioblastomas with distinct genetic etiology. Extent of resection is not a reliable marker of treatment outcomes in PIHGCL due to anatomical and physiological variations at the age of presentations. Adjuvant chemotherapy along with surgical resection results in improved patient survival.
11 June 2026
Read appraisal →Neurosurgical review
Management of pediatric brain arteriovenous malformation: a systematic review of retrospective studies
INTRODUCTION: Pediatric brain arteriovenous malformations (bAVMs) are a major cause of hemorrhagic stroke in children, yet management decisions rely largely on heterogeneous cohorts. Contemporary retrospective studies were systematically reviewed to summarize outcomes across treatment modalities and to characterize interstudy variability. METHODS: PubMed/MEDLINE was searched over the past decade for retrospective pediatric intracranial bAVM cohorts. Eligible studies reported management with observation/conservative care, endovascular embolization, microsurgical resection, stereotactic radiosurgery (SRS), or multimodality therapy. Screened studies had data extracted using a standardized form. Outcomes included obliteration, hemorrhage (first presentation and post-treatment), functional outcomes, complications, and recurrence after apparent cure. Treatment categories were harmonized into mutually exclusive groups for descriptive synthesis. RESULTS: Of 113 identified records, 20 retrospective cohort studies met the inclusion criteria. Hemorrhage was the most common presentation. Across cohorts reporting Spetzler-Martin (SM) grade, low-grade lesions (SM I-II) comprised 740 patients (44.1%), SM III 645 (38.4%), and SM IV-V 293 (17.5%). Treatment strategies varied substantially; among 1,973 classifiable patients, cohorts reported microsurgery-only, SRS-only, endovascular-only, multimodality, and conservative approaches. Definitions of obliteration and assessment methods were inconsistent (DSA-confirmed vs. MRI-based). Microsurgical series reported obliteration rates of 81%-100% in selected populations (unweighted mean ~ 93% across reporting cohorts), whereas SRS-only cohorts demonstrated obliteration rates of 51%-68.5% in the three largest unselected pediatric series (range 36%-84% across all SRS-inclusive cohorts), with latency-period hemorrhage rates of approximately 1.1%-3.2% per series. Recurrence after apparent cure was reported in 7 cohorts, with rates ranging from < 1% to 29%, occurring years after angiographic obliteration. CONCLUSIONS: Contemporary retrospective pediatric bAVM data indicate that modality-specific outcomes are strongly influenced by lesion selection, outcome definitions, and follow-up duration. Recurrence after apparent cure is not uncommon and supports explicit long-term surveillance. Standardized definitions and harmonized reporting are needed to improve interpretability and comparative inference.
29 May 2026
Read appraisal →Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery
The relation between cerebral arteriovenous malformations and heart diseases in the pediatric population: a systematic review and meta-analysis
Cerebral arteriovenous malformations (AVMs) and congenital heart diseases (CHDs) are rare, complex conditions that often present diagnostic and therapeutic challenges-especially when they coexist in pediatric patients. Though individually uncommon, their association raises important questions about shared developmental origins, systemic implications, and long-term outcomes. Understanding this relationship is crucial for improving early recognition, tailoring treatment strategies, and reducing morbidity and mortality in affected children. This systematic review and meta-analysis explored the relationship between AVMs and CHDs in the pediatric population, analyzing 139 studies published between 1971 and 2023. Together, these studies included data from 1042 patients. The co-occurrence of these two conditions highlights a potential interplay between cerebral and cardiac vascular development. By clarifying their association, this study aims to support earlier recognition, improve multidisciplinary management, and ultimately enhance clinical outcomes. The results revealed important patterns in how specific types and locations of AVMs relate to different forms of heart disease. AVMs located in infratentorial regions-such as the brainstem and cerebellum-were generally associated with lower risks of complications, while those in deep cerebral areas showed stronger links to adverse outcomes. Among the heart conditions evaluated, atrial septal defects (ASDs), cardiomyopathies, and high-output cardiac failure were most frequently associated with AVMs, suggesting overlapping embryologic and hemodynamic mechanisms. The most commonly reported complications included hydrocephalus, hemorrhage, and mortality. ROC curve analysis showed that mortality had the highest predictive value (AUC 0.62), followed by hydrocephalus (0.53) and hemorrhage (0.48). A composite risk model (AUC 0.59) suggested that combining clinical variables could improve prognostication. Neurological sequelae such as seizures, developmental delay, and microcephaly were frequent, particularly in hemorrhagic cases. Interestingly, in some patients, AVM treatment led to improvement in cardiac symptoms, suggesting a bidirectional relationship between cerebral and cardiac function. Despite variability in study quality, results were consistent across analyses, even when lower-quality studies were excluded. Most studies were rated moderate to high quality, though classification challenges were noted. Overall, the findings support a meaningful association between specific AVM and CHD subtypes and highlight the importance of early diagnosis and collaborative care involving multidisciplinary approach.
25 May 2026
Read appraisal →Journal of neuro-oncology
Should checkpoint inhibitors be reserved for biomarker-selected pediatric brain tumors?
PURPOSE: Pediatric brain tumors, including high-grade gliomas (HHG), medulloblastomas (MB), and ependymomas (EPN), are a leading cause of death in children. They are often immunologically "cold" with low tumor mutational burden (TMB) and very few tumor-infiltrating lymphocytes (TILs), which may limit the role of immune checkpoint inhibitors (ICI). METHODS: We performed a PRISMA‑guided systematic review of PubMed/MEDLINE, Embase, and Scopus from inception to September 17, 2025, for English-language studies of patients ≤ 21 years with primary CNS tumors treated with PD‑1/PD‑L1 or CTLA‑4 inhibitors. Eligible reports included prospective trials, retrospective series, and observational/case reports with extractable data on efficacy and/or toxicity. Of 479 records identified, 386 unique citations were screened, 127 underwent full‑text review, and 40 met inclusion criteria for qualitative synthesis. RESULTS: Prospective and institutional studies in biomarker-unselected diffuse midline glioma, high-grade glioma, medulloblastoma, and ependymoma showed low objective response rates (generally ≤ 6%), short median progression-free survival (1-3 months), and overall survival similar to historical controls, despite acceptable safety (grade ≥ 3 treatment-related adverse events ~ 15-25% with anti-PD-1 ± anti-CTLA-4). In contrast, across germline MMR-deficient and broader RRD/hypermutant cohorts, PD-1 blockade produced clinically meaningful and sometimes durable responses, including complete remissions in malignant glioma and approximate 2-year overall survival near 50%, often with delayed responses and pseudoprogression. Histology-specific profiling highlighted marked variation in immune contexture: pediatric gliomas segregate into immune "hot," "altered," and "cold" subtypes; medulloblastoma is largely PD-L1-low with prominent B7-H3 and myeloid programs; checkpoint expression is also observed in germ cell and selected sellar tumors. Evidence quality is limited by small, heterogeneous, predominantly non-comparative designs. CONCLUSION: ICIs show manageable safety but limited efficacy in unselected pediatric CNS tumors. Durable benefit is most evident in RRD/hypermutant biology and possibly PD-L1-high niches (e.g., some low-grade gliomas and CNS-GCT). Future trials should be biomarker-driven and pair ICIs with priming combinations (e.g., radiation, epigenetic modulators, metronomic chemotherapy) to convert "cold" tumors into responders.
18 May 2026
Read appraisal →Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery
Postoperative raised ICP after minimally invasive surgery for monosutural craniosynostosis
INTRODUCTION: Minimally invasive craniectomies (MIC) for monosutural craniosynostosis are more and more used. Their results are usually investigated in terms of surgical, esthetic, and neuropsychological outcomes but not specifically about the risk of postoperative raised intracranial pressure (ICP). The goal of this article is to review the literature and the personal experience looking for the possible risk of postoperative raised ICP after MIC. METHODS: A systematic review adhering to PRISMA guidelines was realized. In parallel, the personal experience concerning scaphocephalic children consecutively operated on between 2010 and 2013 by MIC was analyzed. Children operated on by open cranial vault remodeling (OCVR) in the same period were considered as controls. RESULTS: Overall, 3048 patients from 14 studies were included, 5% of them requiring a reoperation after a 59.9-month mean follow-up. A total of 2297 children were treated by MIC: 2.8% (65 cases) showed postoperative raised ICP, the rate of reoperation was 4.4% (102 cases). The risk of reoperation varies across the techniques, being higher in modified or spring-assisted strip craniectomies (2.4-14.6%) than in endoscopy-assisted craniectomies (2.4%). Among 751 cases treated by OCVR, this risk varied from 0.95 to 7.1%. Also in the personal experience, no significant differences were found between MIC (1.5% of reoperation for raised ICP) and OCVR (0.8%). CONCLUSIONS: The risk of postoperative raised ICP seems to be relatively low after surgery for monosutural synostosis without a significant difference between MIC and OCVR. Missing standardized methods for postoperative screening, missing information about the reason of reoperation in some studies, and the retrospective analysis methods in most of them are the main limitations of this study.
13 May 2026
Read appraisal →Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery
Surgical management of anterior sacral meningoceles: a case series and systematic review
PURPOSE: Anterior sacral meningoceles (ASMs) are rare forms of spinal dysraphism caused by anterior herniation of meninges through a sacral defect. Surgical management is the treatment of choice, yet the optimal approach remains debated. This study compares outcomes of three main surgical approaches-anterior transabdominal, posterior transsacral, posterior sagittal (PSARP-type)-focusing on the efficacy and safety of the PSARP-type. METHODS: A retrospective single-center analysis was conducted on eight patients treated for ASM between 2008 and 2025. Demographic, clinical, radiological, functional data were reviewed. Outcomes included recurrence, reoperation, sac size reduction, bladder function; complications were categorized as early or late. A systematic literature review was performed according to PRISMA guidelines. RESULTS: Eight patients (6 females, 2 males; mean age 19.4 years) underwent surgery (five anterior Pfannenstiel incision, two posterior sagittal, one posterior transsacral). All were symptomatic, 87.5% had associated malformations. Sac reduction was achieved in 87.5% of cases, with no new neurological deficits. Complications were mild and self-limiting, including transient urinary retention and cerebrospinal fluid fistula managed with lumboperitoneal shunt. The literature review (49 studies, 68 patients) confirmed female predominance (81%), association with Marfan and Currarino syndromes (35%), preference for posterior approaches (70%), which showed lower morbidity and recurrence rates. One postoperative death (1.4%) was reported. CONCLUSIONS: Posterior approaches-particularly the PSARP-type-provide safe, effective, and functionally favorable alternatives to anterior surgery for ASM, offering optimal exposure, precise dural closure, superior cosmetic outcomes with minimal morbidity. The anterior route should be reserved for large or high-lying lesions. Further multicenter studies with longer follow-up are warranted.
17 Apr 2026
Read appraisal →Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery
The role of age and Galassi type in management and surgical outcomes of pediatric middle fossa arachnoid cysts: a systematic review and meta-analysis
PURPOSE: Arachnoid cysts are found in approximately 2.6% of the pediatric population, mostly asymptomatic, incidental findings. However, those with mass effect symptoms from cyst growth may require surgery. Because of the variability in progression, we investigated the interaction of patient age with middle fossa arachnoid cyst (MFAC) clinical outcomes. METHODS: A systematic review using PubMed, Google Scholar, and Web of Science electronic databases was completed according to the PRISMA guidelines to identify articles detailing pediatric MFACs. RESULTS: Sixty articles meeting the inclusion criteria, totaling 1024 patients, were included. Signs of elevated ICP (194/873, prevalence: 22.2%, 95% CI: 19.5-25.0%) and primary headaches (263/873, prevalence: 30.1%, 95% CI: 27.1-33.2%) were the most common presenting symptoms. In the evaluation of the hemispheric location of MFACs, younger ages at presentation were more likely to present with bilateral MFACs (OR: 0.71, 95% CI: 0.52-0.98, p = 0.034). Most MFACs in the retrospective studies had left laterality (421/677, prevalence: 62.2%, 95% CI: 58.6-65.1%) and were singular (791/817, prevalence:96.8%, 95% CI: 95.6-98.2%). A higher proportion of younger patients required a second operation (OR: 0.82, 95% CI: 0.72-0.93, p = 0.002) and cerebrospinal fluid shunting (OR: 0.88, 95% CI: 0.79-0.97, p = 0.011) across the case reports. There was no significant difference in complications when comparing older children (5-18 years) to young children (≤ 4 years) (p = 0.737) or Galassi type (p = 0.736), but Galassi type 1 was associated with management through observation (p = 0.019). CONCLUSION: Younger patients presented with a higher rate of bilateral MFACs and required more invasive treatment methods and repeat operations more frequently, suggesting that infants and young children with MFACs may require greater surveillance during and after management to minimize complications.
14 Apr 2026
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