Research Appraisals
Evidence-based critical appraisals of the latest medical research, systematically evaluated using Oxford CEBM methodology.
Showing 8 appraisals
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 →JMIR research protocols
Coconstructing CHAMP, an Artificial Intelligence Chatbot for Pediatric Infectious Symptoms Management: Protocol for a Multiphase Participatory Study
BACKGROUND: Acute infectious symptoms are a leading cause of pediatric emergency department visits in Canada, many of which are low acuity and could be safely managed at home. Artificial intelligence (AI) chatbots offer a promising avenue for delivering accessible, evidence-based guidance to support families in managing these symptoms. OBJECTIVE: This study aims to adapt and coconstruct CHAMP (CHatbot to Assist the Management of Pediatric patients), an AI chatbot to support patients and families with acute pediatric infectious symptoms. CHAMP aims to deliver timely, tailored, and validated health information to support safe at-home self-management and informed care-seeking. METHODS: This multiphase, mixed methods participatory study will be conducted at the Montreal Children's Hospital in Montreal, Quebec, Canada. A coconstruction committee comprised of youth, parents, caregivers, and partners will be engaged as coresearchers. Eligible participants will include (1) youth aged 14-17 years and (2) parents and caregivers of children aged 0-17 years. The study comprises 5 phases. Phase 1 involves a qualitative needs assessment using focus groups with 20 participants to explore their informational needs, preferences, and concerns regarding pediatric infections and the use of AI chatbots. Phase 2 focuses on coconstructing and validating CHAMP's knowledge database through 3-5 workshops. Coresearchers will review pediatric clinical guidelines, map care questions and decision-making processes, and shape CHAMP's conversational framework. Phase 3 consists of iterative prototyping and testing through 3-5 workshops. Coresearchers will engage in prototyping and scenario testing, alongside preliminary usability and acceptability assessments. Phase 4 examines equity and accessibility through focus groups with 20 participants at risk of digital exclusion, as well as multilingual evaluation of an automated large language model-based translation layer. Phase 5 uses collaborative ethnography to explore the process of participatory coconstruction and its impact on CHAMP's design. RESULTS: Funding was secured in 2024, and Research Ethics Board approval was obtained in December 2024. As of December 2025, the coconstruction committee is being assembled, and Phase 1 recruitment is underway. CONCLUSIONS: This study will produce a functioning CHAMP prototype grounded in participatory, equitable, and responsible pediatric AI development. Findings will inform usability testing and an implementation-effectiveness evaluation, contributing to best practices for pediatric-centered AI health tools. By providing timely, tailored, and validated health information on acute infections, CHAMP may support safe at-home self-management, reduce preventable emergency department visits, ensure at-risk children are directed to appropriate care, and improve patient and family health care experiences.
28 May 2026
Read appraisal →Critical care explorations
External Validation, Recalibration, and Extension of a Prediction Model of Early Acute Kidney Injury in Critically Ill Children Using Multicenter Data
BACKGROUND: Acute kidney injury (AKI) is common in critically ill children and is associated with high morbidity and mortality. Risk prediction models designed for clinical decision support implementation can facilitate early identification and proactive mitigation of AKI risk. Existing models have primarily been validated using single-center data, partly because of the lack of appropriately detailed multicenter datasets. OBJECTIVE: To determine the performance of a single-center model to predict new AKI at 72 hours of ICU admission in children across two multicenter datasets and refine this model to improve prediction performance while maintaining acceptable alert burden. DERIVATION AND VALIDATION COHORTS: We analyzed two datasets: the Pediatric Learning Health System Network-Virtual Pediatric Systems (PEDSNET-VPS) dataset, created through the linkage of PEDSnet electronic health record (EHR) extraction with VPS (LLC, http://www.myvps.org), and the PICU Data Collaborative dataset, created through EHR extraction and harmonization from eight participating institutions. We divided each dataset into a derivation and test split. PREDICTION MODEL: We first recalibrated an existing single-center model and measured discrimination (area under the receiver operating characteristic curve [AUROC] and area under the precision-recall curve [AUPRC]) and performance at multiple cutpoints. We next added features available at 12 hours of ICU admission, optimizing by precision and recall. We measured discrimination and performance at multiple cutpoints and identified the features contributing most to the risk score. RESULTS: In total we analyzed 186,540 ICU admissions. We found early AKI by serum creatinine criteria within 72 hours of admission in 2.2-2.7%. Initial recalibration of an existing single-center model demonstrated poor discrimination (AUROC 0.65-0.78; AUPRC 0.10-0.12). Following the addition of new features, the model had higher AUROC (0.80-0.88) and AUPRC (0.13-0.22). CONCLUSIONS: In this first use of two new multicenter datasets, we found improved performance in a model designed using features available at 12 hours of ICU admission, balancing sensitivity and precision to predict patients at risk for AKI development.
26 May 2026
Read appraisal →European journal of pediatrics
Pediatric Trauma Score (PTS) combined with blood lactate measurement provides clinical treatment guidance and prognosis prediction for children with compound injuries: a prospective study
UNLABELLED: Compound injuries in children are the leading cause of death and disability, and early and accurate assessment is crucial to optimize treatment. This study aimed to explore the clinical value of Pediatric Trauma Score (PTS) combined with blood lactate measurement in the triage, treatment guidance and prognosis prediction of pediatric compound injuries. From January 2023 to December 2024, a randomized, single-blind controlled trial was conducted in the emergency department of Hebei Provincial Children's Hospital (the only tertiary pediatric medical center in Hebei Province), China. A total of 546 children with compound injuries who presented to the hospital were included and randomly divided into the intervention group (n = 273, managed by PTS combined with dynamic monitoring of blood lactate) and the control group (n = 273, managed by PTS only). The main outcomes were the timeliness of rescue (time to critical intervention) and the accuracy of prognosis prediction (predictive effectiveness of mortality and major complications). Secondary outcomes included emergency department length of stay, unplanned Intensive Care Unit (ICU) admission, and 30-day complication rate. The median time from admission to initiation of key interventions was significantly shorter in the intervention group compared with the control group (45 [interquartile range (IQR): 30-65] min vs. 68 [IQR: 45-95] min, P < 0.001). The area under the receiver operating characteristic (ROC) curve (AUC) of the combined indicator (PTS + initial lactate) to predict 30-day mortality was 0.936 (95% CI: 0.902-0.970), which was significantly higher than PTS alone (AUC: 0.812, P < 0.001) or initial lactate (AUC: 0.855, P = 0.002). The emergency department length of stay, unplanned ICU admission rate and 30-day complication rate of the intervention group were significantly lower than those of the control group (all P < 0.05). Dynamic monitoring showed that 6-h lactate clearance < 30% was a strong independent predictor of the occurrence of multiple organ dysfunction syndrome (MODS) (odds ratio (OR) = 5.42, 95% CI: 2.88-10.21,P < 0.001. CONCLUSION: The combined application of PTS and blood lactate integrates physiological status and metabolic information, which can significantly improve the accuracy of early triage of pediatric compound injuries, guide the implementation of rescue measures, and effectively predict prognosis through dynamic monitoring, providing a reliable basis for precise personalized management. WHAT IS KNOWN: • Compound injuries in children are the leading cause of death and disability, and early and accurate assessment is crucial to optimize treatment. WHAT IS NEW: • The combined application of the Pediatric Trauma Score (PTS) and blood lactate signifi cantly improves the accuracy of early triage and 30-day mortality prediction, shortens the time to critical intervention, and reduces unplanned ICU admission and 30-day complication rates in pediatric compound injuries. • Dynamic monitoring shows that 6-hour lactate clearance 30% is a strong independent predictor of multiple organ dysfunction syndrome (MODS).
25 May 2026
Read appraisal →European journal of pediatrics
Development and validation of an explainable machine learning model for predicting surgical intervention in pediatric intestinal obstruction
Pediatric intestinal obstruction, a critical acute abdomen condition, carries a risk of intestinal necrosis. Decisions for urgent surgery lack standardized criteria. This study aimed to develop an explainable machine learning (ML) model to predict surgical indication, reducing diagnostic variability. A retrospective study included 642 children from our center (split into training/internal validation sets) and 137 from two external centers. Surgical indication was defined based on the presence of one or more of the following: evidence of peritonitis on physical examination, radiographic signs of strangulation, or failure of conservative management for > 24 h; in cases of diagnostic uncertainty, consensus among three senior physicians was used as the adjudication standard. We built models using 35 easily obtainable features across eleven algorithms and evaluated performance via AUC and other metrics. SHapley Additive exPlanations (SHAP) provided model interpretability, and an optimal probability threshold was established for clinical stratification. At the optimal threshold determined by maximizing the Youden index, the model achieved a sensitivity of 89% and a specificity of 99% in the internal validation set. The random forest model demonstrated the highest discriminative performance. A streamlined, interpretable model using the eight most important features (including vital sign dynamics and clinical markers) showed no evidence of overfitting based on the close agreement between training and validation performance metrics. It achieved AUCs of 0.988 (internal validation) and 0.981 (external validation). The model has been translated into a convenient clinical tool.Conclusion: Our explainable ML model demonstrates strong discriminative performance in predicting the need for surgery in pediatric intestinal obstruction and is strongly associated with intestinal necrosis. By mitigating the "black-box" concern through SHAP, it offers transparent, interpretable decision support to clinicians.
20 May 2026
Read appraisal →European journal of pediatrics
Piperacillin-tazobactam versus cefepime monotherapy in pediatric patients with febrile neutropenia: a systematic review and meta-analysis
UNLABELLED: The purpose of this study is to evaluate whether piperacillin-tazobactam therapy, compared with cefepime monotherapy, results in significant difference in treatment success, mortality, and duration outcomes in pediatric patients with febrile neutropenia (FN). A systematic review and meta-analysis of randomized controlled trials (RCTs) was conducted following PRISMA guidelines. PubMed, Embase, Scopus, Web of Science, and Cochrane CENTRAL were searched up to February 3. No starting limits for dates were used. We only included studies of pediatric patients with FN comparing piperacillin-tazobactam to cefepime monotherapy. Risk of bias was assessed using Cochrane RoB 2 tool. Meta-analysis was performed using random-effects model to calculate risk ratios (RR) and mean differences (MD). Five RCTs involving 470 episodes were included. The pooled analysis for treatment success showed no statistically significant difference between groups (RR = 1.02; 95% CI [0.89; 1.18]; P = 0.76) with low heterogeneity (I2 = 0.0%). No significant differences for mortality (RR = 2.09; 95% CI [0.62; 7.03]; P = 0.23; I2 = 0.0%). Duration of treatment was 0.9 day shorter for cefepime group (MD = 0.9 day; 95% CI [0.2; 1.6]; P < 0.1; I2 = 0%). CONCLUSION: No statistically significant difference was found in treatment success or mortality between the groups. Patients receiving cefepime had a treatment duration 0.9 days shorter than those receiving piperacillin-tazobactam, although this finding should be interpreted with caution. The available evidence remains limited. Further RCTs are needed to elucidate potential differences in treatment success, mortality, and duration between the drugs. REGISTRATION: PROSPERO (CRD420261296621). WHAT IS KNOWN: • Cefepime and piperacillin-tazobactam are widely utilized as empirical monotherapy for febrile neutropenia, but evidence regarding their comparative clinical efficacy and safety in children remains inconclusive. WHAT IS NEW: • This meta-analysis quantifies differences in treatment success, mortality and duration of treatment outcomes in pediatric patients with febrile neutropenia.
12 May 2026
Read appraisal →European journal of pediatrics
Impact of protocolized restrictive versus liberal/usual maintenance fluid strategy on fluid overload among mechanically ventilated children: an open-label randomized trial (ReLiSCh-II trial)
UNLABELLED: Fluid overload (FO) is associated with poor clinical outcomes among critically ill children. The objective of this trial was to assess the impact of a protocolized restrictive maintenance fluid strategy on FO among mechanically ventilated children. This open-label randomized controlled trial was conducted in the pediatric intensive care unit (PICU) of a tertiary care hospital in North India over 13 months (November 2023-November 2024). Hemodynamically stable mechanically ventilated children were randomized to protocolized restrictive (n = 64) (40-50% of maintenance fluids with diuretic infusion if FO% > 10%); and liberal/usual (n = 66) (70-80% of maintenance fluid) groups. The primary outcome was the proportion of children with cumulative FO% > 10% through day 5. Secondary outcomes were daily cumulative FO%; inferior vena cava variability index (∆IVC) and N-terminal pro-brain natriuretic peptide (NT-proBNP) levels at 48 h; safety parameters (requirement of fluid boluses or vasoactive drugs); 28-day ventilator and PICU-free days (VFDs and PFDs), and mortality. Significantly fewer children in the protocolized restrictive group had cumulative FO% > 10% than in the liberal/usual group (22% vs. 47%, p = 0.003). Also, the restrictive group had significantly lower daily cumulative FO% on the first 5 days. ∆IVC and NT-proBNP levels at 48 h, as well as safety parameters, were similar between the two groups. Protocolized restrictive and liberal/usual groups had similar VFDs [20 (8-24) vs. 16 (0-23), p = 0.076], PFDs [16 (3-21) vs. 13 (0-19), p = 0.071], and mortality (14% vs. 24%, p = 0.14). CONCLUSION: A protocolized restrictive maintenance fluid strategy resulted in significantly lower FO among hemodynamically stable, mechanically ventilated children without a measurable impact on short-term clinical outcomes. WHAT IS KNOWN: • Fluid overload (FO) is associated with poor clinical outcomes among critically ill mechanically ventilated children. WHAT IS NEW: • Among hemodynamically stable, mechanically ventilated children, a restrictive maintenance fluid strategy may be a useful intervention to limit FO and improve clinical outcomes in LMICs.
10 May 2026
Read appraisal →Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery
Structural equation modeling of findings in pediatric tele-neuroradiology: a 2-year nationwide study of turnaround time predictors and clinical decision support
BACKGROUND: Pediatric neuroradiology faces significant workforce shortages, with teleradiology emerging as a vital solution. However, studies investigating findings and their operational impact from teleradiology centers remain limited. OBJECTIVES: To develop and validate structural equation models for identifying predictors of turnaround time and formulate recommendations for workflow optimization in pediatric tele-neuroradiology services. DESIGN: A retrospective cohort study following STROBE guidelines. SETTINGS: 107 hospitals across 17 states in the United States (US) via a teleradiology platform providing interpretation services through US board-certified radiologists. PATIENTS AND METHODS: We analyzed 9985 pediatric neuroradiology scans from 7958 patients (January 2023-December 2024). We utilized confirmatory factor analysis to validate findings structures, followed by structural equation modeling to predict turnaround times. Binary logistic regression models were developed with area-under-the-curve (AUC) estimation for performance assessment. Bootstrap validation with 5000 samples ensured model stability. MAIN OUTCOME MEASURES: Primary outcome was turnaround time. Secondary outcomes included requirements for multiple imaging studies, follow-up recommendations, and consultations. SAMPLE SIZE: A total of 9985 studies providing over 99% statistical power for detecting significant relationships. RESULTS: Factor analysis demonstrated a two-factor structure (trauma: α = 0.742, structural: α = 0.685). The structural model explained 7.8% of turnaround time variance, with computed tomography (CT) modality (β = -0.164), trauma score (β = 0.125), and structural score (β = 0.142) as significant predictors. Among immediate neurosurgical emergencies (n = 180, 1.8%), 89.4% achieved turnaround time within the 60-min benchmark for time-sensitive consultations. Prediction models demonstrated excellent discrimination: traumatic findings (AUC = 0.91), structural findings (AUC = 0.92), critical findings (AUC = 0.95), and a dedicated neurosurgical emergency model (AUC = 0.94, NPV = 0.996). A severity classification system showed strong validation against imaging needs (AUC = 0.76) and consultations (AUC = 0.89). CONCLUSIONS: Our study establishes a validated SEM framework for pediatric tele-neuroradiology with excellent predictive performance (AUC = 0.91-0.95). Among immediate neurosurgical emergencies (n = 180, 1.8%), 89.4% met the 60-min benchmark, and a dedicated emergency prediction model achieved AUC = 0.94. However, translation to improved neurosurgical care delivery and patient outcomes remains unvalidated, representing the next investigational priorities. LIMITATIONS: Retrospective design limits causal inference; a single platform may limit generalizability; the CT majority (96.8%) limits magnetic resonance imaging conclusions. Critically, post-diagnostic clinical outcomes, including neurosurgical consultations, interventions performed, and patient outcomes, were not tracked, precluding conclusions about whether documented operational efficiency translated to improved neurosurgical care delivery.
2 May 2026
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