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UNASSIGNED: The aim of the study was to explore the factors influencing the availability of medications for children, and establish a machine learning model to provide an empirical basis for the subsequent formulation and improvement of relevant policies.
UNASSIGNED: Design: Cross-sectional survey. Setting: 12 provinces, China. Medical doctors from 25 public hospitals were enrolled. All data were randomly divided into a training set and a validation set at a ratio of 7:3. Three prediction models, namely random forest (RF), logistic regression (LR), and extreme gradient boosting (XGBoost), were developed and compared. The receiver operating characteristic curve (ROC) and the associated area under the curve (AUC) were used to evaluate the three models. A nomogram and clinical impact curve (CIC) for availability of medication were developed.
UNASSIGNED: Fifteen of 29 factors in the database that were most likely to be selected were considered to establish the prediction model. The XGBoost model (AUC = 0.915) demonstrated better performance than the RF model (AUC = 0.902) and the LR model (AUC = 0.890). According to the Shapley additive explanation values, the five factors that most significantly affected the availability of medications for children in the XGboost model were as follows: the relatively small number of specialized dosage forms for children; unaffordable medications for children; public education on the accessibility and safety of medication for children; uneven distribution of medical resources, leading to insufficient access to medication for children; and years of service as a doctor. The CIC was used to assess the practical applicability of the factor prediction nomogram.
UNASSIGNED: The XGBoost model can be used to establish a prediction model to screen the factors associated with the availability of medications for children. The most important contributing factors to the models were the following: the relatively small number of specialized dosage forms for children; unaffordable medications for children; public education on the accessibility and safety of medication for children; uneven distribution of medical resources, leading to insufficient access to medication for children; and years of service as a doctor.

Epilepsy is a common symptom of pediatric cavernous malformations. In medically refractory patients, surgery can achieve high seizure freedom rates with low morbidity. This video depicts the use of a minipterional craniotomy and transsulcal resection of a frontal opercular cavernous malformation in a 13-year-old female with medically intractable epilepsy. At 1-year follow-up, she was evaluated as Engel class I with a significant improvement in her quality of life. Principles of cavernous malformation resection for the treatment of epilepsy are also reviewed. The video can be found here: https://stream.cadmore.media/r10.3171/2024.4.FOCVID2441.

BACKGROUND: In assigning manner of death (MOD) for inclusion on death certificates, medical examiners and coroners do not always apply uniform criteria. Previous research indicates surveillance statistics based on death certificates, such as the National Vital Statistics System, grossly miscount unintentional firearm deaths. The National Violent Death Reporting System (NVDRS) has taken steps to reduce variability in manner of death coding by providing uniform criteria for assigning an \"abstractor manner of death\" (AMD). AMD has five categories: unintentional, suicide, homicide, undetermined, and legal intervention homicide. A previous study found good accuracy of AMD coding for unintentional firearm deaths, all ages, 2003-2006, but a more recent study reported that the NVDRS undercounted self- and other-inflicted unintentional firearm deaths in which both the victim and shooter (for other-inflicted injuries) were under age 15 (2009-2018).
RESULTS: We replicated the recent study\'s sample population, identifying 924 NVDRS incidents from 2009 to 2018 in which both victim and, for other-inflicted injuries, shooter age was under 15 and AMD was homicide, suicide, unintentional or undetermined (there were no legal intervention deaths to children). We assigned a researcher-adjudicated MOD (RMD) by reviewing incident narratives. RMD was compared with AMD and with manner recorded on the death certificate. Based on RMD as the gold standard, the sensitivity, specificity, and predictive values positive and negative of the AMD for unintentional childhood firearm deaths were, respectively, 90%, 99%, 98% and 96%; 86% (24/28) of false negatives were coded by abstractors as homicides. By contrast, death certificate manner had relatively poor sensitivity (63%).
CONCLUSIONS: In our sample of 924 deaths, the abstractor manner of death generally agreed with researcher-adjudicated manner of death, though not perfectly, missing 10% of researcher-adjudicated unintentional deaths, mostly because abstractors coded these unintentional deaths as homicides. A sizable minority of false negatives were unintentional deaths where the narrative explicitly noted that adult negligence contributed to a child\'s unintentional shooting death. While AMD coding in NVDRS is good, it could be improved if NVDRS coding guidelines explicitly affirmed that potential prosecution for negligent manslaughter is not a contraindication to an AMD of unintentional, provided the firearm was not used to intentionally harm, threaten, or coerce.

BACKGROUND: The aim of this study was to find statistically valid criteria to preoperatively divide acute appendicitis into simple and complicated to enable surgeons to administer the most appropriate antibiotic prophylaxis/therapy before surgery.
METHODS: We retrospectively reviewed a cohort of patients who underwent appendectomy from January 2022 to December 2023. Patients included were 0-14 years of age. Exclusion criteria included patients who underwent interval appendectomy or concurrent procedures at the same time of appendectomy. We divided patients into two groups: simple (group S) and complicated (group C) appendicitis according to intraoperative finding. Generalized linear model (GLM) with logit function was developed to identify the predictive variables of the type of appendicitis (S vs C) in terms of CRP value, neutrophils percentage and WBC count adjusted for age and sex of patients. Finally, principal component analysis (PCA) was carried out to identify the cutoff value of statistically significant variables found in the previous analysis.
RESULTS: One hundred and twenty patients were eligible (N female = 49, N male = 71) for the study. 74 and 46 patients were included in groups S and C, respectively. In a preliminary analysis using univariate and multivariate GLM, only CRP (p value =  < 0.001) and neutrophils percentage (p value = 0.02) were predictive variables for the type of appendicitis. The GLM shows a statistical lower value of CRP (adjusted odds ratio [OR] per unit, 0.17 [95% CI, 0.08-0.39]) and neutrophil percentage (adjusted OR per unit, 0.37 [95% CI, 0.16-0.86]) in the S group compared to C adjusted to age and sex. PCA analysis revealed a P-ROC cutoff of 4.2 mg/dl and 80.1 of CRP value (AUC = 84%) and neutrophil percentage (AUC = 70%), respectively.
CONCLUSIONS: We will perform a prospective study giving preoperative prophylactic cefazolin to patients with a CRP value under 4.2 mg/dl and amoxicillin-clavulanate therapy to patient with CRP value over 4.2 mg/dl.

The objective of this study was to examine the utility of the acceleration index observed in an electrocardiogram (ECG) for the prediction of the effectiveness of orthostatic training in pediatric patients diagnosed with postural orthostatic tachycardia syndrome (POTS). This investigation focused on children diagnosed with POTS and undergoing orthostatic training at the Department of Pediatrics of Peking University First Hospital from January 2012 to October 2022. Specifically, patients hospitalized from January 2012 to December 2019 were included in the training set (54 cases), while those hospitalized from January 2020 to October 2022 were included in the external validation set (37 cases). All children received a 3-month orthostatic training, and the baseline symptom score (SS) was calculated in agreement with the pretreatment orthostatic intolerance symptom frequency. Additionally, we determined post-treatment SS during follow-up via telephone after the 3-month treatment. Children with a decrease in post-treatment SS by ≥ 50% of the baseline were considered as responders; otherwise, they were considered as non-responders. Demographic data (age, sex, and body mass index), hemodynamic parameters (supine blood pressure, time to achieve a positive standing test, maximum increase in heart rate during the standing test, maximal heart rate reached during the standing test, and blood pressure at the point of maximal heart rate during the standing test), and electrocardiographic parameters (RR interval in the supine position, shortest RR interval in the upright position, and acceleration index) were collected from all the children prior to treatment. Univariate and multivariate regression analysis were conducted to investigate factors associated with the efficacy of orthostatic training. The predictive value of these indicators for the therapeutic effectiveness of orthostatic training in children with POTS was evaluated using receiver operating characteristic (ROC) analysis, and the indicators were validated using the validation set. Among the 54 children in the training set, 28 responded to orthostatic training, and 26 were nonresponsive. Compared with the non-responders, the responders demonstrated a significant reduction in acceleration index (P < 0.01). The ROC curve for the predictive value of the acceleration index exhibited an area under the curve = 0.81 (95% confidence interval: 0.685-0.926). With the acceleration index threshold < 27.93%, the sensitivity and specificity in the prediction of orthostatic training efficacy among children with POTS were 85.7% and 69.2%, respectively. The external validation results demonstrated that using acceleration index < 27.93% as the threshold, the sensitivity, specificity, and accuracy of predicting orthostatic training efficacy among children with POTS were 89.5%, 77.8%, and 83.8%, respectively.
CONCLUSIONS: Electrocardiographic acceleration index can be used to predict the effectiveness of orthostatic training in treating children with POTS.
BACKGROUND: • Postural orthostatic tachycardia syndrome (POTS) is a chronic orthostatic intolerance involving multiple mechanisms. Autonomic dysfunction is one of the main mechanisms of POTS in children and could be treated with orthostatic training. • In order to improve the efficacy of orthostatic training in children with POTS, it is particularly important to identify the patients with autonomic dysfunction as the main mechanism before the treatment.
BACKGROUND: • We found acceleration index of the electrocardiogram (ECG) can be used as a satisfactory index to predict the efficacy of orthostatic training in the treatment of POTS in children. • Using the acceleration index to predict the efficacy of orthostatic training on POTS in children is easy to be popularized in hospitals at all levels because it is non-invasive, convenient, and not expensive.

OBJECTIVE: A dysfunctional hypothalamus may result in decreased feelings of satiety (hyperphagia), decreased energy expenditure, and increased fat storage as a consequence of hyperinsulinemia. Hypothalamic dysfunction may thus lead to morbid obesity and can be encountered in childhood as a consequence of congenital, genetic, or acquired disorders. There is currently no effective treatment for hypothalamic obesity (HO). However, comparable to alimentary obesity, dietary and lifestyle interventions may be considered the cornerstones of obesity treatment. We questioned the effect of dietary or lifestyle interventions for HO and systematically searched the literature for evidence on feasibility, safety, or efficacy of dietary or lifestyle interventions for childhood hypothalamic overweight or obesity.
METHODS: A systematic search was conducted in MEDLINE (including Cochrane Library), EMBASE, and CINAHL (May 2023). Studies assessing feasibility, safety, or efficacy of any dietary or lifestyle intervention in children with hypothalamic overweight or obesity, were included. Animal studies, studies on non-diet interventions, and studies with no full text available were excluded. Because the number of studies to be included was low, the search was repeated for adults with hypothalamic overweight or obesity. Risk of bias was assessed with an adapted Cochrane Risk of Bias Tool. Level of evidence was assessed using the GRADE system. Descriptive data were described, as pooled-data analysis was not possible due to heterogeneity of included studies.
RESULTS: In total, twelve studies were included, with a total number of 118 patients (age 1-19 years) of whom one with craniopharyngioma, one with ROHHAD-NET syndrome, 50 with monogenic obesity, and 66 with Prader-Willi syndrome (PWS). Four studies reported a dietary intervention as feasible. However, parents did experience difficulties with children still stealing food, and especially lowering carbohydrates was considered to be challenging. Seven studies reported on efficacy of a dietary intervention: a well-balanced restrictive caloric diet (30% fat, 45% carbohydrates, and 25% protein) and various hypocaloric diets (8-10 kcal/cm/day) were considered effective in terms of weight stabilization or decrease. No negative effect on linear growth was reported. Four studies reported on specific lifestyle interventions, of which three also included a dietary intervention. Combined dietary and lifestyle intervention resulted in decreased BMI, although BMI returned to baseline values on long-term. One additional study was identified in adults after brain trauma and showed a significant reduction in BMI in one out of eight patients after a combined dietary and lifestyle intervention.
CONCLUSIONS: Hypocaloric diet or restrictive macronutrient diet with lower percentage of carbohydrates seems feasible and effective for childhood HO, although most of the studies had a high risk of bias, small cohorts without control groups, and were conducted in children with PWS only, compromising the generalizability. Lifestyle interventions only resulted in BMI decrease in short-term, indicating that additional guidance is needed to sustain its effect in the long-term. Literature on feasibility and efficacy of a dietary or lifestyle intervention for hypothalamic overweight or obesity is scarce, especially in children with acquired HO (following treatment for a suprasellar tumor). There is need for prospective (controlled) studies to determine which dietary and lifestyle intervention are most helpful for this specific patient group.

OBJECTIVE: To evaluate the effectiveness and safety of nusinersen for the treatment of 5q-spinal muscular atrophy (SMA) among Chinese pediatric patients.
METHODS: Using a longitudinal, multi-center registry, both prospective and retrospective data were collected from pediatric patients with 5q-SMA receiving nusinersen treatment across 18 centers in China. All patients fulfilling the eligibility criteria were included consecutively. Motor function outcomes were assessed post-treatment by SMA type. Safety profile was evaluated among patients starting nusinersen treatment post-enrollment. Descriptive analyses were used to report baseline characteristics, effectiveness, and safety results.
RESULTS: As of March 2nd, 2023, 385 patients were included. Most patients demonstrated improvements or stability in motor function across all SMA types. Type II patients demonstrated mean changes [95% confidence interval (CI)] of 4.4 (3.4-5.4) and 4.1 (2.8-5.4) in Hammersmith Functional Motor Scale-Expanded (HFMSE), and 2.4 (1.7-3.1) and 2.3 (1.2-3.4) in Revised Upper Limb Module (RULM) scores at months 6 and 10. Type III patients exhibited mean changes (95% CI) of 3.9 (2.5-5.3) and 4.3 (2.6-6.0) in HFMSE, and 2.1 (1.2-3.0) and 1.5 (0.0-3.0) in RULM scores at months 6 and 10. Of the 132 patients, 62.9% experienced adverse events (AEs). Two patients experienced mild AEs (aseptic meningitis and myalgia) considered to be related to nusinersen by the investigator, with no sequelae.
CONCLUSIONS: These data underscore the significance of nusinersen in Chinese pediatric patients with SMA regarding motor function improvement or stability, and support recommendations on nusinersen treatment by Chinese SMA guidelines and continuous coverage of nusinersen by basic medical insurance.

To conduct a systematic review of the literature regarding rates and predictors of favorable seizure outcome after resective surgery for epileptic spasms (ES) in pediatric patients. The Preferred Reporting Items for Systematic Reviews and Meta-Analyses standards were followed. We searched PubMed, EMBASE, and Cochrane CENTRAL for articles published on the prevalence or incidence of epileptic spasm since 1985. Abstract, full-text review, and data extraction were conducted by two independent reviewers. Meta-analysis was performed to assess overall seizure freedom rate. Subject-level analysis was performed on a subset of studies to identify prognostic indicators. A total of 21 retrospective studies (n = 531) were included. Meta-analysis of all studies demonstrated a pooled seizure freedom rate of 68.8%. Subject-level analysis on 18 studies (n = 360) demonstrated a significant association between duration of spasms and recurrence of spasms after surgery, with an estimated increased risk of 7% per additional year of spasms prior to operation. Patients who underwent resective surgery that was not a hemispherectomy (i.e., lobectomy, lesionectomy, etc.) had an increased recurrence risk of 57% compared to patients who had undergone hemispherectomy. Resective surgery results in seizure freedom for the majority of pediatric patients with epileptic spasms. Patients who undergo hemispherectomy have lower risk of recurrence than patients who undergo other types of surgical resection. Increased duration of spasms prior to surgery is associated with increased recurrence risk after surgery. PLAIN LANGUAGE SUMMARY: Children with epileptic spasms (ES) that do not respond to medications may benefit from surgical treatment. Our study reviewed existing research to understand how effective surgery is in treating ES in children and what factors predict better outcomes. Researchers followed strict guidelines to search for and analyze studies published since 1985, finding 21 studies with a total of 531 patients. They found that, on average, nearly 70% of children became seizure-free after surgery. Further individual analysis of 360 patients showed that longer duration of spasms before surgery increased the risk of spasms returning by 7% per year. Additionally, children who had less extensive surgeries, such as removal of only a specific part of the brain, had a 57% higher risk of seizure recurrence compared to those who had a hemispherectomy, which removed or disconnected half of the brain. Overall, the study concludes that surgery can often stop seizures, especially when more extensive surgery is performed and when the surgery is done sooner rather than later.

Traumatic brain injury (TBI) is a condition that occurs commonly in children from infancy through adolescence and is a global health concern. Pediatric TBI presents with a bimodal age distribution, with very young children (0-4 years) and adolescents (15-19 years) more commonly injured. Because children\'s brains are still developing, there is increased vulnerability to the effects of head trauma, which results in entirely different patterns of injury than in adults. Pediatric TBI has a profound and lasting impact on a child\'s development and quality of life, resulting in long-lasting consequences to physical, cognitive, and emotional development. Chronic issues like learning disabilities, behavioral problems, and emotional disturbances can develop. Early intervention and ongoing support are critical for minimizing these long-term deficits. Many animal models of TBI exist, and each varies significantly, displaying different characteristics of clinical TBI. The neurodevelopment differs in the rodent from the human in timing and effect, so TBI outcomes in the juvenile rodent can thus vary from the human child. The current review compares findings from preclinical TBI work in juvenile and adult rodents to clinical TBI research in pediatric and adult humans. We focus on the four brain regions most affected by TBI: the prefrontal cortex, corpus callosum, hippocampus, and hypothalamus. Each has its unique developmental projections and thus is impacted by TBI differently. This review aims to compare the healthy neurodevelopment of these four brain regions in humans to the developmental processes in rodents.