Pediatric Investigation最新文献

Dengue fever remains a significant global health threat, placing nearly half of the world's population at risk. Despite decades of research, developing an effective dengue vaccine continues to face multiple challenges, including antibody-dependent enhancement (ADE), serotype-specific efficacy, and logistical barriers to vaccine delivery. This review provides a comprehensive analysis of the historical and current landscape of dengue vaccine development, focusing on CYD-TDV, TAK-003, and Butantan-DV. It examines their efficacy, safety profiles, and limitations, particularly in achieving balanced quadrivalent protection. Additionally, this review highlights key areas for future research, including the impact of ADE, advancements in vaccine platforms, the need for region-specific vaccine formulations, and the integration of vaccination into broader dengue prevention strategies. Ultimately, sustained investment and global collaboration are crucial for achieving the goal of a dengue-free world.

Importance: Understanding the epidemiology and cost burden of pediatric-onset inflammatory bowel disease (PIBD) is crucial for shedding light on its pathogenesis and making medical insurance decisions, particularly for very early-onset IBD (VEO-IBD).

Objective: To assess the epidemiology and cost burden of hospitalized PIBD and VEO-IBD.

Methods: A multicenter study identified hospitalized PIBD patients aged 0-18 years using the FUTang Updating medical REcords database from 28 hospitals in mainland China (2016-2021). Sociodemographic, clinical characteristics, length of stay (LOS), and expense data were analyzed. The cost burden was evaluated by dividing annual out-of-pocket expenditure by household disposable income. Clinical epidemiology and cost burden were compared between Crohn's disease (CD), ulcerative colitis (UC), IBD-unclassified (IBD-U), and different age groups.

Results: A total of 5230 hospitalized cases of PIBD were identified. Mean ages for CD (11.8 ± 3.9 years) and UC (8.0 ± 5.2 years) were significantly higher than those of patients with IBD-U (4.5 ± 4.1 years). Hospitalizations for CD showed a notable annual increase. Patients with CD had a shorter LOS (median 3 days) than those with UC (median 6 days) and IBD-U (median 7 days). The number of VEO-CD hospitalizations increased, while that of VEO-UC declined (2016-2021). The median total hospitalization expenses were $1006.7 for CD and $841.6 for UC. The average proportion of annual cost burden of IBD for each PIBD patient from 2016 to 2021 was 16.47%, 14.34%, 20.55%, 18.45%, 12.13%, and 11.16%, respectively, which has declined significantly since 2020.

Interpretation: This study provides insight into the clinical characteristics and cost burden of PIBD and VEO-IBD in China. Hospitalizations for PIBD, particularly for CD, are increasing. Guidelines and health insurance policies can help mitigate this cost burden.

The global incidence of myopia is increasing, and high myopia increases the risk of pathological myopia, which can lead to irreversible visual impairment, posing a significant global health concern. Artificial intelligence (AI) may be a solution to the myopia pandemic, with potential applications in early identification, risk stratification, progression prediction, and timely intervention to address unmet needs. AI has been developed to detect, diagnose, and predict the progression of myopia in both children and adults. In this review, the current state of AI technology applications in the field of myopia has been comprehensively reviewed, and the challenges, current development status, and future directions of AI have also been discussed, which hold great significance for the further application of AI in myopia management.

The incidence of juvenile-onset recurrent respiratory papillomatosis (JORRP) varies worldwide, and the lack of well-adopted guidelines for use in China suggests that patients with JORRP do not receive optimal care. In America, where data is available, pediatric patients undergo an average of 4 surgeries annually and a total of >40 surgeries in their lifetimes primarily due to this condition. It is widely accepted that timely diagnosis and implementation of scientifically sound and effective interventions can prevent JORRP progression and mitigate serious complications. Notably, evidence-based guidelines to coordinate care are lacking, and there is a need to standardize clinical practice to improve outcomes for patients. The International Pediatric Otolaryngology Group (IPOG) issued guidelines in 2020 to improve care for patients with JORRP. However, this guideline was majorly tailored to the healthcare system in Europe and America, posing a challenge to its adoption in China. To this effect, we assembled a guideline development working group to formulate guidelines for the diagnosis and treatment of JORRP tailored to the Chinese context. The working group consisting of multidisciplinary experts with experience in managing patients with JORRP undertook qualitative and quantitative studies, conducted two rounds of Delphi consensus, and carried out multiple systematic reviews/meta-analyses to provide 24 key recommendations to 12 questions of clinical interest. We anticipate that healthcare workers, including primary care physicians and specialists managing JORRP, will find the guidelines useful, and their utilization will translate to improved outcomes for patients with the disease.

Importance: Perampanel (PER) is used less extensively in children than in adults. Currently, there is a lack of data from PER clinical studies with large sample sizes in Chinese children and adolescents with epilepsy, especially those with refractory epilepsy.

Objective: To evaluate the effectiveness, retention, and safety of PER in the treatment of children and adolescents with epilepsy in China.

Methods: This was a multicenter, prospective, observational study. Children and adolescents with epilepsy who received PER as adjunctive therapy were included. The primary effectiveness endpoint was the proportion of patients achieving a ≥50% reduction in seizure frequency after 6 months of treatment compared to baseline. The secondary effectiveness endpoints included retention and seizure-free rates. The safety outcome was the incidence of treatment-emergent adverse events (TEAEs).

Results: A total of 240 patients with epilepsy were enrolled in the study. Prior to initiating PER treatment, approximately 87.9% of them took two or more antiseizure medications. After a 6-month treatment regimen with PER, 70.4% of the patients experienced a reduced seizure frequency of at least 50%, and 22.1% achieved complete seizure freedom. The retention rate was 90.2%. TEAEs were reported by 89 patients, leading to the discontinuation of PER in seven cases. No severe TEAEs were observed in this study.

Interpretation: Under routine clinical conditions, PER demonstrated good effectiveness and retention in Chinese children with epilepsy, particularly in those with refractory epilepsy.

Mycoplasma pneumoniae pneumonia (MPP) is the most common atypical pneumonia among children, with non-specific clinical manifestations. Despite various laboratory diagnostic methods, the diagnostic criteria remain inconsistent, potentially leading to missed or overdiagnosis. The incidence of severe and refractory cases of MPP is increasing, and there are issues with non-standard treatment in clinical practice. To standardize the diagnosis and treatment of MPP in Chinese children, the Subspecialty Group of Respiratory, the Society of Pediatrics, Chinese Medical Association, China National Clinical Research Center of Respiratory Diseases and the Editorial Board of Chinese Journal of Pediatrics jointly established a guideline expert group to formulate the "Evidence-Based Guideline for the Diagnosis and Treatment of Mycoplasma Pneumoniae Pneumonia in Children (2023)" based on both domestic and international research findings.

Retinoma, also referred to as retinocytoma, is a benign manifestation of biallelic retinoblastoma gene (RB1) inactivation. Genetic or epigenetic loss of retinoblastoma protein in maturing cone precursors induces genomic instability which leads to upregulation of senescence-associated p16^INK4a and p130, resulting in non-proliferative retinoma. When senescence pathways fail and genetic instability accumulates to a critical level through altered gene copies of oncogenes and tumor suppression genes, transformation into RB1 ^-/- retinoblastoma occurs. Thus, the management of retinoma involves frequent ophthalmic examination and imaging to monitor the size and characteristics of the tumor, ensure stability, and rule out malignant transformation. Key ophthalmoscopic features of retinoma often include a translucent whitish-gray retinal mass, calcification, retinal pigment epithelial alterations with well-defined margins, located typically around the lesion, as well as a zone of chorioretinal atrophy. This review aims to provide a comprehensive overview of this non-malignant tumor drawing from current understanding of its molecular genetics, clinical characteristics, diagnostic modalities, differential diagnosis, management, and prognosis. A deeper understanding of retinoma could offer valuable insights into how retinoblastoma develops and oncogenesis more broadly, paving the way for improved strategies to prevent and treat this malignant tumor.