World Journal of Pediatrics最新文献

Background: Pediatric inflammatory bowel disease (pIBD) often begins early in life, progresses rapidly, and is associated with impaired growth and delayed development. These challenges demand treatment strategies that address both intestinal inflammation and the broader developmental needs of children.

Data sources: This review summarizes current advances in small-molecule therapies for pIBD based on published clinical trials, real-world studies, and mechanistic investigations retrieved from PubMed and clinical trial registries. Special emphasis is placed on Janus kinase (JAK) inhibitors and sphingosine-1-phosphate (S1P) modulators, which represent the main translational research focus in pediatric IBD.

Results: JAK inhibitors such as tofacitinib and upadacitinib have demonstrated promising efficacy in pediatric patients with refractory disease, although their use remains off-label worldwide. Long-term safety concerns persist, including infection risk, developmental effects, and potential risks of malignancy or major adverse cardiovascular events. S1P modulators such as ozanimod are under clinical evaluation in children, but robust long-term data are still lacking. Emerging technologies such as single-cell and spatial profiling have begun to reveal age-dependent remodeling of gut immune architecture, emphasizing the importance of developmentally informed therapeutic approaches.

Conclusions: Small-molecule therapies offer a promising and mechanistically precise direction for the management of pIBD. Future progress will depend on age-specific clinical trials, physiologically based pharmacokinetic modeling, and biomarker discovery through integrated multiomics. Collaborative multicenter research is essential to optimize the safety and efficacy of these agents in children.

Background: Undifferentiated autoinflammatory diseases (uAIDs) represent a newly recognized category of AIDs that fail to meet the diagnostic criteria for established monogenic or polygenic AIDs. This systematic review aims to clarify the prevalence of uAIDs and summarize the clinical features and treatment responses of affected patients.

Methods: A systematic literature review of PubMed, Web of Science, EMBASE, Cochrane and Scopus was performed in accordance with the 2020 PRISMA guidelines. English-language studies mentioning uAIDs were included for analysis. The classification criteria used for the diagnosis of uAIDs, as well as the demographic data, clinical manifestations and treatment responses of patients, were extracted and analyzed. Study quality was assessed via the tool developed by Hoy et al. The primary outcome, the pooled prevalence of uAIDs, was calculated via common- or random-effects meta-analyses, as appropriate.

Results: Thirty-five articles were included. Although termed variably, the definition of uAIDs consistently includes the following key points: patients with clinical signs and symptoms of systemic inflammation; patients who fail to meet the criteria for any well-defined polygenic AIDs; next-generation sequencing is negative or inconclusive; and other confounding conditions are excluded. The pooled prevalence of uAIDs among patients with AIDs was 21% (95% confidence interval: 14-29). Fever, arthralgia and abdominal pain were the most common clinical manifestations. Colchicine was the most frequently reported immunosuppressant in the literature, while interleukin (IL)-1-targeted biologics achieved remission in 70% of treated patients.

Conclusions: A diagnosis of uAIDs should be considered in patients who present with autoinflammatory features, such as fever, arthralgia and abdominal pain and who do not meet the diagnostic criteria for monogenic or polygenic AIDs or other confounding conditions. The responsiveness to IL-1-targeted biologics implies that the IL-1 pathway may be a potential gateway in uAIDs. Future prospective studies with standardized criteria are needed to overcome current limitations of exclusion-based diagnosis and study heterogeneity in uAIDs research.

Background: As an emerging type of immune cell therapy, chimeric antigen receptor natural killer (CAR-NK) cells have shown great potential in the treatment of both tumors and autoimmune diseases. Compared with CAR-T cells, CAR-NK cells have a lower risk of cytokine release syndrome and neurotoxicity and have the potential for "off-the-shelf" treatment from a wide range of sources including peripheral blood, umbilical cord blood, induction blood, and pluripotent stem cells (iPSCs).

Data sources: We systematically review the basic biological characteristics of CAR-NK cells, strategies for CAR construct design and optimization, cell sources and expansion techniques, gene transfer methods and the latest progress in preclinical and clinical research.

Results: CAR-NKs have shown good safety and preliminary efficacy in treating hematological malignancies, solid tumors, autoimmune diseases and infectious diseases. Challenges relating to persistence in the body and adaptability to the microenvironment of solid tumors remain, With continuous optimization of the genetic engineering, combination therapy and personalized strategies, CAR-NK therapy is expected to be an important pillar of the next generation of immunotherapies and promote the development of precision medicine.

Conclusions: CAR-NK cell therapy, with its superior safety profile and "off-the-shelf" potential, has emerged as a highly promising star in immunotherapy for tumors and autoimmune diseases. Although challenges remain in its in vivo persistence and efficacy against solid tumors, continuous engineering optimization and combination strategies are expected to advance the development of precision medicine.

Background: Ultrasound and serum matrix metalloproteinase-7 (MMP-7) hold great value in distinguishing biliary atresia (BA) from other cholestatic diseases. This study aims to assess the accuracy of an artificial intelligence (AI) based diagnostic model of ultrasound combined with serum MMP-7 in discriminative diagnosis of BA.

Methods: This is a multicenter diagnostic study involving six medical centers in China. Patients with obstructive jaundice were enrolled. A set of morphological operators were employed to extract features of the ultrasound images to construct an AI algorithm. Logistic regression model was established with validation.

Results: Two cohorts with a total of 348 children with obstructive jaundice were recruited from January 2020 to April 2023. A retrospective cohort of 187 infants served as a training cohort; this included 56 BA and 131 non-BA patients. Serum MMP-7 testing model yielded an area under the receiver-operating characteristic curve (AUROC) of 0.916 [95% confidence interval (CI) = 0.876-0.956], sensitivity of 94.6% (95% CI = 85.1%-98.9%), specificity of 88.6% (95% CI = 81.8%-93.5%), and accuracy of 90.4% (95% CI = 85.2%-94.2%). Values for ultrasound testing model were 0.945 (95% CI = 0.902-0.987), 98.2% (95% CI = 90.5%-99.9%), 91.6% (95% CI = 85.5%-95.7%), and 93.6% (95% CI = 89.1%-96.6%), respectively. The combined AI model obtained an AUROC of 0.985 (95% CI = 0.971-0.999), sensitivity of 98.2% (95% CI = 90.5%-99.9%), specificity of 93.1% (95% CI = 84.4%-96.4%), and accuracy of 94.7% (95% CI = 90.4%-97.4%), respectively. Performance was confirmed using a multicenter prospective validation cohort of 161 patients that included 100 BA cases.

Conclusion: An AI model combining ultrasound and serum MMP-7 demonstrated robust high sensitivity and specificity in the differential diagnosis of BA.

Background: Childhood cancer survivors (CCSs) are at increased risk of long-term treatment-related complications. Although international guidelines support risk-based long-term follow-up (LTFU) care, its standardized implementation in China has been limited. To address this gap, the National Children's Medical Center-Shanghai convened a multidisciplinary expert panel to develop disease-based LTFU care plans tailored to the Chinese healthcare context.

Methods: Guided by established international frameworks (Children's Oncology Group, International Guideline Harmonization Group, and PanCareFollowUp), an expert group representing 25 institutions across China developed consensus-based LTFU care plans for common pediatric cancer patients and post-hematopoietic cell transplant survivors. Each care plan includes core components: a treatment summary, risk stratification for late effects, recommended surveillance, psychosocial evaluation, and lifestyle guidance. The panel also developed a consensus on the specific roles of oncologists, primary care providers, and subspecialists.

Results: Finalized care plans provide structured, risk-adapted follow-up pathways for CCSs. The model emphasizes multidisciplinary collaboration, clinical feasibility, and scalability across diverse settings. As part of the care process, a centralized survivorship database has been integrated to facilitate clinical use and data collection. This system supports the generation of standardized treatment summaries and longitudinal documentation of late effects across the continuum of survivorship care. Tools, such as clinician checklists and survivor education templates, were also developed to support clinical use and promote consistency across institutions. A list of outcome metrics was proposed to evaluate the implementation outcomes of this initiative.

Conclusions: This expert consensus establishes an innovative, nationally coordinated, disease-specific LTFU care framework for CCSs in China. This study provides a practical foundation for improving survivorship care quality and guiding clinical practice nationwide. This model can serve as a blueprint for other low- and middle-income countries seeking to strengthen LTFU care for CCSs.

Background: Posterior drooling and saliva aspiration are hidden threats to progressive respiratory morbidity in patients with cerebral palsy (CP). This study aimed to identify and synthesize current evidence on the definitions, prevalence, pathophysiology, diagnosis and treatment of posterior drooling and saliva aspiration in children with CP.

Methods: This was a scoping review. The inclusion criteria were articles focused on the objectives of the study; published between January 2000 and June 2025; written in English, Portuguese, Spanish, French or Italian; and whose full texts were available. The PubMed, Virtual Health Library, Scielo, Cochrane Library, Scopus and EMBASE databases were searched.

Results: A total of 1195 citations were identified by our search strategy, with 637 remaining after the removal of 558 duplicates. After screening titles and abstracts, 304 citations progressed to full-text review. A total of 86 articles met the eligibility criteria and were included in the review. While 28 articles were excluded because they lacked access to full texts and/or were not written in the selected languages, only 114 (9.5%) studies involved posterior drooling/saliva aspiration.

Conclusions: A large discrepancy was evident, with a much larger volume of studies on anterior drooling than on posterior drooling/saliva aspiration. This article highlights the need for more research and greater emphasis on these conditions in pediatric clinical practice. Early and reliable diagnosis and proactive interventions are essential to prevent progressive lung injury, lower respiratory tract infections, hospitalizations, impaired quality of life, and premature death.

Background: Spinal cord injury is a catastrophic medical condition and a growing global public health priority, with divergent etiologies, risk factors, and epidemiology. Comprehensive analyses of trends in pediatric spinal cord injury burden both in China and globally are lacking.

Methods: We investigated temporal trends in pediatric spinal cord injury burden [incidence, prevalence, and years lived with disability (YLDs)] from 1990 to 2021 and projected future burden to 2045 using Global Burden of Disease (GBD) 2021 data. The year with the most significant changes in trends was identified via joinpoint regression analysis. Age-standardized rates, average annual percentage changes (AAPC), and subgroup analyses by injury location, sex, age, and sociodemographic index were calculated. To project burden data to 2045, we employed a Bayesian age‒period‒cohort model.

Results: Our analyses revealed that global pediatric spinal cord injury incidence [AAPC: - 1.13; 95% confidence interval (CI): - 1.49 to - 0.76], prevalence (AAPC: - 1.13; 95% CI: - 1.16 to - 1.11), and YLDs rates (AAPC: - 1.37; 95% CI: - 1.41 to - 1.35) decreased significantly from 1990 to 2021. The highest burden was observed in males, adolescents (15-19 years), and high-sociodemographic index regions; falls were the leading cause. China exhibited similar declining trends (e.g., incidence AAPC: - 1.33; 95% CI: - 1.71 to - 0.83). It is predicted that by 2045, the global trend of pediatric spinal cord injury will decline; China will exhibit an upward trend.

Conclusions: Persistent disparities in pediatric spinal cord injury disease burden and causes necessitate targeted prevention strategies, optimized rehabilitation services, and equitable resource allocation. This is particularly important in high-risk groups and regions with rising burdens.