There is a substantial unmet need for treatments in the field of pediatric rare diseases, and investigator initiated trial(IIT) provide a critical pathway for testing and developing new drugs or treatment strategies. However, healthcare institutions, when conducting such research, must address compliance risks related to project approval, contract management, data protection, and conflict of interest management. This study aims to analyze the particularities and challenges of IIT in pediatric rare diseases, review relevant regulations and regulatory requirements, and provide healthcare institutions with a reference framework for compliance risk management to maximize the benefits of IIT. Based on literature review, analysis of laws and regulations, practical work experience, and frameworks from other institutions, we summarize the unique aspects of pediatric rare disease IIT in terms of participant characteristics, innovative technologies, and organizational structures.On this basis, targeted compliance management recommendations are proposed, which include establishing a risk rating and full-cycle risk monitoring mechanism, a consent and ethical review mechanism tailored to pediatric participants, a robust contract management mechanism, a comprehensive data security management mechanism, and a multidisciplinary team and multi-channel compensation mechanism. The study concludes that healthcare institutions, funders, and other collaborating entities should implement compliance management in line with the characteristics of IIT to ensure the safety and effectiveness of research and facilitate innovation and development in the treatment of pediatric rare diseases.

Objective To analyze the current research status,hotspots,and trends in the field of mechanical ventilation weaning.Methods Literatures in the field of mechanical ventilation wea-ning were retrieved from the Web of Science Core Collection,PubMed,China National Knowledge In-frastructure(CNKI),and Wanfang Data Knowledge Platform from January 1,2010 to November 27,2024.CiteSpace software was used to analyze the research status and future trends.Results A total of 705 English and 494 Chinese eligible articles were included in this study.The author with the most publications was Laurent Brochard(14 articles).Research hotspots focused on mechanical ventila-tion,weaning,non-invasive ventilation,respiratory failure,etc.New research hotspots have shifted towards mechanical ventilation weaning failure,weaning complications,and prolonged weaning.Con-clusion Further research is needed on weaning failure,complications,and prolonged weaning,par-ticularly in the direction of invasive-high-flow oxygen therapy sequential therapy,which will help opti-mize clinical treatment plans and reduce patients' suffering and economic burdens.

Despite primary glomerulonephritis (PGN) being a leading cause of chronic kidney disease and end-stage renal disease, specific and sensitive biomarkers for the early detection and monitoring of this condition are lacking. We evaluated the value of the combined detection of serum cystatin C (CYSC), β2-microglobulin (β2-MG), and urine transferrin (TRF) for diagnosing early-stage PGN. From May 2021 to May 2023, we enrolled 105 patients in our hospital as the observation group and 50 healthy volunteers as the control group. Their serum expression levels of CYSC, β2-MG, and TRF were evaluated. We plotted separate ROC curves and calculated the area under the curve (AUC) values of CYSC, β2-MG, and TRF to assess their diagnostic performance in PGN. The levels of CYSC, β2-MG, and TRF were significantly higher (P < 0.05) in the observation group than in the healthy control group. CYSC, β2-MG, and TRF were expressed at significantly higher levels in G2, G3a, and G3b of PGN than in G1. The combined use of CYSC, β2-MG, and TRF as biomarkers could significantly improve the early diagnosis and monitoring of PGN and may lead to better patient outcomes by facilitating earlier intervention and treatment strategies.

Objective To explore the detection rate and distribution characteristics of gene mutations in pa-tients with non-small cell lung cancer,and to analyze their relationship with clinicopathological characteristics.Methods The genetic testing results and clinicopathological data of 213 patients with non-small cell lung cancer who underwent high-throughput genetic testing at the Gene Detection Center,Changchun Cancer Hos-pital from April 2020 to December 2023 were collected.High-throughput sequencing technology was used to detect mutations in 26 genes,and the relationship between the mutation frequency and its distribution and the clinicopathological characteristics of patients was analyzed.Results Among 213 patients with non-small cell lung cancer,192 cases(90.14％)had at least one gene mutation detected.Among them,the genes with rela-tively high mutation frequencies were TP53(60.56％),EGFR(46.48％),KRAS(14.55％),ALK fusion(11.74％),and PIK3CA(8.92％).There were 132 cases(30.28％)of evidence level for Class 1 drugs and 11 cases(2.52％)of evidence level for Class 2 drugs.The incidence of EGFR gene mutations was higher in women,non-smokers and patients with lung adenocarcinoma(P＜0.05).TP53 mutations usually occured in women,smokers and patients with stage Ⅳ.ALK mutations were more common in young patients,while KRAS mutations were more frequently seen in male smokers.Conclusion Analyzing the distribution charac-teristics of gene mutations in non-small cell lung cancer and their relationship with clinicopathological charac-teristics can provide a scientific basis for further optimizing genetic testing for patients with non-small cell lung cancer and offer guidance for clinical treatment.

Objective:To investigate the pathway of self-disclosure to posttraumatic growth in patients after cervical cancer surgery, and to provide reference for improving the level of posttraumatic growth in patients.Methods:A convenient sampling method was used to investigate 300 patients with cervical cancer after surgery in Shandong Provincial Hospital Affiliated to Shandong First Medical University from June to November 2022 by using general data questionnaire, Distress Disclosure Index (DDI), Connor-Davidson Resilience Scale (CD-RISC), the Shortened Chinese Version of the Family Resilience Assessment Scale (FRAS-C) and the Posttraumatic Growth Inventory (PTGI) in a cross-section study.Results:A total of 290 valid questionnaires were collected, with an effective recovery rate of 96.7%. The patients were aged 23-70(48.13 ± 10.39) years. The scores of self-disclosure, resilience, family resilience and posttraumatic growth were (46.41 ± 9.82), (67.06 ± 14.63), (108.18 ± 11.06) and (58.24 ± 17.86) respectively. The results of pathway analysis showed that self-disclosure could not only directly predict posttraumatic growth, but also indirectly predict posttraumatic growth through the mediating role of resilience and family resilience, and the chain mediating role of resilience and family resilience, respectively. The direct effect of self-disclosure on posttraumatic growth was 0.236(95% CI 0.138-0.335), and the chain mediating effect of family resilience and resilience between self-disclosure and posttraumatic growth was 0.036(95% CI 0.018-0.060). Conclusions:Medical staff should not only consider the direct influence of self-disclosure on posttraumatic growth, but also pay attention to improve the resilience and family flexibility of patients after cervical cancer surgery, so as to promote their posttraumatic growth.

Objective:To investigate the application value of nectin-4 targeting radiotracer 68Ga-N188 in the diagnosis of pancreatic cancer. Methods:The prospective study was conducted. The clinicopathologic data of 16 patients diagnosed as pancreatic cancer on enhanced computed tomography (CT) who were admitted to the Peking University First Hospital from August to December 2022 were collected. There were 9 males and 7 females, aged (62±8)years. All patients underwent 18F-flurodeoxyglucose ( 18F-FDG) and 68Ga-N188 positron emission tomography (PET)/CT examination. Observation indicators: (1) distribution of 68Ga-N188 in different tissues and tumor primary lesion of patients; (2) expression of Nectin-4 and uptake of 68Ga-N188 in pancreatic cancer; (3) comparison of examination results between 68Ga-N188 and 18F-FDG PET/CT. Measurement data with normal distribution were represented as Mean± SD, and comparison between groups was conducted using the independent sample t test. Count data were described as absolute numbers or percentages. Results:(1) Distribution of 68Ga-N188 in different tissues and tumor primary lesion of patients. Results of PET/CT examination showed that in 1 hour after injection, the maximum standard uptake value (SUVmax) and mean standard uptake value (SUVmean) of 68Ga-N188 in fat, muscle, skin, and brain tissues of 16 patients were 0.40±0.16 and 0.25±0.09, 0.68±0.20 and 0.44±0.12, 0.39±0.14 and 0.28±0.11, 0.09±0.04 and 0.05±0.02, respectively. In the tissues of the esophagus, liver, spleen, and pancreas, the above indicators were 1.53±0.48 and 1.16±0.31, 1.49±0.45 and 0.91±0.30, 1.40±0.30 and 1.02±0.24, 1.24±0.31 and 0.96±0.25, respectively. In tumor primary lesion, the above indicators were 3.28±1.02 and 2.14±0.62, respectively, showing significant differences in SUVmax and SUVmean compared with pancreatic tissue ( t=8.03, 6.75, P<0.05). The tumor background ratio in tumor primary lesion based on SUVmax was 1.82±0.58. (2) Expression of Nectin-4 and uptake of 68Ga-N188 in pancreatic cancer. Results of immunohistochemical staining in 16 patients showed that there were 7 patients with high Nectin-4 expression and 9 patients with low Nectin-4 expression. Results of PET/CT examination showed that the SUVmax of 68Ga-N188 in tumor primary lesion of the 7 patients with high Nectin-4 expression and 9 patients with low Nectin-4 expression were 3.77±1.10 and 2.64±0.68, showing a significant difference between them ( t=2.64, P<0.05). The SUVmax of 18F-FDG in tumor primary lesion of the 7 patients with high Nectin-4 expression and 9 patients with low Nectin-4 expression were 6.73±3.24 and 6.43±3.45, showing no significant difference between them ( t=0.17, P>0.05). (3) Comparison of examination results between 68Ga-N188 and 18F-FDG PET/CT. Of the 16 patients, cases with positive results of tumor primary lesion on 68Ga-N188 and 18F-FDG PET/CT were 14 and 11, respectively, for the 14 pancreatic cancer patients diagnosed by postoperative histopathology. Among them, cases with positive results of tumor primary lesion on 68Ga-N188 and 18F-FDG PET/CT were 3 and 1 for the 3 pancreatic cancer patients receiving evaluation for chemotherapy. The SUVmax of 18F-FDG in tumor primary lesion of the 3 patients with chemotherapy and the 11 patients without chemotherapy were 2.80±0.69 and 6.97±2.11, showing a significant difference between them ( t=3.29, P<0.05). The SUVmax of 68Ga-N188 in tumor primary lesion of the 3 patients with chemotherapy and the 11 patients without chemotherapy were 3.38±1.12 and 2.93±0.50, showing no significant difference between them ( t=0.66, P>0.05). Cases with positive results of lymph node metastases in 68Ga-N188 and 18F-FDG PET/CT were 6 and 4, respectively, for the 6 pancreatic cancer patients diagnosed with lymph node metastases by postoperative histopathology, and the SUVmax of 68Ga-N188 and 18F-FDG in lymph node metastases were 2.25±1.12 and 4.02±1.27. Conclusion:68Ga-N188 PET/CT can be used for imaging diagnosis of tumor primary lesion and lymph node metastases of pancreatic cancer.

Objective:To build a systematic and standardized nutrition management plan for patients with chronic kidney disease.Methods:Based on the nutrition care procedure and model, a preliminary draft of a nutrition management plan for chronic kidney disease patients was developed through a literature search, quality evaluation, and group discussions. After two rounds of expert consultation and revision of the preliminary draft of the nutrition management plan, the final plan was formed.Results:A total of 32 experts were invited to complete two rounds of consultation. In two rounds of expert consultation, 32 questionnaires were distributed, and 32 and 31 valid questionnaires were collected, with valid response rates of 100.0% and 96.9%, respectively. The expert authority coefficients were 0.853 and 0.871, respectively. The final nutrition management plan for chronic kidney disease patients included six first-level items of nutrition management personnel: nutrition risk screening, nutrition assessment, nutrition treatment, nutrition monitoring, and nutrition health education, with 23 second-level and 52 third-level items.Conclusions:The constructed nutrition management plan for chronic kidney disease patients is scientific and can provide a reference for nutrition guidance.

Anaplastic thyroid cancer (ATC) is the most malignant thyroid cancer with a low incidence but high mortality. ATC is highly aggressive, rapidly progressing, and has poor prognosis. Current treatment options is not efficacious, so there is an urgent need to investigate its pathogenesis to update the treatment and improve the survival rate. Previous studies have found that most ATC can develop from well-differentiated thyroid cancer, and BRAF and RAS mutations are the key driving factors of ATC. TP53, PI3K pathway, PTEN, TERT, SWI/SNF complex Subunit, NF2 and other mutations also play an important role in the occurrence of ATC. Recent studies have found that single gene mutation is often not sufficient to drive the occurrence of ATC, and ATC is usually developed from the accumulation of multiple mutations in well-differentiated thyroid cancer. Therefore, this paper reviews the role of common combined mutations in ATC, deepens the understanding of the pathogenesis, and provides a basis for finding effective therapeutic targets.

Objective:To investigate the role of serum hepatitis B virus RNA (HBV RNA) in predicting HBeAg serological conversion in children with chronic hepatitis B.Methods:175 children aged 1~17 years with chronic hepatitis B who received interferon α (IFNα) for 48 weeks were selected. Patients were divided into HBeAg seroconversion and non-conversion based on whether HBeAg seroconversion occurred at 48 weeks of treatment.T-test and Mann-Whitney U test were used to compare between groups; chisquare test or Fisher exact probability method was used to compare the frequency between groups of classified variables; and Pearson correlation was used to analyze the correlation between indicators. Univariate and multivariate logistic regression analyses were used to identify influencing factors associated with HBeAg serological conversion. The predictive effect of HBV RNA, HBV DNA, and HBsAg on HBeAg serological conversion was compared and analyzed by the receiver operating characteristic curve (ROC).Results:The seroconversion rate of HBeAg at 48 weeks was 36.0% (63/175). The reduction in HBVRNA levels from baseline to the 12th, 24th, 36th, and 48th weeks of antiviral therapy was significantly greater in the HBeAg serological conversion group than that in the non-conversion group, and the difference was statistically significant between the two groups (P < 0.05). Univariate and multivariate regression analyses showed that age and a decline in HBV RNA levels at week 12 were independent predictors of HBeAg serological conversion. The area under the ROC curve (AUROC) of HBV RNA decline at week 12 was 0.677(95% CI∶0.549-0.806, P = 0.012), which was significantly better than the same period of AUROC of HBV DNA (0.657, 95% CI∶0.527-0.788, P = 0.025) and HBsAg (0.660, 95% CI∶0.526-0.795, P = 0.023) decline. HBV RNA levels decreased (>1.385 log10 copies/ml) at week 12, with a positive predictive value of 53.2%, a negative predictive value of 72.2%, a sensitivity of 77.4%, and a specificity of 57.9% for HBeAg seroconversion. Conclusion:HBV RNA level lowering during the 12th week of antiviral therapy can serve as an early predictor marker for HBeAg serological conversion in children with chronic hepatitis B.