Objective To evaluate the cost-utility of risdiplam in the treatment of pediatric spinal muscular atrophy （SMA） and analyze changes in patients’ out-of-pocket economic burden under the medical insurance scenarios. Methods A Markov model was developed from the healthcare system perspective to compare the long-term costs and quality-adjusted life years （QALYs） of risdiplam treatment versus conventional therapy. The incremental cost-utility ratio （ICUR） was calculated. One-way sensitivity analysis and probabilistic sensitivity analysis were conducted to assess the robustness of the model. On this basis, health insurance scenarios were simulated to evaluate patients’ individual economic burden. Results Base-case analysis showed that risdiplam treatment generated additional health benefits but substantially increased medical costs. The resulting ICUR did not demonstrate a clear cost-utility advantage under the current willingness-to-pay threshold. Sensitivity analyses indicated that the model results were relatively robust, with drug costs and key utility parameters being the main drivers of economic outcomes. Health insurance scenario analysis demonstrated that increasing reimbursement levels and reducing patient copayment rates could significantly alleviate patients’ financial burden. Conclusion Risdiplam for pediatric SMA faces economic challenges under the current health insurance payment framework in China. However, optimizing health insurance reimbursement policies and developing multi-tiered healthcare security mechanisms may improve its affordability and accessibility.

ObjectiveTo evaluate the methodological quality and reporting quality of published guidelines/consensuses on traditional Chinese medicine （TCM）/integrated traditional Chinese and Western medicine diagnosis and treatment of nonalcoholic fatty liver disease （NAFLD）， and to provide a basis for formulating guidelines/consensuses on TCM/integrated traditional Chinese and Western medicine diagnosis and treatment of NAFLD in the future. MethodsDatabases including PubMed， Embase， Web of Science， CNKI， Wanfang Data， and CBM and the websites of China Association of Chinese Medicine and China Association of Integrative Medicine were searched for related articles published up to September 1， 2024. Two clinical researchers independently assessed the methodological quality and reporting quality of the guidelines/consensuses on TCM/integrated traditional Chinese and Western medicine diagnosis and treatment of NAFLD by using Appraisal of Guidelines for Research and Evaluation Ⅱ （AGREE Ⅱ） and Reporting Items for Practice Guidelines in Healthcare （RIGHT）. ResultsA total of nine guidelines/consensuses were included after literature screening， with four guidelines and five expert consensuses. The scores of different domains assessed by AGREE Ⅱ for the nine guidelines/consensuses were as follows： scope and purpose （47.1%）， stakeholder involvement （41.0%）， rigor of development （21.6%）， clarity of presentation （40.2%）， applicability （19.0%）， and editorial independence （19.6%）. The recommendation level of the articles was B level （recommended after revision） for four articles and C level （not recommended） for five articles. The RIGHT assessment showed high reporting rates for “Basic Information” and “Background”， while other areas needed to be improved. Currently， there was no international standard for the guidelines/consensuses on TCM/integrated traditional Chinese and Western medicine diagnosis and treatment of NAFLD， and the quality of these guidelines/consensuses needed to be enhanced to ensure comprehensiveness and credibility. ConclusionThere is still potential for improving the quality of guidelines/consensuses on TCM/integrated traditional Chinese and Western medicine diagnosis and treatment of NAFLD， and AGREE Ⅱ and RIGHT checklists should be strictly followed to ensure the fairness， scientific rigor， and transparency of these guidelines/consensuses.

OBJECTIVES: To investigate the effects of live combined Bacillus subtilis and Enterococcus faecium (LCBE) on glucose and lipid metabolism in mice with type 2 diabetes mellitus (T2DM) and circadian rhythm disorder (CRD) and explore the possible mechanisms. METHODS: KM mice were randomized into normal diet (ND) group (n=8), high-fat diet (HFD) group (n=8), and rhythm-intervention with HFD group (n=16). After 8 weeks of feeding, the mice were given an intraperitoneal injection of streptozotocin (100 mg/kg) to induce T2DM. The mice in CRD-T2DM group were further randomized into two equal groups for treatment with LCBE (225 mg/kg) or saline by gavage; the mice in ND and HFD groups also received saline gavage for 8 weeks. Blood glucose level of the mice was measured using a glucometer, and serum levels of Bmal1, PER2, insulin, C-peptide and lipids were determined with ELISA. Colon morphology and hepatic lipid metabolism of the mice were examined using HE staining and Oil Red O staining, respectively, and fecal short-chain fatty acids (SCFAs) was detected using LC-MS; GPR43 and GLP-1 expression levels were analyzed using RT-qPCR and Western blotting. RESULTS: Compared with those in CRD-T2DM group, the LCBE-treated mice exhibited significant body weight loss, lowered levels of PER2, insulin, C-peptide, total cholesterol (TC) and LDL-C, and increased levels of Bmal1 and HDL-C levels. LCBE treatment significantly increased SCFAs, upregulated GPR43 and GLP-1 expressions at both the mRNA and protein levels, and improved hepatic steatosis and colon histology. CONCLUSIONS LCBE ameliorates lipid metabolism disorder in CRD-T2DM mice by reducing body weight and improving lipid profiles and circadian regulators possibly via the SCFAs/GPR43/GLP-1 pathway.
Animals ; Mice ; Lipid Metabolism ; Diabetes Mellitus, Type 2/metabolism* ; Enterococcus faecium ; Glucagon-Like Peptide 1/metabolism* ; Bacillus subtilis ; Diabetes Mellitus, Experimental/metabolism* ; Circadian Rhythm ; Blood Glucose/metabolism* ; Receptors, G-Protein-Coupled/metabolism* ; Fatty Acids, Volatile/metabolism* ; Male ; Chronobiology Disorders/metabolism*

Objective To study the relationship between marital status and prognosis of prostate cancer patients.Methods A total of 16 815 prostate cancer patients'data were extracted from the US SEER database,and the relationship between marital status and the prognosis of prostate cancer patients was assessed by using Log-rank unifactorial and multifactorial Cox risk-proportional models.Results ①At different stages of cancer,marital status can serve as an independent factor for tumor prognosis;②When the prostate-specific antigen(PSA)＜10ng/ml,the strongest correlation between marital status and prostate cancer survival rate was observed.There was a significant difference in tumor survival rate between different marital statuses with PSA＜10ng/ml and PSA levels between 10-20ng/ml;③ The survival curve and 5-year survival rate of patients with Gleason=7 points and Gleason ≥ 8 points in married group were significantly higher than those in unmarried group and divorced/widowed group;④ Patients who undergo surgical treatment have a longer survival time,and regardless of whether surgery is performed or not,the survival rate of married patients is significantly higher than that of patients with other marital statuses;⑤ In this study,the mortality risk of married prostate cancer patients was lower than that of patients with other marital statuses under different grouping conditions.Conclusion Marital status can be used as an independent prognostic factor for prostate cancer patients to a certain extent,and the mortality risk coefficient of married prostate cancer patients is lower than that of patients with other marital status.

Objective:To analyze the research status and comment of radiation-induced heart injury based on relevant literature analysis in China.Methods:A literature database was established with studies published from 1983 to 2021 as the data source from "China National Knowledge Infrastructure (CNKI) database", "Wanfang Database", "VIP Chinese Science and Technology Journal Database" and "China Biology Medicine Disc". The publication date, type and journal distribution of studied related to radiation-induced heart injury were quantitatively analyzed.Results:A total of 300 literatures were included. The chronological distribution of literatures: from 1983 to 2021, the number of literatures related to radiation-induced heart injury was gradually increased, and the overall trend was on the rise. The top three publication journals were Chinese Journal of Radiation Oncology, Chinese Journal of Radiation Medicineand Protection, and Chinese Journal of Cancer Prevention and Treatment. Literature type distribution: clinical research accounted for 48.50%, 25.58% for review, and 17.61% for animal experiment, respectively. The distribution of clinical research types: lung cancer, esophageal cancer and breast cancer were the most, followed by thymoma and lymphoma. In animal experiments, rat was the most frequently studied animal model, and the maximum radiation dose was 20 Gy. Conclusions:The number of studies related to radiation-induced heart injury is gradually increasing in China. The quantity of clinical studies is higher than that of animal experiments, most clinical studies focus on chest tumors, but there is great potential in animal experimental research. We still need to explore the pathogenesis of radiation-induced heart injury and develop drug prevention and intervention strategies.

Objective:To evaluate the quality, content and feasibility of health information on breast cancer treatment in Chinese websites, for reference in standardizing online health information.Methods:In January 2022, three Chinese search engines, namely Baidu, Sogou and Haosou, were used to search with " breast cancer" and " breast cancer treatment" as keywords. The first 50 links identified by each engine were accessed to screen breast cancer treatment health information websites. Subsequently, the DISCERN scoring tool was adopted to evaluate the quality of health information on the websites(16 items). The information content(10 items)assessment was conducted by referring to the " Guidelines and Specifications for Breast Cancer Diagnosis and Treatment of the Chinese Anti-Cancer Association(2019 Edition)". The feasibility of website health information was evaluated with Patient Education Materials Assessment Tool(PEMAT).Results:A total of 19 websites were included, and their service mode was mainly active information service(14 websites). The overall scoring for the quality of health information about breast cancer treatment was 2.0, with the highest score of " clarity of purpose" (3.4). According to the assessment results of information content, these websites were divided into 4 grades: excellent(3), good(11), fair(3), and poor(2). All the websites contained the information on clinical manifestations, etiology, examination and treatment of breast cancer. The overall feasibility of website health information was 44.5%, indicating incomplete feasibility.Conclusions:The Chinese website′s health information on breast cancer treatment is poor in quality and implementation. Hence, it′s urgent to further improve and standardize online health information, establishing a unified evaluation standard and actively exploring the new mode of " Internet+ Medical" service.

OBJECTIVE: To assess the association of CYP2C19 and CYP3A5 gene polymorphisms with the risk of myocardial infarction. METHODS: Five hundred patients with myocardial infarction and 500 healthy controls were randomly selected. Fluorescent PCR and Sanger sequencing were used to detect the CYP2C19 and CYP3A5 gene polymorphisms. Logistic regression was used to analyze the correlation between the polymorphisms and myocardial infarction. Quanto software was used to evaluate the statistical power. RESULTS: The two groups had significant difference in the frequency of AG, GG genotypes and A allele of the CYP2C19 gene rs4986893 locus and the AA, AG, GG genotypes and G allele of the CYP3A5 gene rs776746 locus ( P<0.05), but not in the frequency of genotypes and alleles of CYP2C19 gene rs4244285 and rs12248560 loci, and the AA genotype of the rs4986893 locus. After correction for age, gender, and body mass index, Logistic regression indicated that the AG genotype and A allele of the CYP2C19 gene rs4986893 locus, and the GG genotype and G allele of CYP3A5 gene rs776746 locus are associated with susceptibility of myocardial infarction, while rs4986893 GG genotype and AA and AG genotypes of rs776746 may confer a protective effect. Based on the sample size and allele frequency, analysis with Quanto software suggested that the result of this study has a statistical power of 99%. CONCLUSION CYP2C19 and CYP3A5 gene polymorphisms may increase the risk for myocardial infarction.
Cytochrome P-450 CYP2C19/genetics* ; Cytochrome P-450 CYP3A/genetics* ; Gene Frequency ; Genotype ; Humans ; Myocardial Infarction/genetics* ; Polymorphism, Genetic ; Polymorphism, Single Nucleotide

Glutaricacidemia type 1(GA1) is an autosomal recessive disease caused by reduced or missing glutaryl-CoA dehydrogenase activity which hamps metabolism of lysine, hydroxylysine and tryptophan. The catabolic products of glutarylcarnitine and glutaric acid are abnormally accumulated in the body, resulting in metabolic disorders which primarily lead to damage to the nervous system. Clinical manifestations of patients include macrocephaly, dystonia, dyskinesia, and developmental retardation. Acute encephalopathy may be induced in infants and young children due to infection, vaccination and surgery. For GA1 is a rare disease and its clinical manifestations are similar to other neurological diseases, it may be easily missed or misdiagnosed. To facilitate early diagnosis and treatment and improve the prognosis, this consensus was formulated by pediatric experts from the fields of endocrinology and genetic metabolism through full discussion and reference to the latest literature and guidelines home and abroad.

Objective To discuss the feasibility of biodegradable covered magnesium alloys stent for a rabbit model of lateral aneurysm in the common carotid artery (CCA). Methods The left CCA was ligated in 20 conventional New Zealand rabbits. MRA of the neck was performed 1 month after ligation. The rabbits with thickening of the right CCA and non-or slight thickening of the bilateral vertebral arteries were selected for lateral aneurysm model making. The venous pouch and the right CCA wall by discontinuous extroversion sutures, to form a lateral aneurysm model. The biodegradable covered magnesium alloys stents or Willis covered stents were inserted two weeks after model making. Angiography was performed prior to the procedure, 3, 6 and 12 months after stent implantation to evaluate the disappearance of the aneurysms and patency of the right CCA. The degradation behaviour is invastagated with molybdenum target 2 weeks, 1, 2, 4, 6,9 and 12 months after stent placement. Results The left CCAs were successful ligated in all rabbits. MRA 1 month after ligation showed thickening of the right CCA and non-thickening of the bilateral vertebral arteries in 17 of 20 rabbits. In these animals, the CCA lateral aneurysm model was deemed successful, and biodegradable covered magnesium alloys stents and Willis covered stents were implanted in 9 and 8 aneurysms, respectively. DSA after biodegradable covered magnesium alloys stent placement displayed disappearance of the aneurysms and patency of the CCA in all 9 rabbits during follow-ups. DSA 3 months after Willis stent placement displayed patency of the CCA in 7 rabbits and occlusion of the artery in one. No occlusion of the right CCA was observed on angiography at 6 and 12 months. The degradation of the biodegradable covered magnesium alloys stent was investigated with molybdenum target during follow-ups, and no changes was observed in Willis covered stent. Conclusion Biodegradable covered magnesium alloys stent is a feasible approach for the treatment of a lateral aneurysm in the right CCA.

Objective To identify differentially expressed N-linked glycoproteins between hepatocellular carcinoma ( HCC) and adjacent non-tumorous liver tissues .Methods N-linked glycoproteome was extracted by multi-lectin affinity chromatography comprising concanavalin A (ConA), lentil lectin (LCH), and snowdrop lectin (GNA) and subsequently subjected to two-dimensional electrophoresis ( 2DE ) and mass spectrometry ( MS ) for identification of differential glycoproteins between 10 pairs of HCC and adjacent non-cancer tissue .Western blotting was used to verify different expression of human liver carboxylesterase 1 (hCE1), haptoglobin (HP)and cathepsin D (CD).Invasion potential in vitro was examined after si-RNA mediated CD gene scilencing .Results LC-ESI-MS/MS identified a total of 28 differentially expressed glycoproteins (14 up-regulation and 14 down-regulated).Western blotting detected consistent down-regulation of hCE1 and HP, and up-regulation of pro-cathepsin D (pCD) in HCC.Up-regulation of ConA-binding CD (ConA-CD), however , was verified in HCC only after ConA-CD enrichment by ConA chromatography .Down-regulation of CD expression mediated by CD-siRNA markedly inhibited the in vitro invasive potential of SNU449 and SNU473.Conclusion Dysregulation of HP , hCE1 expression and alteration of glycans linked to CD may play crucial roles in pathogenesis of HCC.