1.Cost-effectiveness analysis of cefiderocol for the treatment of confirmed or suspected carbapenem-resistant Gram-negative bacteria serious infections
Yuan GONG ; Shuo KANG ; Yibing HOU ; Xiaohui WANG ; Ying NIE ; Jing WANG ; Zhenhua PAN
China Pharmacy 2026;37(2):192-197
OBJECTIVE To evaluate the cost-effectiveness of cefiderocol versus best available therapy (BAT) or standard-of- care (SOC) for the treatment of confirmed or suspected carbapenem-resistant Gram-negative bacterial (CRGNB) serious infections from the perspective of the Chinese healthcare system, and to explore its reasonable pricing. METHODS A decision tree model was constructed based on data from two phase Ⅲ clinical trials (CREDIBLE-CR and GAME CHANGER) to simulate the cost- effectiveness of cefiderocol in two scenarios: salvage therapy for confirmed CRGNB infection (scenario 1) and empirical therapy for suspected CRGNB infection (scenario 2). The primary outcome measure was the incremental cost-effectiveness ratio (ICER). The willingness-to-pay (WTP) was set at 1 to 3 times China’s per capita GDP in 2024. To verify the robustness of the results, one- way and probabilistic sensitivity analyses were conducted, and based on these, a reasonable price range for cefiderocol in the Chinese market was explored. RESULTS The results for scenario 1 showed that the clinical cure rate in the cefiderocol group was higher than that in the BAT group (47.50% vs. 34.21%), but its ICER was 415 065.03 yuan per cured case, exceeding three times China’s GDP per capita. Scenario 2 revealed that the ICER for cefiderocol relative to SOC was as high as 1 362 446.16 yuan per cured case, far exceeding the WTP. Sensitivity analysis indicated that the treatment duration and price of cefiderocol were key factors affecting its cost-effectiveness. In the two scenarios described above, the unit price of cefiderocol must fall below 683.47 and 242.00 yuan/g, respectively, to be considered cost-effective. CONCLUSIONS Based on the current market price, cefiderocol lacks sufficient cost-effectiveness for treating confirmed or suspected CRGNB serious infections within China’s healthcare system. To improve its accessibility, price negotiations or a tiered medical insurance payment strategy are required.
2.Rapid health technology assessment of serplulimab in the first-line treatment of small-cell lung cancer
Yibing HOU ; Shuo KANG ; Yuan GONG ; Xiaohui WANG ; Ying NIE ; Huanlong LIU
China Pharmacy 2025;36(11):1405-1410
OBJECTIVE To evaluate the efficacy, safety and cost-effectiveness of serplulimab as a first-line treatment of small- cell lung cancer (SCLC), and provide an evidence-based basis for drug selection in hospitals. METHODS Rapid health technology assessment was adopted; PubMed, Cochrane Library, Embase, CNKI, Wanfang, VIP and official websites of domestic and international health technology assessment agencies were systematically searched from the inception to Oct. 2024. Two reviewers independently screened the literature, assessed the quality of included studies and carried out the qualitative analysis according to the inclusion and exclusion criteria. RESULTS A total of 13 systematic reviews/meta-analyses and 9 economic studies were included, and the literature quality was generally good. In terms of effectiveness, compared with chemotherapy alone, serplulimab combined with chemotherapy significantly improved progression-free survival, overall survival, and objective response rate in patients with SCLC. In terms of safety, serplulimab combined with chemotherapy showed no significant difference in the incidence of ≥3 grade adverse events compared with chemotherapy alone in the treatment of SCLC, indicating a good safety profile; compared with combination therapies involving other immunosuppressive agents, the incidence rate of adverse events was also lower. In terms of cost-effectiveness, compared with chemotherapy alone, serplulimab combined with chemotherapy is not cost- effective, which may be related to the high price of serplulimab. CONCLUSIONS Serplulimab is effective and safe in the treatment of SCLC, but has no obvious advantage in terms of cost-effectiveness.
3.Efficacy and safety of albumin-binding paclitaxel combined with PD-1 inhibitors in the treatment of bone and soft tissue sarcoma after first-line therapy failure
HUANG Zhen ; LIU Weifeng ; LI Yuan ; XU Hairong ; ZHANG Qing ; HAO Lin ; NIU Xiaohui
Chinese Journal of Cancer Biotherapy 2025;32(11):1169-1174
[摘 要] 目的:探讨白蛋白结合型紫杉醇联合PD-1抑制剂用于治疗一线化疗失败的骨与软组织肉瘤的疗效及安全性。方法:回顾性分析北京积水潭医院骨肿瘤科2017年8月至2020年8月收治的一线化疗失败的晚期骨与软组织肉瘤患者。患者接受白蛋白结合型紫杉醇(125~140 mg/m2,第1天和第8天)与PD-1抑制剂(信迪利单抗或特瑞普利单抗,每21 d一次)联合治疗。每2个治疗周期评估1次疗效,按RECIST 1.1标准评估肿瘤疗效,按NCI-CTCAE5.0标准评估不良反应。结果:共20名患者纳入研究,完成1至8个治疗周期,中位治疗周期数为3个。所有患者均可评估疗效,完全缓解4例(20%),部分缓解0例,稳定9例(45%),疾病进展7例(35%)。客观缓解率(ORR)为20%,疾病控制率(DCR)为65%。中位无进展生存期(PFS)为3.0个月。治疗期间主要不良反应包括2级白细胞减少(40%)、1-2级神经毒性反应(20%),以及2级甲状腺功能减退(10%)。结论:白蛋白结合型紫杉醇联合PD-1抑制剂治疗为一线化疗失败的晚期骨与软组织肉瘤患者提供了一种潜在的治疗选择,其不良反应可控,值得开展更大样本的前瞻性研究进一步验证其疗效。
4.Single-cell transcriptomics identifies PDGFRA+ progenitors orchestrating angiogenesis and periodontal tissue regeneration.
Jianing LIU ; Junxi HE ; Ziqi ZHANG ; Lu LIU ; Yuan CAO ; Xiaohui ZHANG ; Xinyue CAI ; Xinyan LUO ; Xiao LEI ; Nan ZHANG ; Hao WANG ; Ji CHEN ; Peisheng LIU ; Jiongyi TIAN ; Jiexi LIU ; Yuru GAO ; Haokun XU ; Chao MA ; Shengfeng BAI ; Yubohan ZHANG ; Yan JIN ; Chenxi ZHENG ; Bingdong SUI ; Fang JIN
International Journal of Oral Science 2025;17(1):56-56
Periodontal bone defects, primarily caused by periodontitis, are highly prevalent in clinical settings and manifest as bone fenestration, dehiscence, or attachment loss, presenting a significant challenge to oral health. In regenerative medicine, harnessing developmental principles for tissue repair offers promising therapeutic potential. Of particular interest is the condensation of progenitor cells, an essential event in organogenesis that has inspired clinically effective cell aggregation approaches in dental regeneration. However, the precise cellular coordination mechanisms during condensation and regeneration remain elusive. Here, taking the tooth as a model organ, we employed single-cell RNA sequencing to dissect the cellular composition and heterogeneity of human dental follicle and dental papilla, revealing a distinct Platelet-derived growth factor receptor alpha (PDGFRA) mesenchymal stem/stromal cell (MSC) population with remarkable odontogenic potential. Interestingly, a reciprocal paracrine interaction between PDGFRA+ dental follicle stem cells (DFSCs) and CD31+ Endomucin+ endothelial cells (ECs) was mediated by Vascular endothelial growth factor A (VEGFA) and Platelet-derived growth factor subunit BB (PDGFBB). This crosstalk not only maintains the functionality of PDGFRA+ DFSCs but also drives specialized angiogenesis. In vivo periodontal bone regeneration experiments further reveal that communication between PDGFRA+ DFSC aggregates and recipient ECs is essential for effective angiogenic-osteogenic coupling and rapid tissue repair. Collectively, our results unravel the importance of MSC-EC crosstalk mediated by the VEGFA and PDGFBB-PDGFRA reciprocal signaling in orchestrating angiogenesis and osteogenesis. These findings not only establish a framework for deciphering and promoting periodontal bone regeneration in potential clinical applications but also offer insights for future therapeutic strategies in dental or broader regenerative medicine.
Receptor, Platelet-Derived Growth Factor alpha/metabolism*
;
Humans
;
Neovascularization, Physiologic/physiology*
;
Dental Sac/cytology*
;
Single-Cell Analysis
;
Transcriptome
;
Mesenchymal Stem Cells/metabolism*
;
Bone Regeneration
;
Animals
;
Dental Papilla/cytology*
;
Periodontium/physiology*
;
Stem Cells/metabolism*
;
Regeneration
;
Angiogenesis
5.Predictive Value of High-frequency Ultrasound for Cervical Lymph Node Metastasis in Papillary Thyroid Carcinoma
Sijie YUAN ; Yufang ZHAO ; Xiaohui YAN
Journal of Medical Research 2024;53(1):88-92
Objective To investigate the risk factors and independent risk factors associated with papillary thyroid carcinoma(PTC)cervical lymph node metastasis,and to assess the predictive efficacy of independent risk factors on metastatic lymph nodes.Methods Clinical and ultrasonographic data of 279 patients with PTC were collected,and the patients were divided into two groups according to the presence of cervical lymph node metastasis based on postoperative pathology,and the relevant characteristics of the two groups were ana-lyzed to explore the risk factors and independent risk factors associated with cervical lymph node metastasis in PTC,and the predictive ef-ficacy of independent risk factors on cervical metastatic lymph nodes was compared by receiver operating characteristic(ROC)curves.Results Age,gender and the maximum diameter,border,shape,microcalcifications,and distance from the thyroid capsule of cancer nodes were risk factors for cervical lymph node metastasis in both groups(P<0.05);male,maximum diameter of cancer nodes 10mm,microcalcifications,and distance from the thyroid capsule ≤2mm were independent risk factors for cervical lymph node metastasis in PTC(P<0.05);the AUC of the combination of four independent risk factors was 0.785(95%CI:0.721-0.849,P<0.05),with the highest efficacy in predicting cervical lymph node metastasis.Conclusion Male,maximum diameter of cancer nodes ≥10mm,mi-crocalcifications,and distance from the thyroid capsule ≤2mm were independent risk factors for cervical lymph node metastasis of PTC,and the combination of four independent risk factors could assist in assessing the risk of cervical lymph node metastasis of PTC and improve patient prognosis.
6.Association of dietary patterns with serum uric acid and hyperuricemia in Chinese adults
Mengru DONG ; Yifei OUYANG ; Yanli WEI ; Huijun WANG ; Aidong LIU ; Zhihong WANG ; Xiaorong YUAN ; Xiaohui DONG ; Jiguo ZHANG
Chinese Journal of Epidemiology 2024;45(10):1403-1409
Objective:To analyze the dietary patterns of Chinese adults and explore the relationship with serum uric acid (SUA) and hyperuricemia (HUA).Methods:A total of 9 358 adults were selected in the 2018 China Health and Nutrition Survey. Dietary intake data were collected by three consecutive 24-hour dietary recalls and weighing method. The social demographic information of the survey subjects was obtained through questionnaire surveys. The dietary patterns were extracted using factor analysis, and the relationship between dietary patterns and SUA was analyzed using multiple linear regression analysis. The correlation between HUA and dietary patterns was analyzed using logistic regression analysis models.Results:Four dietary patterns were identified: northern (high intakes of wheat, other cereals,and tubers); modern (high intakes of fruit, dairy, eggs, and nuts); southern (high intakes of rice and vegetables);animal food-wine (high intake of organ meats, seafood, and wine). The multiple linear regression analysis results showed that the northern pattern was negatively correlated with SUA ( β=-0.438, 95% CI: -0.500--0.376); the modern pattern was negatively correlated with SUA ( β=-0.134, 95% CI: -0.219--0.049); the southern model was significantly correlated with higher SUA ( β=0.146, 95% CI: 0.079-0.214); the animal food-wine pattern was positively correlated with SUA ( β=0.188, 95% CI: 0.123-0.252). Logistic regression analysis showed that compared with the northern model score Q1 group, the risk of developing HUA was reduced in Q3 and Q4 groups, with ORs values of 0.777 (95% CI: 0.650-0.929) and 0.509 (95% CI: 0.423-0.613), respectively; and compared with the modern model score Q1 group, the higher the scores in Q3 and Q4 groups, the HUA was lower, with ORs of 0.793 (95% CI: 0.660-0.953) and 0.768 (95% CI: 0.631-0.934), respectively. Compared with the animal food-wine pattern score Q1 group, the risk of developing HUA was increased in both Q3 and Q4 groups ( Q3 group: OR=1.224, 95% CI: 1.012-1.480; Q4 group: OR=1.312, 95% CI: 1.086-1.584). Conclusions:Dietary patterns are associated with HUA. The northern and modern patterns are related to lower SUA levels and reduced risk of HUA, while the animal food-wine pattern increases the risk of HUA.
7.The mediating role of family support between type D personality and intolerance of uncertainty in first-stroke patients
Xiaoping YANG ; Huijuan WANG ; Xiaohui LIU ; Haihua GAO ; Jialin YUAN ; Miaomiao CHEN ; Lijun WANG
Chinese Journal of Practical Nursing 2024;40(7):548-555
Objective:To analyze the status quo of type D personality, intolerance of uncertainty and family support in first-episode stroke patients, and to explore the mediating role of family support between type D personality and intolerance of uncertainty in first-episode stroke patients, in order to provide reference for formulating relevant clinical intervention measures to promote the physical and mental health of first-episode stroke patients.Methods:This study was a cross-sectional investigation. A total of 300 patients with acute first-episode stroke who met the inclusion and exclusion criteria in the Department of Neurology of the General Hospital of Ningxia Medical University and the First People′s Hospital of Yinchuan from May 2023 to September 2023 were selected as the study objects by convenience sampling method. The general data questionnaire, Type D personality Scale-14, Family Caring Index Scale and the Intolerance of Uncertainty Scale were used to investigate them. Pearson correlation analysis was used to test the correlation between variables, and SPSS plug-in PROCESS 3.5 was used to test the mediation effect.Results:Finally, 300 questionnaires were effectively collected, including 228 males and 72 females. Patients aged ≥ 60 years old were the majority, accounting for 49.3% (148/300). The detection rate of type D personality in the first stroke patients was 37.3% (112/300), and the total score of Type D personality inventory, family support and intolerance of uncertainty of type D personality in the first stroke patients were (22.16 ± 9.95), (6.40 ± 2.23), (27.82 ± 7.93) points. The correlation analysis results showed that the intolerance of uncertainty of type D personality in the first stroke patients was positively correlated with type D personality scores ( r=0.675, P<0.001). There was a negative correlation with family support score ( r=-0.644, P<0.001). The results of mediating effect analysis showed that family support played a partial mediating role in the relationship between type D personality and intolerability of uncertainty in first-stroke patients, and the mediating effect accounted for 34.94% of the total effect. Conclusions:The mediating role of family support between type D personality and intolerability of uncertainty in first-stroke patients is established. In the future, the level of family support of patients can be continuously improved to reduce their intolerability of uncertainty, so as to promote the physical and mental health of patients and improve their quality of life.
8.Practices on"Center-Multidisciplinary Team"Structure for Rare Disease Management in General Hospitals
Yu ZHUANG ; Yuan ZHONG ; Xinxia WU ; Xuedong XU ; Rong MU ; Xiaohui ZHU ; Rong LI ; Wei FU
Chinese Hospital Management 2024;44(9):37-40
Objectives It was a preliminary exploration on rare disease management of comprehensive hospital based on the"Center-Multidisciplinary Team"structure.Methods By applying interrupted time series analysis,the characteristics and related medical service of rare disease patients discharged from sample hospitals from 2021 to 2023 were measured.Results The results showed that the hospital served 19 825 rare disease patients from 2021 to 2023.The proportion of patients whose main diagnosis were rare diseases was approximately 27%.The interrupted time series technique indicated that the expected proportion of rare disease patients increased instantly by 1.89 thousand points.The capability to diagnose has significantly improved,with a growing trend on volume of bioinformatics analysis services for rare diseases patients.Conclusion The rare disease management based on the"Center-Team"structure can increase the attention of medical staff to rare diseases,strengthen the diagnostic capacity of rare diseases,and make patient management more continuous and disciplinary co-operation more efficient without significantly increasing the input burden on hospitals.
9.Practices on"Center-Multidisciplinary Team"Structure for Rare Disease Management in General Hospitals
Yu ZHUANG ; Yuan ZHONG ; Xinxia WU ; Xuedong XU ; Rong MU ; Xiaohui ZHU ; Rong LI ; Wei FU
Chinese Hospital Management 2024;44(9):37-40
Objectives It was a preliminary exploration on rare disease management of comprehensive hospital based on the"Center-Multidisciplinary Team"structure.Methods By applying interrupted time series analysis,the characteristics and related medical service of rare disease patients discharged from sample hospitals from 2021 to 2023 were measured.Results The results showed that the hospital served 19 825 rare disease patients from 2021 to 2023.The proportion of patients whose main diagnosis were rare diseases was approximately 27%.The interrupted time series technique indicated that the expected proportion of rare disease patients increased instantly by 1.89 thousand points.The capability to diagnose has significantly improved,with a growing trend on volume of bioinformatics analysis services for rare diseases patients.Conclusion The rare disease management based on the"Center-Team"structure can increase the attention of medical staff to rare diseases,strengthen the diagnostic capacity of rare diseases,and make patient management more continuous and disciplinary co-operation more efficient without significantly increasing the input burden on hospitals.
10.Practices on"Center-Multidisciplinary Team"Structure for Rare Disease Management in General Hospitals
Yu ZHUANG ; Yuan ZHONG ; Xinxia WU ; Xuedong XU ; Rong MU ; Xiaohui ZHU ; Rong LI ; Wei FU
Chinese Hospital Management 2024;44(9):37-40
Objectives It was a preliminary exploration on rare disease management of comprehensive hospital based on the"Center-Multidisciplinary Team"structure.Methods By applying interrupted time series analysis,the characteristics and related medical service of rare disease patients discharged from sample hospitals from 2021 to 2023 were measured.Results The results showed that the hospital served 19 825 rare disease patients from 2021 to 2023.The proportion of patients whose main diagnosis were rare diseases was approximately 27%.The interrupted time series technique indicated that the expected proportion of rare disease patients increased instantly by 1.89 thousand points.The capability to diagnose has significantly improved,with a growing trend on volume of bioinformatics analysis services for rare diseases patients.Conclusion The rare disease management based on the"Center-Team"structure can increase the attention of medical staff to rare diseases,strengthen the diagnostic capacity of rare diseases,and make patient management more continuous and disciplinary co-operation more efficient without significantly increasing the input burden on hospitals.

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