1.Percutaneous coronary intervention vs . medical therapy in patients on dialysis with coronary artery disease in China.
Enmin XIE ; Yaxin WU ; Zixiang YE ; Yong HE ; Hesong ZENG ; Jianfang LUO ; Mulei CHEN ; Wenyue PANG ; Yanmin XU ; Chuanyu GAO ; Xiaogang GUO ; Lin CAI ; Qingwei JI ; Yining YANG ; Di WU ; Yiqiang YUAN ; Jing WAN ; Yuliang MA ; Jun ZHANG ; Zhimin DU ; Qing YANG ; Jinsong CHENG ; Chunhua DING ; Xiang MA ; Chunlin YIN ; Zeyuan FAN ; Qiang TANG ; Yue LI ; Lihua SUN ; Chengzhi LU ; Jufang CHI ; Zhuhua YAO ; Yanxiang GAO ; Changan YU ; Jingyi REN ; Jingang ZHENG
Chinese Medical Journal 2025;138(3):301-310
BACKGROUND:
The available evidence regarding the benefits of percutaneous coronary intervention (PCI) on patients receiving dialysis with coronary artery disease (CAD) is limited and inconsistent. This study aimed to evaluate the association between PCI and clinical outcomes as compared with medical therapy alone in patients undergoing dialysis with CAD in China.
METHODS:
This multicenter, retrospective study was conducted in 30 tertiary medical centers across 12 provinces in China from January 2015 to June 2021 to include patients on dialysis with CAD. The primary outcome was major adverse cardiovascular events (MACE), defined as a composite of cardiovascular death, non-fatal myocardial infarction, and non-fatal stroke. Secondary outcomes included all-cause death, the individual components of MACE, and Bleeding Academic Research Consortium criteria types 2, 3, or 5 bleeding. Multivariable Cox proportional hazard models were used to assess the association between PCI and outcomes. Inverse probability of treatment weighting (IPTW) and propensity score matching (PSM) were performed to account for potential between-group differences.
RESULTS:
Of the 1146 patients on dialysis with significant CAD, 821 (71.6%) underwent PCI. After a median follow-up of 23.0 months, PCI was associated with a 43.0% significantly lower risk for MACE (33.9% [ n = 278] vs . 43.7% [ n = 142]; adjusted hazards ratio 0.57, 95% confidence interval 0.45-0.71), along with a slightly increased risk for bleeding outcomes that did not reach statistical significance (11.1% vs . 8.3%; adjusted hazards ratio 1.31, 95% confidence interval, 0.82-2.11). Furthermore, PCI was associated with a significant reduction in all-cause and cardiovascular mortalities. Subgroup analysis did not modify the association of PCI with patient outcomes. These primary findings were consistent across IPTW, PSM, and competing risk analyses.
CONCLUSION
This study indicated that PCI in patients on dialysis with CAD was significantly associated with lower MACE and mortality when comparing with those with medical therapy alone, albeit with a slightly increased risk for bleeding events that did not reach statistical significance.
Humans
;
Percutaneous Coronary Intervention/methods*
;
Male
;
Female
;
Coronary Artery Disease/drug therapy*
;
Retrospective Studies
;
Renal Dialysis/methods*
;
Middle Aged
;
Aged
;
China
;
Proportional Hazards Models
;
Treatment Outcome
2.Regulatory roles of JAZ in the growth and development of horticultural plants.
Xinxin ZHANG ; Tao TAO ; Hangchun LI ; Zhi QIAO ; Qinglin TANG ; Dayong WEI ; Yang YANG ; Zhimin WANG
Chinese Journal of Biotechnology 2025;41(2):530-545
Jasmonic acid (JA) is a common plant hormone with regulatory effects on plant growth and development. The jasmonate ZIM-domain (JAZ) proteins (JAZs), as key regulators in the JA signaling pathway, are involved in multiple biological processes such as anthocyanin accumulation, flowering time modulation, and secondary metabolite synthesis in plants. JAZs are essential components of many regulatory signaling networks. The JAZ genes, members of the plant-specific TIFY family, have been identified in the genomes of a variety of horticultural plants. Here, we summarized the research progress in the roles of JAZs in horticultural plants, aiming to give insights into the further study of the biological functions and regulatory networks of JAZ genes in plants.
Horticulture
;
Repressor Proteins/metabolism*
;
Plant Proteins/metabolism*
;
Cyclopentanes/metabolism*
;
Oxylipins/metabolism*
;
Plants/metabolism*
;
Plant Development
3.Brassica juncea WRKY12 mediates bolting and flowering by interacting with the SOC1 and FUL promoters.
Yifang HUANG ; Yue DONG ; Yue YU ; Dakun LIU ; Qinlin DENG ; Yuanda WANG ; Dayong WEI ; Zhimin WANG ; Qinglin TANG
Chinese Journal of Biotechnology 2025;41(7):2818-2828
Flowering and bolting are important agronomic traits in cruciferous crops such as Brassica juncea. Timely flowering can ensure the crop organ yield and quality, as well as seed propagation. The WRKY family plays an important role in regulating plant bolting and flowering, while the function and mechanism of WRKY12 in B. juncea remain unknown. To explore its function and mechanism in bolting and flowering of B. juncea, we cloned and characterized the BjuWRKY12 gene in B. juncea and found that its expression levels were significantly higher in flowers and inflorescences than in leaves. BjuWRKY12 belonged to the Ⅱc subfamily of the WRKY family, and subcellular localization indicated that the protein was located in the nucleus. Ectopic overexpression of BjuWRKY12 in transgenic lines promoted bolting and flowering, leading to significant increases in the expression levels of flowering integrators SOC1 and FUL. Furthermore, yeast one-hybrid and dual luciferase reporter system assays confirmed that BjuWRKY12 directly bound to the promoters of BjuSOC1 and BjuFUL, undergoing protein-DNA interactions. This discovery gives new insights into the regulation network and molecular mechanisms of BjuWRKY12, laying a theoretical foundation for the breeding of high-yield and high-quality varieties of B. juncea.
Mustard Plant/metabolism*
;
Flowers/growth & development*
;
Plant Proteins/physiology*
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Promoter Regions, Genetic/genetics*
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Gene Expression Regulation, Plant
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Plants, Genetically Modified/genetics*
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Transcription Factors/metabolism*
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MADS Domain Proteins/metabolism*
4.Predict the effect of the number of positive preoperative serum tumor markers on the surgical method and prognosis of intrahepatic cholangiocarcinoma patients based on mediation analysis
Zonglong LI ; Jialu CHEN ; Yue TANG ; Delong QIN ; Chen CHEN ; Yinghe QIU ; Hong WU ; Yu HE ; Xianhai MAO ; Wenlong ZHAI ; Jingdong LI ; Xiao LIANG ; Chuandong SUN ; Kai MA ; Zhimin GENG ; Zhaohui TANG ; Zhiwei QUAN
Chinese Journal of Surgery 2024;62(7):685-696
Objective:To investigate the effect of the number of positive preoperative serological tumor markers on the surgical approach and prognosis of patients with intrahepatic cholangiocarcinoma.Methods:This is a retrospective case-series study. Data from 548 patients with intrahepatic cholangiocarcinoma after radical resection from October 2010 to April 2019 were retrospectively collected in 10 hospitals of China. There were 277 males and 271 females with an age of (57.8±10.2)years(range:23 to 84 years). Four hundred and twenty-six patients(77.7%) had at least one positive preoperative serum tumor marker. The data collection included the results of 4 preoperative serological tumor markers,other preoperative indicators(5 prodromal symptoms, 6 medical history,8 preoperative serological indicators,5 preoperative imaging indicators,and 14 preoperative pathological examination indicators),baseline data (gender and age),surgical methods,and prognostic follow-up data. Four preoperative results of serologic tumor marker and surgical procedure were converted into categorical variables. The number of positive preoperative serum tumor markers was used as the treatment variable,the surgical method was used as the mediating variable,and the survival time was used as the outcome variable. Univariate and multivariate analysis were used to screen for other preoperative indicators which were independent factors that influenced the surgical procedure and the prognosis of patients as covariates to analyze the mediating effect.Results:Of the 548 patients included in the study, 176 patients (32.1%) underwent partial hepatectomy,151 patients(27.5%) underwent hemihepatectomy, and 221 patients(40.3%) underwent partial hepatectomy or hemihepatectomy combined with other treatments. The results of the univariate and multivariate analysis showed that the number of positive serum tumor markers,intrahepatic bile duct dilatation,portal vein invasion,pathological differentiation,pathological type,vascular invasion,T stage,N stage and maximum tumor diameter were independent factors influencing the surgical procedure(all P<0.05). Intrahepatic bile duct dilatation,pathological differentiation and T stage were independent prognostic factors for patients with intrahepatic cholangiocarcinoma(all P<0.05). Intrahepatic bile duct dilatation,differentiation and T stage were included as covariates in the mediation effect model. The results showed that the number of positive serum tumor markers before surgery had a negative predictive effect on the survival time of patients with intrahepatic cholangiocarcinoma ( β=-0.092, P=0.039),and had a positive predictive effect on the surgical method ( β=0.244, P<0.01). The number of positive serum tumor markers had a negative predictive effect on the survival time of patients with intrahepatic cholangiocarcinoma ( β=-0.151, P=0.002). Direct and indirect effects accounted for 71.3% and 28.7% of total effects,respectively. Conclusions:The higher the positive number of preoperative tumor markers,the worse the prognosis of patients with intrahepatic cholangiocarcinoma. The number of positive cells not only directly affects the prognosis of patients,but also indirectly affects the prognosis of patients by affecting the surgical method.
5.Efficacy analysis of liver wedge resection and liver Ⅳb and Ⅴ segmentectomy for T2 gallblad-der carcinoma
Qi LI ; Zhenqi TANG ; Hengchao LIU ; Yubo MA ; Chen CHEN ; Dong ZHANG ; Zhimin GENG
Chinese Journal of Digestive Surgery 2024;23(7):934-943
Objective:To investigate the efficacy of liver wedge resection and liver Ⅳb and Ⅴ segmentectomy for T2 gallbladder carcinoma (GBC).Methods:The retrospective cohort study was conducted. The clinicopathological data of 168 patients who underwent radical resection of T2 GBC in The First Affiliated Hospital of Xi′an Jiaotong University from January 2011 to December 2021 were collected. There were 59 males and 109 females, aged (65±10)years. Of 168 patients, there were 112 cases in T2a stage and 56 cases in T2b stage. Of 112 patients in T2a stage, 73 cases underwent liver wedge resection and 39 cases underwent liver Ⅳb and Ⅴ segmentectomy. Of 56 patients in T2b stage, 27 cases underwent liver wedge resection and 29 cases underwent liver Ⅳb and Ⅴ segmen-tectomy. Measurement data with normal distribution were represented as Mean± SD, and measure-ment data with skewed distribution were represented as M(range). Count data were described as absolute numbers, and comparison between groups was conducted using the chi-square test or Fisher exact probability. Comparison of ordinal data was conducted using the Mann-Whitney U test. The Kaplan-Meier method was used to calculate survival rate and draw survival curve, and the Log-rank test was used for survival analysis. The COX proportional risk model was used for univariate and multivariate analyses. Results:(1) Clinical data analysis of patients undergoing different extent of hepatic resection for T2 GBC. There was no significant difference in gender, age, cholecystoli-thiasis, preoperative total bilirubin, carcinoembryonic antigen, CA19-9, CA125, incidental GBC, perineural invasion, microvascular invasion, pathological differentiation, histopathological subtypes, N staging, TNM staging between patients with T2a and T2b GBC who underwent different extent of hepatic resection ( P>0.05). (2) Prognostic analysis of T2 GBC patients undergoing different extent of hepatic resection. The 1-, 3- and 5-year cumulative disease-free survival rates of T2 GBC patients undergoing liver wedge resection were 78.0%, 60.1% and 51.4%, respectively, versus 86.8%, 80.0% and 68.0% of T2 GBC patients undergoing liver Ⅳb and Ⅴ segmentectomy, showing a significant difference between them ( χ2 =5.205, P<0.05). The 1-, 3-, and 5-year cumulative overall survival rates of T2 GBC patients undergoing liver wedge resection were 85.0%, 62.5%, and 55.1%, respectively, versus 92.6%, 81.6%, and 68.8% for T2 GBC patients undergoing liver Ⅳb and Ⅴ segmentectomy, showing a significant difference in cumulative overall survival rate between them ( χ2=4.351, P<0.05). The 1-, 3-, and 5-year cumulative disease-free survival rates of T2b GBC patients undergoing liver wedge resection were 70.4%, 45.9% and 39.2%, respectively, versus 89.7%, 71.3% and 54.0% of T2b GBC patients undergoing liver Ⅳb and Ⅴ segmentectomy, showing a significant difference between them ( χ2=5.047, P<0.05). The 1-, 3-, and 5-year cumulative overall survival rates of T2b GBC patients undergoing liver wedge resection were 81.5%, 53.2%, and 41.0%, respectively, versus 89.7%, 77.0%, and 60.7% of T2b GBC patients undergoing liver Ⅳb and Ⅴ segmentectomy, showing no significant difference in cumulative overall survival rate between them ( χ2=4.014, P<0.05). (3) Analysis of factors influencing prognosis of patients undergoing radical resection for T2 GBC. Results of multivariate analysis showed that CA19-9>39.0 U/mL, perineural invasion, N1 and N2 stage were independent risk factors influencing disease-free survival time of patients undergoing radical resection for T2 GBC ( hazard ratio=2.736, 3.496, 2.638, 17.440, 95% confidence interval as 1.195-6.266, 1.213-10.073, 1.429-4.869, 8.362-36.374, P<0.05). Liver Ⅳb and Ⅴ segmentectomy was an independent protective factor influencing disease-free survival time of patients undergoing radical resection for T2 GBC ( hazard ratio=0.418, 95% confidence interval as 0.230-0.759, P<0.05). CA19-9 >39.0 U/mL, perineural invasion, ⅡB stage, ⅢB stage and ⅣB stage of TNM staging were independent risk factors influencing overall survival time of patients undergoing radical resection for T2 GBC ( hazard ratio=2.740, 3.210, 2.037, 3.439, 24.466, 95% confidence interval as 1.127-6.664, 1.049-9.819, 1.004-4.125, 1.730-6.846, 10.733-55.842, P<0.05). Liver Ⅳb and Ⅴ segmentectomy was an independent protective factor influencing overall survival time of patients undergoing radical resec-tion for T2 GBC ( hazard ratio=0.476, 95% confidence interval as 0.261-0.867, P<0.05). (4) Analysis of postoperative complications in patients undergoing different extent of hepatic resection for T2 GBC. There was no significant difference in postoperative complications of patients with T2a and T2b GBC undergoing liver wedge resection or liver Ⅳb and Ⅴ segmentectomy ( P>0.05). Conclusions:Compared to liver wedge resection, liver Ⅳb and Ⅴ segmentectomy can effectively prolong the disease-free survival overall survival time of T2b GBC patients. There is no significant difference in the major complications. Liver Ⅳb and Ⅴ segmentectomy is an independent protective factor for prognosis of patients undergoing radical resection for T2 GBC.
6.Predict the effect of the number of positive preoperative serum tumor markers on the surgical method and prognosis of intrahepatic cholangiocarcinoma patients based on mediation analysis
Zonglong LI ; Jialu CHEN ; Yue TANG ; Delong QIN ; Chen CHEN ; Yinghe QIU ; Hong WU ; Yu HE ; Xianhai MAO ; Wenlong ZHAI ; Jingdong LI ; Xiao LIANG ; Chuandong SUN ; Kai MA ; Zhimin GENG ; Zhaohui TANG ; Zhiwei QUAN
Chinese Journal of Surgery 2024;62(7):685-696
Objective:To investigate the effect of the number of positive preoperative serological tumor markers on the surgical approach and prognosis of patients with intrahepatic cholangiocarcinoma.Methods:This is a retrospective case-series study. Data from 548 patients with intrahepatic cholangiocarcinoma after radical resection from October 2010 to April 2019 were retrospectively collected in 10 hospitals of China. There were 277 males and 271 females with an age of (57.8±10.2)years(range:23 to 84 years). Four hundred and twenty-six patients(77.7%) had at least one positive preoperative serum tumor marker. The data collection included the results of 4 preoperative serological tumor markers,other preoperative indicators(5 prodromal symptoms, 6 medical history,8 preoperative serological indicators,5 preoperative imaging indicators,and 14 preoperative pathological examination indicators),baseline data (gender and age),surgical methods,and prognostic follow-up data. Four preoperative results of serologic tumor marker and surgical procedure were converted into categorical variables. The number of positive preoperative serum tumor markers was used as the treatment variable,the surgical method was used as the mediating variable,and the survival time was used as the outcome variable. Univariate and multivariate analysis were used to screen for other preoperative indicators which were independent factors that influenced the surgical procedure and the prognosis of patients as covariates to analyze the mediating effect.Results:Of the 548 patients included in the study, 176 patients (32.1%) underwent partial hepatectomy,151 patients(27.5%) underwent hemihepatectomy, and 221 patients(40.3%) underwent partial hepatectomy or hemihepatectomy combined with other treatments. The results of the univariate and multivariate analysis showed that the number of positive serum tumor markers,intrahepatic bile duct dilatation,portal vein invasion,pathological differentiation,pathological type,vascular invasion,T stage,N stage and maximum tumor diameter were independent factors influencing the surgical procedure(all P<0.05). Intrahepatic bile duct dilatation,pathological differentiation and T stage were independent prognostic factors for patients with intrahepatic cholangiocarcinoma(all P<0.05). Intrahepatic bile duct dilatation,differentiation and T stage were included as covariates in the mediation effect model. The results showed that the number of positive serum tumor markers before surgery had a negative predictive effect on the survival time of patients with intrahepatic cholangiocarcinoma ( β=-0.092, P=0.039),and had a positive predictive effect on the surgical method ( β=0.244, P<0.01). The number of positive serum tumor markers had a negative predictive effect on the survival time of patients with intrahepatic cholangiocarcinoma ( β=-0.151, P=0.002). Direct and indirect effects accounted for 71.3% and 28.7% of total effects,respectively. Conclusions:The higher the positive number of preoperative tumor markers,the worse the prognosis of patients with intrahepatic cholangiocarcinoma. The number of positive cells not only directly affects the prognosis of patients,but also indirectly affects the prognosis of patients by affecting the surgical method.
7.The regulatory role of the RUS family in plant growth and development.
Yao HU ; Sirui LI ; Xinxin ZHANG ; Qinglin TANG ; Dayong WEI ; Shibing TIAN ; Yang YANG ; Zhimin WANG
Chinese Journal of Biotechnology 2024;40(1):81-93
The chloroplast genome encodes many key proteins involved in photosynthesis and other metabolic processes, and metabolites synthesized in chloroplasts are essential for normal plant growth and development. Root-UVB (ultraviolet radiation B)-sensitive (RUS) family proteins composed of highly conserved DUF647 domain belong to chloroplast proteins. They play an important role in the regulation of various life activities such as plant morphogenesis, material transport and energy metabolism. This article summarizes the recent advances of the RUS family proteins in the growth and development of plants such as embryonic development, photomorphological construction, VB6 homeostasis, auxin transport and anther development, with the aim to facilitate further study of its molecular regulation mechanism in plant growth and development.
Female
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Pregnancy
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Humans
;
Ultraviolet Rays
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Biological Transport
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Chloroplasts/genetics*
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Embryonic Development
;
Plant Development/genetics*
8.Association between body composition and coronary artery calcification in patients with chronic kidney disease
Jiajin HAN ; Jingwei GAO ; Zhenjian XU ; Zhimin YUAN ; Ying TANG ; Haifeng ZHANG ; Yangxin CHEN ; Jingfeng WANG ; Pinming LIU
Chinese Journal of Cardiology 2024;52(6):676-683
Objective:To investigate the association between body composition and coronary artery calcification in patients with chronic kidney disease (CKD).Methods:This cross-sectional study enrolled patients with CKD hospitalized from May 2019 to April 2022 at Sun Yat-sen Memorial Hospital, Guangzhou, China. Skeletal muscle mass index and visceral fat area were measured by bioelectrical impedance analysis. Coronary artery calcification was assessed by computed tomography. Patients were divided into coronary artery calcification group and non-coronary artery calcification group according to the incidence of coronary artery calcification. Patients were categorized into tertile groups according to their skeletal muscle mass index and visceral fat area levels ranging from the lowest to the highest levels (T1 to T3). We defined skeletal muscle mass index≤30.4% as low muscle mass and visceral fat area≥80.6 cm 2 as high visceral fat based on the results of the restricted cubic spline graph. All individuals were divided into 4 phenotypes: normal body composition, low muscle mass, high visceral fat, and low muscle mass with high visceral fat. Spearman correlation analysis and logistic regression analysis were used to assess the association between skeletal muscle mass index, visceral fat area and coronary artery calcification. Results:A total of 107 patients with CKD were enrolled, with an age of (60.0±14.1) years, including 41 female patients (38.3%). Patients of coronary artery calcification group had lower skeletal muscle mass index ((32.0±4.8) vs. (34.3±4.8), P=0.016) and higher visceral fat area ((70.8±32.6) cm 2 vs. (47.9±23.8) cm 2, P<0.001) than those of non-coronary artery calcification group. Patients in the T3 group of skeletal muscle mass index had a lower prevalence of coronary artery calcification (17 (48.6%) vs. 28 (77.8%)) and a lower coronary artery calcification score (0.5 (0, 124.0) vs. 12.0 (0.3, 131.0)) than those in the T1 group ( P<0.05). Similarly, patients in the T1 group of visceral fat area had a lower prevalence of coronary artery calcification (14 (40.0%) vs. 29 (80.6%)) and a lower coronary artery calcification score (0 (0, 3.0) vs. 37.0 (2.0, 131.0)) than those in the T3 group ( P<0.05). Likewise, patients with both low muscle mass and low muscle mass with high visceral fat had a higher prevalence of coronary artery calcification (11(78.6%) vs. 33 (47.8%); 15 (83.3%) vs. 33 (47.8%)) and a higher coronary artery calcification score (31.1 (0.8, 175.8) vs. 0 (0, 16.4); 27.6 (6.4, 211.4) vs. 0 (0, 16.4)) than those with normal body composition ( P<0.05). Spearman correlation analysis showed that skeletal muscle mass index was inversely correlated with coronary artery calcification score ( r=-0.212, P=0.028), and visceral fat area was positively correlated with coronary artery calcification score ( r=0.408, P<0.001). Multivariate logistic regression analysis showed that increased skeletal muscle mass index was inversely associated with coronary artery calcification prevalence (T2: OR=0.208, 95% CI: 0.056-0.770, P=0.019; T3: OR=0.195, 95% CI: 0.043-0.887, P=0.034), and reduced visceral fat area was inversely associated with coronary artery calcification prevalence (T1: OR=0.256, 95% CI: 0.071-0.923, P=0.037; T2: OR=0.263, 95% CI: 0.078-0.888, P=0.031). Consistently, both low muscle mass and low muscle mass with high visceral fat were associated with coronary artery calcification prevalence ( OR=6.616, 95% CI: 1.383-31.656, P=0.018; OR=5.548, 95% CI: 1.062-28.973, P=0.042). Conclusion:Reduced skeletal muscle mass index and increased visceral fat area are significantly associated with both the prevalence and severity of coronary artery calcification in patients with CKD.
9.Consideration on"Patient-Centered"Drug Development for Rare Diseases
Ling TANG ; Xing AI ; Tian ZHAO ; Zhimin YANG
JOURNAL OF RARE DISEASES 2024;3(2):168-174
The unmet clinical needs of patients with rare diseases persist.Many rare diseases lack effec-tive treatments,and drug development for rare diseases faces greater challenges than that for common multiple diseases.In recent years,the concept of"patient-centered"drug development has been widely adopted.The Center for Drug Evaluation(CDE)of the National Medical Products Administration has successively issued a series of relevant guiding principles,such as the Technical Guidelines for the Implementation of Patient-Centered Clinical Trials,to promote a"patient-centered"drug development model.The implementation of the"patient-centered"approach in rare disease drug research and development,with a focus on patient perspectives and ac-tive engagement,can effectively facilitate a comprehensive understanding of rare diseases and patient needs among drug research and development enterprises,researchers,and regulatory agencies.This approach also en-hances the accuracy and efficiency of rare disease drug research and development.The CDE will continue to pri-oritize the integration of the"patient-centered"concept into rare disease drug research and development,effec-tively enhance the involvement of rare patients in the drug research and development process,and leverage the guiding role of patients'perspectives on drug research and development.
10.Application of Decentralized Clinical Trials in the Research and Development of Drugs for Rare Diseases
Huanhuan CUI ; Ling TANG ; Can CUI ; Zhuxing YAO ; Zhimin YANG ; Haixue WANG
JOURNAL OF RARE DISEASES 2024;3(2):175-180
Clinical trials of drugs for rare diseases face special challenges such as a limited number of patients,difficult recruitment,long trial period,and frequent video interviews during the trial.Therefore,in the clinical operation of rare diseases,a decentralized clinical trials(DCT)model based on the"patient-cen-tred"research and development concept is implemented.With the help of decentralized elements and digital health technology,the barriers of geographical restrictions can be overcome and subjects do not have to be limit-ed to traditional clinical trial sites(hospitals/research centers),which can significantly reduce the burden on subjects,increase their representation,and obtain a wider range of scientific research data.To guide the indus-try's scientific and standardized application of DCT in the research and development of drugs for rare diseases,the Center for Drug Evaluation of the National Medical Products Administration(NMPA)organized the stake holders to draft the Technical Guideline for the Application of Decentralized Clinical Trials in the Research and Development of Drugs for Rare Diseases.This guideline provides scientific recommendations for the development and implementation of DCT for rare disease drugs.It aims to solve the difficult and key problems during rare disease drug research and development,improve the efficiency and optimize patient experience.This article,combining the research and development concepts in the guideline,explains the current research and develop-ment thinking on the application of DCT in the research and development of rare disease drugs,with a view of providing reference for the industry.

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