1.Research Progress on the Efficacy and Safety of Deflazacort in the Treatment of Duchenne Muscular Dystrophy
Tingting XU ; Wei ZUO ; Xin LIU ; Shaohong WANG ; Zhuo SUN ; Junmei SHANG ; Luyao QIAO ; Bo ZHANG
JOURNAL OF RARE DISEASES 2025;4(2):248-257
Deflazacort,as a glucocorticoid medication,is conductive to improving motor function and muscle strength,delaying the loss of ambulation,enhancing pulmonary function,reducing the risk of scoliosis,slowing the progression of cardiomyopathy,and increasing survival rates in patients with Duchenne muscular dystrophy(DMD).In February 2017,the U.S.Food and Drug Administration(FDA)approved deflazacort for the treatment of DMD.In May 2024,deflazacort entered Peking Union Medical College Hospital for desig-nated use through the " temporary import" pathway.This article provides an overview of deflazacort from the perspectives of its mechanism of action,pharmacokinetics,clinical efficacy,and adverse effects,aiming to offer a reference for its rational and safe application in clinical practice.
2.Research Progress on the Efficacy and Safety of Deflazacort in the Treatment of Duchenne Muscular Dystrophy
Tingting XU ; Wei ZUO ; Xin LIU ; Shaohong WANG ; Zhuo SUN ; Junmei SHANG ; Luyao QIAO ; Bo ZHANG
JOURNAL OF RARE DISEASES 2025;4(2):248-257
Deflazacort,as a glucocorticoid medication,is conductive to improving motor function and muscle strength,delaying the loss of ambulation,enhancing pulmonary function,reducing the risk of scoliosis,slowing the progression of cardiomyopathy,and increasing survival rates in patients with Duchenne muscular dystrophy(DMD).In February 2017,the U.S.Food and Drug Administration(FDA)approved deflazacort for the treatment of DMD.In May 2024,deflazacort entered Peking Union Medical College Hospital for desig-nated use through the " temporary import" pathway.This article provides an overview of deflazacort from the perspectives of its mechanism of action,pharmacokinetics,clinical efficacy,and adverse effects,aiming to offer a reference for its rational and safe application in clinical practice.
3.Literature Based Analysis on Adverse Reactions in Simultaneously Clinical Use of Banxia (Rhizoma Pinelliae)- Wutou (Aconitum)
Can CAO ; Wenyong LIAO ; Jiwen ZHANG ; Yinghao WU ; Xiangnan XU ; Meijing WU ; Xiaoqing LIU ; Shaohong CHEN ; Haiyan LIU ; Linlin XIU ; Xiangqing CUI ; Gaoyang LI ; Ying ZHANG ; Gansheng ZHONG
Journal of Traditional Chinese Medicine 2025;66(9):955-962
ObjectiveTo analyze the adverse reactions associated with the clinical use of Banxia (Rhizoma Pinelliae)- Wutou (Aconitum) in the same formula, with the aim of providing a reference for the safety of their clinical application. MethodsLiterature on the clinical application of antagonistic herbs "Banxia-Wutou" used in the same formula, published from January 1st, 2014, to June 30th, 2023, was retrieved from databases including CNKI, VIP, Wanfang, SinoMed, PubMed, Cochrane Library, and Embase. A database was established, and information related to adverse reactions was extracted, including descriptions, classifications, specific manifestations, management and outcomes, patients' primary diseases (western medicine diseases and traditional Chinese medicine diagnoses and syndromes), and medication information (dosage, ratio, administration routes, and dosage forms). ResultsA total of 79 researches simultaneously used antagonistic herbs Banxia-Wutou in the same formula and reported associated advers reactions. Gastrointestinal adverse reactions were the most common, with 8 studies reporting management of adverse reactions and 3 studies reporting improvement with no intervention. Among the 11 researches, the adverse reaction relieved to extant, while other 69 researches didn't report the managment of adverse reaction and its prognosis. For the primary disease in western medicine system, chronic bronchitis and chronic obstructive pulmonary disease (COPD) were most common, while gastric pain was the most common symptom in traditional Chinese medicine with spleen and kidney deficiency and spleen stomach cold deficiency being the most frequent syndromes. The most common Banxia dosage was 10 g, while for the Wutou, Fuzi (Radix Aconiti Lateralis Praeparata) was predominant with the highest dose at 15 g. The most frequent herbal combination was Banxia-fuzi, with a 1∶1 ratio. The main administration route was oral, and the primary dosage form was decoction. ConclusionGastrointestinal adverse reactions are the most common in the clinical use of Banxia-Wutou antagonistic herb combinations. Research on the safety of "Banxia-Wutou" combinations should focus on respiratory system diseases and spleen-stomach related conditions.
4.Analysis of the curative efficacy and safety of ultrasound-guided MWA combined with PEI in treating thyroid cystic solid nodule
Fan ZHOU ; Shaohong ZHANG ; Changsong XU ; Zhengqing MU ; Jun LU
China Medical Equipment 2025;22(4):84-89
Objective:To explore the curative efficacy and safety of microwave ablation(MWA)combined with percutaneous ethanol injection(PEI)in treating thyroid cystic solid nodule.Methods:A total of 62 patients with thyroid cystic solid nodule,who admitted to The Affiliated Huaian No.1 People's Hospital of Nanjing Medical University from January 2021 to December 2022,were selected,and they were divided into microwave group(n=28,only single MWA)and combination group(n=34,MWA combined with PEI)according to different treatment plans.The reduction rate of the volume of thyroid nodules,indicators of thyroid function,clinical symptom and the score of physical signs,as well as the incidence of perioperative complications,were compared between two groups after treatment.Results:After treatment,the reduction rate of the volume of thyroid nodule was(88.97±6.36)%in the combination group,which was higher than that(88.15±5.69)%in the microwave group,and the differences in those indicators between the two groups were statistically significant(t=2.465,P<0.05).The differences of the indicators of thyroid function before and after treatment between two groups were not significant(P>0.05).The clinical symptoms and the scores of physical signs of both groups after treatment were lower than that before treatment(P>0.05).The incidence of perioperative complications in the combination group was 17.65%,which was lower than 42.68%in the microwave group(P<0.05),and the difference of that between the two groups was significant(x2=4.736,P<0.05).Conclusion:The curative efficacy of the combination of MWA and PEI is better than MWA alone,which has better safety.
5.Observation on the efficacy of 4R technology combined with prone position five-direction cervical muscle strength training for cervical spondylosis of cervical type
Haoyue DENG ; Xiaofeng XIA ; Jie LIU ; Qin XU ; Zeling LIAO ; Shaohong GUI
Chongqing Medicine 2025;54(11):2492-2496,2502
Objective To explore the efficacy of 4R technology combined with prone five-direction cer-vical muscle strength training for neck type cervical spondylosis.Methods A total of 112 patients with neck and shoulder pain who visited the Affiliated Shapingba Hospital of Chongqing University and the Shapingba District Traditional Chinese Medicine Hospital in Chongqing from January to November 2024 were selected as research subjects.They were randomly assigned using a random number table method into an observation group and a control group,with 56 patients in each group.The observation group received 4R technology com-bined with prone five-direction cervical muscle strength training,while the control group received conventional rehabilitation treatment.Both interventions lasted for 4 weeks.Differences in Visual Analogue Scale(VAS)scores,Neck Disability Index(NDI)scores,and cervical range of motion in flexion,extension,lateral bending,and rota-tion were assessed before treatment and at 1 and 6 months after treatment for both groups.Results At 1 and 6 months of treatment,VAS scores in both groups decreased compared to before treatment,with the observation group lower than the control group,and the difference was statistically significant(P<0.05).At 1 and 6 months of treatment,NDI scores in both groups decreased compared to before treatment,with the observation group lower than the control group,and the difference was statistically significant(P<0.05).At 1 and 6 months of treatment,cervical flexion,cervical extension,and cervical lateral flexion in both groups increased compared to before treatment,and cervical rotation in the observation group increased compared to before treatment.Cervical extension and cervical rotation in the observation group were greater than those in the con-trol group,and only at 6 months of treatment was cervical lateral flexion in the observation group greater than in the control group,with all differences statistically significant(P<0.05).Conclusion The 4R technology combined with prone five-direction cervical muscle strength training can effectively improve cervical-type cer-vical spondylosis.
6.Analysis of the curative efficacy and safety of ultrasound-guided MWA combined with PEI in treating thyroid cystic solid nodule
Fan ZHOU ; Shaohong ZHANG ; Changsong XU ; Zhengqing MU ; Jun LU
China Medical Equipment 2025;22(4):84-89
Objective:To explore the curative efficacy and safety of microwave ablation(MWA)combined with percutaneous ethanol injection(PEI)in treating thyroid cystic solid nodule.Methods:A total of 62 patients with thyroid cystic solid nodule,who admitted to The Affiliated Huaian No.1 People's Hospital of Nanjing Medical University from January 2021 to December 2022,were selected,and they were divided into microwave group(n=28,only single MWA)and combination group(n=34,MWA combined with PEI)according to different treatment plans.The reduction rate of the volume of thyroid nodules,indicators of thyroid function,clinical symptom and the score of physical signs,as well as the incidence of perioperative complications,were compared between two groups after treatment.Results:After treatment,the reduction rate of the volume of thyroid nodule was(88.97±6.36)%in the combination group,which was higher than that(88.15±5.69)%in the microwave group,and the differences in those indicators between the two groups were statistically significant(t=2.465,P<0.05).The differences of the indicators of thyroid function before and after treatment between two groups were not significant(P>0.05).The clinical symptoms and the scores of physical signs of both groups after treatment were lower than that before treatment(P>0.05).The incidence of perioperative complications in the combination group was 17.65%,which was lower than 42.68%in the microwave group(P<0.05),and the difference of that between the two groups was significant(x2=4.736,P<0.05).Conclusion:The curative efficacy of the combination of MWA and PEI is better than MWA alone,which has better safety.
7.Effect of Sargassum and Glycyrrhizae Radix et Rhizoma Incompatible Pair with Haizao Yuhutang on Oxidative Stress in Liver of Goiter Rats
Xiao DONG ; Yi TIAN ; Xiaoqing LIU ; Can CAO ; Wenyong LIAO ; Xiangnan XU ; Meijing WU ; Haiyan LIU ; Shaohong CHEN ; Xue YU ; Angran FAN ; Linlin XIU ; Gansheng ZHONG
Chinese Journal of Experimental Traditional Medical Formulae 2024;30(14):37-45
ObjectiveTo observe the effects of Sargassum and Glycyrrhizae Radix et Rhizoma incompatible pair with the Haizao Yuhutang (HYT) on oxidative stress in the liver of goiter rats under the condition of 2 times the dose limit of the Pharmacopoeia of the People's Republic of China 2020. MethodA total of 128 male Wistar rats were randomly divided into a blank group, a model group, a euthyrox group (20 μg·kg-1), a HYT group (12.06 g·kg-1), a HYT without Sargassum (HYT-H) group (9.90 g·kg-1), a HYT without Glycyrrhizae Radix et Rhizoma (HYT-G) group (10.26 g·kg-1), a HYT without Sargassum and Glycyrrhizae Radix et Rhizoma (HYT-HG) group (8.10 g·kg-1), and a Sargassum and Glycyrrhizae Radix et Rhizoma (HG) group (3.96 g·kg-1). The blank group was given deionized water by gavage, and the others were given propylthiouracil (PTU) to replicate the goiter pathological model. Euthyrox was taken as a positive control drug, and the rest of the Chinese medicine groups were given the corresponding decoction by gavage, the material was collected 12 hours after the last dose. The serum levels of aspartate aminotransferase (AST), alanine aminotransferase (ALT), alkaline phosphatase (ALP), and the contents of superoxide dismutase (SOD), glutathione peroxidase (GSH-Px), malondialdehyde (MDA), reactive oxygen species (ROS) in liver tissue were detected in each group. The pathological changes in the liver were observed via hematoxylin-eosin (HE) staining. Real-time fluorescence quantitative polymerase chain reaction (Real-time PCR) was utilized to detect the mRNA expressions of Kelch-like Ech-associated protein 1 (Keap1), nuclear factor E2-related factor 2 (Nrf2), heme oxygenase-1 (HO-1), p53 and Caspase-3 in liver tissues. Western blot was adopted to detect the protein expressions of Nrf2 and HO-1 in liver tissues in oxidative stress-related signaling pathways. ResultCompared with control group, the model group showed significantly increased serum ALT level and contents of MDA and ROS in liver tissues (P<0.05, P<0.01), significantly reduced activities of SOD and GSH-Px in the liver (P<0.01), significantly increased mRNA expression of Keap1 (P<0.01), and significantly decreased mRNA and protein expressions of Nrf2 and HO-1 (P<0.05, P<0.01). Compared with the model group, the HYT group manifested significantly reduced serum levels of AST, ALT, and ALP (P<0.05, P<0.01), significantly reduced contents of MDA and ROS in liver tissue (P<0.01), significantly increased the activities of SOD and GSH-Px (P<0.01), significantly decreased mRNA expressions of Keap1, p53, and Caspase-3 (P<0.01), and significantly increased mRNA and protein expressions of Nrf2 and HO-1 (P<0.05, P<0.01). ConclusionUnder the condition of 2 times the dose limit of the Pharmacopoeia of the People's Republic of China 2020, Sargassum and Glycyrrhizae Radix et Rhizoma incompatible pair with the HYT on oxidative stress in the liver of goiter rats had different effects. The HYT that contains Sargassum and Glycyrrhizae Radix et Rhizoma has a protective effect on the liver of goiter rats, and the effect is better than that of the HG group, the euthyrox group, and the incomplete groups. Its mechanism may be related to activating the Nrf2/HO-1 signaling pathway to alleviate liver oxidative stress and inhibiting the p53/Caspase-3 signaling pathway to reduce hepatocyte apoptosis.
8.Risk factors for central venous catheter-associated bloodstream infection in patients with emergency hemorrhagic shock
Jianping ZHU ; Yanxin XU ; Shaohong WU ; Feiyao WANG ; Weixing ZHANG ; Ruilan WANG
Chinese Journal of Emergency Medicine 2024;33(5):683-689
Objective:To analyze the risk factors for central venous catheter-associated bloodstream infection in patients with emergency hemorrhagic shock.Methods:Patients with hemorrhagic shock and central venous catheterization admitted to the emergency department of Shanghai First People's Hospital from June 2016 to June 2022 were retrospectively analyzed. The patients were divided into infected group and non-infected group according to whether catheter-related bloodstream infection occurred. The puncture site samples and catheter tip samples of infected patients were collected for etiological detection and drug sensitivity test. The difference of baseline data between the two groups was analyzed, and the relationship between central venous catheter-associated infection and influencing factors was analyzed by multivariate logistic regression.Results:A total of 249 patients were included in this study, including 54 patients in the infected group and 195 patients in the non-infected group. There were significant differences in age, catheterization position, ultrasound-guided puncture, acute physiology and chronic health evaluationⅡ(APACHEⅡ) score, catheter retention time and application of broad-spectrum antibiotics between the infected group and the non-infected group (all P<0.05). Multivariate logistic regression analysis showed that advanced age, femoral vein catheterization, no ultrasound-guided puncture, high APACHEⅡ score, long catheter indent time and application of broad-spectrum antibiotics were risk factors for central venous catheter-associated bloodstream infection. Among the 54 infected patients, Staphylococcus epidermidis accounted for 44.4%, followed by Escherichia coli accounted for 24.1% and fungus accounted for 11.1%. The resistance rate of gram-positive cocci to vancomycin was the lowest (3.4%) followed by tetracycline (34.5%), and the highest resistance rate was amoxicillin (100.0%). The resistance rates of gram-negative bacilli to amikacin and ceftriaxone were relatively high, both of which were 94.7%. Conclusions:The risk factors for central venous catheter-related blood stream infection in patients with emergency hemorrhagic shock included advanced age, femoral vein catheterization, no ultrasound-guided puncture, high APACHEⅡ score, long catheter retention time and use of broad-spectrum antibiotics. The common pathogenic bacteria were Staphylococcus epidermidis and Escherichia coli.
9.Expert consensus on recombinant B subunit/inactivated whole-cell cholera vaccine in preventing infectious diarrhea of enterotoxigenic Escherichia coli
Chai JI ; Yu HU ; Mingyan LI ; Yan LIU ; Yuyang XU ; Hua YU ; Jianyong SHEN ; Jingan LOU ; Wei ZHOU ; Jie HU ; Zhiying YIN ; Jingjiao WEI ; Junfen LIN ; Zhenyu SHEN ; Ziping MIAO ; Baodong LI ; Jiabing WU ; Xiaoyuan LI ; Hongmei XU ; Jianming OU ; Qi LI ; Jun XIANG ; Chen DONG ; Haihua YI ; Changjun BAO ; Shicheng GUO ; Shaohong YAN ; Lili LIU ; Zengqiang KOU ; Shaoying CHANG ; Shaobai ZHANG ; Xiang GUO ; Xiaoping ZHU ; Ying ZHANG ; Bangmao WANG ; Shuguang CAO ; Peisheng WANG ; Zhixian ZHAO ; Da WANG ; Enfu CHEN
Chinese Journal of Clinical Infectious Diseases 2023;16(6):420-426
Enterotoxigenic Escherichia coli(ETEC)infection can induce watery diarrhea,leading to dehydration,electrolyte disturbance,and even death in severe cases. Recombinant B subunit/inactivated whole-cell cholera(rBS/WC)vaccine is effective in preventing ETEC infectious diarrhea. On the basis of the latest evidence on etiology and epidemiology of ETEC,as well as the effectiveness,safety,and health economics of rBS/WC vaccine,National Clinical Research Center for Child Health(The Children’s Hospital,Zhejiang University School of Medicine)and Zhejiang Provincial Center for Disease Control and Prevention invited experts to develop expert consensus on rBS/WC vaccine in prevention of ETEC infectious diarrhea. It aims to provide the clinicians and vaccination professionals with guidelines on using rBS/WC vaccine to reduce the incidence of ETEC infectious diarrhea.
10.Disease burden of prostate cancer from 2014 to 2019 in the United States: estimation from the Global Burden of Disease Study 2019 and Medical Expenditure Panel Survey
Shen LIN ; Dong LIN ; Yiyuan LI ; Lixian ZHONG ; Wei ZHOU ; Yajing WU ; Chen XIE ; Shaohong LUO ; Xiaoting HUANG ; Xiongwei XU ; Xiuhua WENG
Epidemiology and Health 2023;45(1):e2023038-
OBJECTIVES:
The aim of this study was to evaluate the disease burden of prostate cancer (PC) and assess key influencing factors associated with the disease expenditures of PC in the United States.
METHODS:
The total deaths, incidence, prevalence, and disability-adjusted life-years of PC were obtained from the Global Burden of Disease Study 2019. The Medical Expenditure Panel Survey was used to estimate healthcare expenditures and productivity loss and to investigate patterns of payment and use of healthcare resources in the United States. A multivariable logistic regression model was conducted to identify key factors influencing expenditures.
RESULTS:
For patients aged 50 and older, the burden for all age groups showed a modest increase over the 6-year period. Annual medical expenditures were estimated to range from US$24.8 billion to US$39.2 billion from 2014 to 2019. The annual loss in productivity for patients was approximately US$1,200. The top 3 major components of medical costs were hospital inpatient stays, prescription medicines, and office-based visits. Medicare was the largest source of payments for survivors. In terms of drug consumption, genitourinary tract agents (57.0%) and antineoplastics (18.6%) were the main therapeutic drugs. High medical expenditures were positively associated with age (p=0.005), having private health insurance (p=0.016), more comorbidities, not currently smoking (p=0.001), and patient self-perception of fair/poor health status (p<0.001).
CONCLUSIONS
From 2014 to 2019, the national real-world data of PC revealed that the disease burden in the United States continued to increase, which was partly related to patient characteristics.

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