1.Key points of the International consensus guidelines on the implementation and monitoring of vosoritide therapy in individuals with Achondroplasia.
Hangyu PING ; Ran DING ; Cheng HUANG ; Yue PENG ; Zikang ZHONG ; Weiguo WANG
Chinese Journal of Medical Genetics 2026;43(1):5-12
Achondroplasia (ACH) is a common inherited skeletal dysplasia (inherited dwarfism) that compromises quality of life across the lifespan. In 2021, vosoritide became the first approved precision therapy for ACH and is now available in more than 40 countries. Compared with prior symptomatic measures, vosoritide has demonstrated favorable efficacy and a reassuring safety profile. Nevertheless, existing international ACH guidelines largely emphasize complication management and symptomatic care, and there is no unified consensus on pharmacologic therapy. To address this gap, an international expert group developed the International Consensus Guidelines for the Implementation and Monitoring of Vosoritide Therapy in Patients with Achondroplasia providing systematic recommendations that span the continuum of care - from initial patient contact and pre-treatment assessment to medication counseling, injection training, and long-term outcome monitoring. These recommendations complement and refine current management and nursing protocols for individuals with ACH and offer practical guidance for clinicians across diverse regions. This article highlights key elements of the guideline to provide evidence-based support and clinical direction for healthcare professionals in China treating children with ACH using vosoritide.
Humans
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Achondroplasia/drug therapy*
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Consensus
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Practice Guidelines as Topic
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Child
2.Pharmacodynamic Substances and Mechanisms of Xinglou Chengqi Tang in Treating Post-stroke Complications: A Review
Yujin ZHANG ; Xiangzhuo LIU ; Zhouyang CHEN ; Zihao SONG ; Xinyi LIU ; Yizhi YAN ; Chaoya LI ; Yingyan FANG ; Shasha YANG ; Xueqin CHENG ; Zhou XIE ; Sijie TAN ; Peng ZENG ; Yue ZHANG
Chinese Journal of Experimental Traditional Medical Formulae 2026;32(1):327-337
Stroke is the leading cause of death and disability among adults in China, and its common complications include digestive system abnormalities, cognitive impairment, depression, stroke-associated pneumonia, and hemiplegia. The combination of traditional Chinese and Western medicine has great potential in treating post-stroke complications. Xinglou Chengqitang (XLCQT) is a representative prescription of alleviating the disease in the upper part by treating the lower part. It has definite therapeutic effect and high safety. Clinically, XLCQT is often used to treat stroke and its complications. However, the quantity and quality of clinical trials of XLCQT in treating post-stroke complications need to be improved. Additionally, since the basic research is weak, the material basis and multi-target mechanism for the efficacy of this prescription are unknown. This article reviews XLCQT in terms of the pharmacodynamic basis, medicinal properties, safety evaluation, and progress in clinical research and mechanisms in treating post-stroke complications. This article summarizes 22 key active ingredients of XLCQT in treating acute stroke complicated with syndrome of phlegm heat and fu-organ excess. Among these key active ingredients, resveratrol, kaempferol, luteolin, chrysoeriol, apigenin, (+)-catechin, and adenosine have good pharmacokinetic properties and high bioavailability. The mechanisms of XLCQT in treating post-stroke complications are complex, including inflammatory response, brain-gut axis, hypothalamic-pituitary-adrenal (HPA) axis, intestinal flora, neurotrophic factors, autophagy, oxidative stress, and free radical damage. This review helps to deeply understand the pharmacodynamic basis and mechanisms of XLCQT in treating post-stroke complications and provides a theoretical basis for the clinical application of XLCQT against post-stroke complications and the development of drugs.
3.Evaluation of the quality of Jingangteng capsules based on UPLC fingerprinting combined with multi-component content determination
Li SHEN ; Yue SHEN ; Yuying YANG ; Dandan ZHANG ; Yuxi WU ; Xuxiang ZHOU ; Jingyu YANG ; Peng HU ; Lei WANG ; Heming WU ; Dan LIU ; Xiaochuan YE
China Pharmacy 2026;37(10):1290-1294
OBJECTIVE To establish the UPLC fingerprint and the method for multi-component content determination in Jingangteng capsules, and to evaluate its quality by combining chemical pattern recognition analysis. METHODS An UPLC method was established. Separation was performed on a Zorbax SB-C 18 Rapid Resolution HD column, with acetonitrile-0.1% formic acid as the mobile phase for gradient elution.Using the Similarity Evaluation System for Chromatographic Fingerprints of Traditional Chinese Medicines (2012 edition), UPLC fi ngerprints were established for 10 batches of Jingangteng capsules, and similarity was evaluated. SPSS 22.0 and SIMCA 14.1 software were used to perform hierarchial-cluster analysis and orthogonal partial least squares discriminant analysis (OPLS-DA), respectively. The same UPLC method was employed to determine the contents of chlorogenic acid, 3,5-dihydroxy-2-methylbenzoic acid-3- O -glucoside (M1), caffeic acid, astilbin, oxyresveratrol, quercitrin and resveratrol in the 10 batches of samples. RESULTS A total of 17 common peaks were identified in UPLC fingerprints of the 10 batches of samples, of which 7 were identified as chlorogenic acid, M1, caffeic acid, astilbin, oxyresveratrol, quercitrin, and resveratrol. The similarities of 10 batches of samples ranged from 0.820 to 0.985. The results of hierarchial-cluster analysis showed that 10 batches of samples were grouped into four categories: S1-S4 formed one group, S5 and S6 formed another, S7, S8 and S10 formed a third, and S9 formed a fourth, consistent with the OPLS-DA results; the variable importance projection values for peaks 7, 10, 2, 16 (resveratrol), 13 (oxyresveratrol), 11, 6 (caffeic acid), 5 (M1) and 15 (quercitrin) were >1. Quantitative analysis results showed that the contents of chlorogenic acid, M1, caffeic acid, astilbin, oxyresveratrol, quercitrin, and resveratrol were 1.650 8-4.213 7, 0.636 2-2.161 7, 0.031 0-0.086 5, 0.239 1-1.069 3, 0.211 9-1.104 0, 0.488 8-2.399 2, and 0.164 0-0.699 8 mg/g, respectively. CONCLUSIONS UPLC fingerprint and content determination methods established in this study are simple to operate, accurate, reliable and reproducible; when combined with chemical pattern recognition analysis, they can be used to evaluate the quality of Jingangteng capsules. Nine components, such as resveratrol, oxyresveratrol, caffeic acid, M1 and quercitrin, may serve as markers of quality variation.
4.Activation of the Gamma-Aminobutyric Acid (GABA)ergic Neural Circuit in Salicylate-Induced Tinnitus: the Inferior Colliculus to the Medial Geniculate Body
Xu-Yuan PENG ; Jiang WANG ; Ming-Yue GONG ; Li-Yuan ZHANG ; Min ZHANG ; Zhi-Bin CHEN ; Zheng-Quan TANG ; Lei CHENG
Clinical and Experimental Otorhinolaryngology 2026;19(1):55-69
Objectives:
. This study aimed to investigate the regulatory functions of gamma-aminobutyric acid (GABA)ergic neural circuits from the inferior colliculus (IC) to the medial geniculate body (MGB) in salicylate-induced tinnitus.
Methods:
. Mice were treated with salicylate to induce tinnitus, and tinnitus-like behaviors were evaluated via gap prepulse inhibition of acoustic startle. Using combined viral tracing methodologies, we identified and mapped the pathways and connections from the IC to the MGB. Furthermore, we employed Gq-coupled human M3 designer receptors exclusively activated by designer drugs (DREADDs) and Gi-coupled human M4 DREADDs to achieve targeted excitation or suppression of GABAergic neurons in the IC and MGB. Following the administration of clozapine N-oxide, which binds to these receptors, we modulated these neural circuits to assess their impact on tinnitus severity in a mouse model.
Results:
. Our findings demonstrated that mice exposed to salicylate exhibited tinnitus-like behaviors. GABAergic neurons projecting retrogradely from the MGB to the IC were primarily concentrated in the external nucleus of the IC. After clozapine N-oxide administration, chemogenetic activation of IC-MGB GABAergic neurons aggravated salicylate-induced tinnitus. Additionally, activation of GABAergic neurons between the IC and MGB induced the perception of tinnitus even without salicylate. However, chemogenetic inhibition of the IC-MGB GABAergic circuit did not reverse salicylate-induced tinnitus.
Conclusion
. These findings suggest that activation of the IC-MGB GABAergic neural circuit may contribute to tinnitus generation through a mechanism distinct from that of salicylate-induced tinnitus. This study provides novel insights into the mechanisms underlying tinnitus.
5.Umbrella decision-making model for diagnosis and treatment of elderly lung cancer patients: Construction and practice
Lunxu LIU ; Jian ZHOU ; Xiang DING ; Nan CHEN ; Jianxin XUE ; Xuelei MA ; Ye WANG ; Weiya WANG ; Liqing PENG ; Xin YOU ; Minggang SU ; Xu CHENG ; Jiao WANG ; Ning GE ; Deying KANG ; Yuchen HUANG ; Jinghan WANG ; Yu TONG ; Yaoxi ZHANG ; Jirong YUE ; Hu LIAO
Chinese Journal of Clinical Thoracic and Cardiovascular Surgery 2026;33(06):833-839
With the accelerating trend of population aging, the number of elderly patients with lung cancer continues to rise, and the disease burden is becoming increasingly heavy. The clinical management of these patients faces severe challenges due to their decreased physiological reserve, complex comorbidities, and significant individual heterogeneity. Consequently, under traditional diagnosis and treatment models, doctors often struggle to identify the individualized risks of elderly patients in a timely and comprehensive manner, which can easily lead to decision biases such as undertreatment or overtreatment. In view of this, this study advocates for the establishment of an umbrella decision-making model specifically tailored for elderly lung cancer patients. Grounded in a multidisciplinary team (MDT) platform, this model deeply integrates oncological indicators with the comprehensive geriatric assessment (CGA) system. By holistically considering multidimensional variables including tumor burden, organ function, frailty index, cognitive status, and social support, the model establishes an operational mechanism characterized by "single entry, precise stratification, and targeted selection". Accordingly, patients can be scientifically triaged into distinct intervention tiers, such as active surveillance, minimally invasive surgery, drug therapy, radiotherapy, and best supportive care, thereby achieving real-time alignment between treatment intensity and patient fitness. This article elaborates on the construction logic and key operational procedures of this novel decision-making framework, aiming to guide clinical practice beyond the limitations of a tumor-centric perspective toward a holistic, dynamic, whole-course management strategy. This transition seeks to ensure optimal quality of life and clinical net benefit for elderly patients alongside survival prolongation.
6.Study on anti-atherosclerosis mechanism of blood components of Guanxin Qiwei tablets based on HPLC-Q-Exactive-MS/MS and network pharmacology
Yuan-hong LIAO ; Jing-kun LU ; Yan NIU ; Jun LI ; Ren BU ; Peng-peng ZHANG ; Yue KANG ; Yue-wu WANG
Acta Pharmaceutica Sinica 2025;60(2):449-458
The analysis presented here is based on the blood components of Guanxin Qiwei tablets, the key anti-atherosclerosis pathway of Guanxin Qiwei tablets was screened by network pharmacology, and the anti-atherosclerosis mechanism of Guanxin Qiwei tablets was clarified and verified by cell experiments. HPLC-Q-Exactive-MS/MS technique was used to analyze the components of Guanxin Qiwei tablets into blood, to determine the precise mass charge ratio of the compounds, and to conduct a comprehensive analysis of the components by using secondary mass spectrometry fragments and literature comparison. Finally, a total of 42 components of Guanxin Qiwei tablets into blood were identified. To better understand the interactions, we employed the Swiss Target Prediction database to predict the associated targets. Atherosclerosis (AS) disease targets were searched in disease databases Genecard, OMIM and Disgent, and 181 intersection targets of disease targets and component targets were obtained by Venny 2.1.0 software. Protein interactions were analyzed by String database. The 32 core targets were selected by Cytscape software. Gene Ontology (GO) enrichment analysis and Kyoto Encyclopedia of Genes and Genomes (KEGG) pathway analysis were performed in DAVID database. It was found that the anti-atherosclerosis pathways of Guanxin Qiwei tablets mainly include lipid metabolism and atherosclerosis and AGE-RAGE signaling pathway in diabetic complications and other signal pathways. The core targets and the core compounds were interlinked, and it was found that cryptotanshinone and tanshinone ⅡA in Guanxin Qiwei tablets were well bound to TNF, PPAR
7.Pharmacokinetic study of 3 blood-absorbed components of Xiangshao sanjie oral liquid in rats with hyperplasia of mammary gland
Yu ZHANG ; Jiaming LI ; Dan PENG ; Ruoqiu FU ; Yue MING ; Zhengbi LIU ; Jingjing WANG ; Shiqi CHENG ; Hongjun XIE ; Yao LIU
China Pharmacy 2025;36(6):680-685
OBJECTIVE To explore the pharmacokinetic characteristics of 3 blood-absorbed components of Xiangshao sanjie oral liquid in rats with hyperplasia of mammary gland (HMG). METHODS Female SD rats were divided into control group and HMG group according to body weight, with 6 rats in each group. The HMG group was given estrogen+progesterone to construct HMG model. After modeling, two groups were given 1.485 g/kg of Xiangshao sanjie oral liquid (calculated by crude drug) intragastrically, once a day, for 7 consecutive days. Blood samples were collected before the first administration (0 h), and at 5, 15, 30 minutes and 1, 2, 4, 8, 12, 24 hours after the last administration, respectively. Using chlorzoxazone as the internal standard, the plasma concentrations of ferulic acid, paeoniflorin and rosmarinic acid in rats were detected by UPLC-Q/TOF-MS. The pharmacokinetic parameters [area under the drug time curve (AUC0-24 h, AUC0-∞), mean residence time (MRT0-∞), half-life (t1/2), peak time (tmax), peak concentration (cmax)] were calculated by the non-atrioventricular model using Phoenix WinNonlin 8.1 software. RESULTS Compared with the control group, the AUC0-24 h, AUC0-∞ and cmax of ferulic acid in the HMG group were significantly increased (P<0.05); the AUC0-24 h, AUC0-∞ , MRT0-∞ , t1/2 and cmax of paeoniflorin increased, but there was no significant difference between 2 groups (P>0.05); the AUC0-24 h and MRT0-∞ of rosmarinic acid were significantly increased or prolonged (P<0.05). C ONCLUSIONS In HMG model rats, the exposure of ferulic acid, paeoniflorin and rosmarinic acid in Xiangshao sanjie oral liquid all increase, and the retention time of rosmarinic acid is significantly prolonged.
8.Pharmacodynamic Substances and Mechanisms of Da Chengqitang in Treating Stroke: A Review
Yizhi YAN ; Xinyi LIU ; Yang DUAN ; Miaoqing LONG ; Chaoya LI ; Qiang LI ; Yi'an CHEN ; Shasha YANG ; Yue ZHANG ; Peng ZENG
Chinese Journal of Experimental Traditional Medical Formulae 2025;31(9):297-306
Stroke is the main cause of death and disability among adults in China and is characterized by high incidence, disability, mortality, and recurrence rates. The combination of traditional Chinese and Western medicine has great potential in treating stroke and its sequelae. The classic traditional Chinese medicine prescription Da Chengqitang (DCQT) has a long history and proven efficacy in treating stroke. Clinically, DCQT is often used to treat stroke and its sequelae. However, the number and quality of clinical trials of DCQT in treating stroke need to be improved. Because of the insufficient basic research, the active ingredients and multi-target mechanism of action of DCQT remain unclear. Our research group has previously confirmed that DCQT can effectively reverse neurological damage, reduce iron deposition, and downregulate the levels of pro-inflammatory cytokines in the rat model of hemorrhagic stroke. The treatment mechanism is related to the nuclear factor erythroid 2-related factor 2 (Nrf2)-mediated signaling pathway and p38 mitogen-activated protein kinase (MAPK) signaling-mediated microglia activation. To clarify the pharmacodynamic basis and anti-stroke mechanism of DCQT, this article reviews the research progress in the treatment of stroke with DCQT in terms of clinical trials, pharmacodynamic material basis, safety evaluation, and mechanisms of absorbed components. This article summarizes 45 major phytochemical components of DCQT, 11 of which are currently confirmed absorbed components. Among them, emodin, rhein, chrysophanol, aloe-emodin, synephrine, hesperidin, naringin, magnolol, and honokiol can be used as quality markers (Q-markers) of DCQT. The mechanism of DCQT in treating stroke is complex, involving regulation of inflammatory responses, neuronal damage, oxidative stress, blood-brain barrier, brain-derived neurotrophic factor, and anti-platelet aggregation. This article helps to deeply understand the pharmacodynamic basis and mechanism of DCQT in treating stroke and provides a theoretical basis for the clinical application of DCQT in treating stroke and the development of stroke drugs.
9.Discriminating Tumor Deposits From Metastatic Lymph Nodes in Rectal Cancer: A Pilot Study Utilizing Dynamic Contrast-Enhanced MRI
Xue-han WU ; Yu-tao QUE ; Xin-yue YANG ; Zi-qiang WEN ; Yu-ru MA ; Zhi-wen ZHANG ; Quan-meng LIU ; Wen-jie FAN ; Li DING ; Yue-jiao LANG ; Yun-zhu WU ; Jian-peng YUAN ; Shen-ping YU ; Yi-yan LIU ; Yan CHEN
Korean Journal of Radiology 2025;26(5):400-410
Objective:
To evaluate the feasibility of dynamic contrast-enhanced MRI (DCE-MRI) in differentiating tumor deposits (TDs) from metastatic lymph nodes (MLNs) in rectal cancer.
Materials and Methods:
A retrospective analysis was conducted on 70 patients with rectal cancer, including 168 lesions (70 TDs and 98 MLNs confirmed by histopathology), who underwent pretreatment MRI and subsequent surgery between March 2019 and December 2022. The morphological characteristics of TDs and MLNs, along with quantitative parameters derived from DCE-MRI (K trans , kep, and v e) and DWI (ADCmin, ADCmax, and ADCmean), were analyzed and compared between the two groups.Multivariable binary logistic regression and receiver operating characteristic (ROC) curve analyses were performed to assess the diagnostic performance of significant individual quantitative parameters and combined parameters in distinguishing TDs from MLNs.
Results:
All morphological features, including size, shape, border, and signal intensity, as well as all DCE-MRI parameters showed significant differences between TDs and MLNs (all P < 0.05). However, ADC values did not demonstrate significant differences (all P > 0.05). Among the single quantitative parameters, v e had the highest diagnostic accuracy, with an area under the ROC curve (AUC) of 0.772 for distinguishing TDs from MLNs. A multivariable logistic regression model incorporating short axis, border, v e, and ADC mean improved diagnostic performance, achieving an AUC of 0.833 (P = 0.027).
Conclusion
The combination of morphological features, DCE-MRI parameters, and ADC values can effectively aid in the preoperative differentiation of TDs from MLNs in rectal cancer.
10.Discriminating Tumor Deposits From Metastatic Lymph Nodes in Rectal Cancer: A Pilot Study Utilizing Dynamic Contrast-Enhanced MRI
Xue-han WU ; Yu-tao QUE ; Xin-yue YANG ; Zi-qiang WEN ; Yu-ru MA ; Zhi-wen ZHANG ; Quan-meng LIU ; Wen-jie FAN ; Li DING ; Yue-jiao LANG ; Yun-zhu WU ; Jian-peng YUAN ; Shen-ping YU ; Yi-yan LIU ; Yan CHEN
Korean Journal of Radiology 2025;26(5):400-410
Objective:
To evaluate the feasibility of dynamic contrast-enhanced MRI (DCE-MRI) in differentiating tumor deposits (TDs) from metastatic lymph nodes (MLNs) in rectal cancer.
Materials and Methods:
A retrospective analysis was conducted on 70 patients with rectal cancer, including 168 lesions (70 TDs and 98 MLNs confirmed by histopathology), who underwent pretreatment MRI and subsequent surgery between March 2019 and December 2022. The morphological characteristics of TDs and MLNs, along with quantitative parameters derived from DCE-MRI (K trans , kep, and v e) and DWI (ADCmin, ADCmax, and ADCmean), were analyzed and compared between the two groups.Multivariable binary logistic regression and receiver operating characteristic (ROC) curve analyses were performed to assess the diagnostic performance of significant individual quantitative parameters and combined parameters in distinguishing TDs from MLNs.
Results:
All morphological features, including size, shape, border, and signal intensity, as well as all DCE-MRI parameters showed significant differences between TDs and MLNs (all P < 0.05). However, ADC values did not demonstrate significant differences (all P > 0.05). Among the single quantitative parameters, v e had the highest diagnostic accuracy, with an area under the ROC curve (AUC) of 0.772 for distinguishing TDs from MLNs. A multivariable logistic regression model incorporating short axis, border, v e, and ADC mean improved diagnostic performance, achieving an AUC of 0.833 (P = 0.027).
Conclusion
The combination of morphological features, DCE-MRI parameters, and ADC values can effectively aid in the preoperative differentiation of TDs from MLNs in rectal cancer.

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