Dyslipidemia is a core risk factor for atherosclerotic cardiovascular diseases (ASCVD). With advances in biotechnology, lipid-lowering strategies have moved from traditional agents (statins and cholesterol-absorption inhibitors) to targeted approaches such as proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors, angiopoietin-like protein 3 (ANGPTL3) inhibitors, and emerging gene-editing tools. This review systematically summarizes the latest therapeutic advances that lower low-density lipoprotein cholesterol (LDL-C) and triglycerides (TG), raise high-density lipoprotein cholesterol (HDL-C), or reduce lipoprotein(a) [Lp(a)], and then dissects the mechanisms of action, clinical evidence, and unmet clinical needs to provide optimized strategies for dyslipidemia management in Chinese populations.
Spinal muscular atrophy (SMA) is a fatal and disabling inherited neuromuscular disorder characterized by progressive weakness in the proximal limbs and trunk muscles, and is often accompanied by multi-system involvement including respiratory, digestive, and skeletal complications. Based on a comprehensive literature review, this article details the clinical trial and real-world evidence of currently approved disease-modifying treatments (DMT) for SMA-nusinersen, onasemnogene abeparvovec, and risdiplam. It also summarizes DMT drugs currently under development and discusses combination and switching strategies, with the aim of providing evidence based pharmacological treatment for SMA in the DMT era.
Polycystic ovary syndrome (PCOS) is a reproductive endocrine disorder influenced by multiple factors, including genetics and environment. Its main pathological features include ovulatory dysfunction, polycystic ovarian morphology, and hyperandrogenemia, often accompanied by metabolic disturbances such as overweight/obesity, insulin resistance, and abnormal glucose and lipid metabolism. Glucagon-like-peptide-1 receptor agonist (GLP-1RA) exert therapeutic effects in PCOS through multiple mechanisms, including modulation of the hypothalamic appetite regulating center, regulation of vagus nerve-mediated signal transduction, induction of white adipose tissue browning, and intervention in the hypothalamic-pituitary-ovarian axis. These actions contribute to weight loss, improvement of reproductive function, alleviation of chronic inflammation, and regulation of hormone levels in PCOS patients, demonstrating promising clinical potential. This article systematically reviews the mechanisms of action of GLP-1RA in the treatment of PCOS and discuss its efficacy and safety in clinical application.
Personalized precision therapy has emerged as a critical direction in antitumor drug treatment. However, its implementation in clinical practice remains challenging due to the complexity of data integration, difficulties in optimizing treatment decisions for certain patient populations, and the heavy workload faced by clinicians. Machine learning, with its powerful data mining capabilities, can efficiently integrate and analyze multidimensional medical data, assist in identifying potential risks during the treatment process, and optimize therapeutic regimens. These capabilities can help enhance treatment outcomes, improve patient quality of life, and reduce the incidence of adverse reactions. This review summarizes recent progress in the application of machine learning in the field of antitumor pharmacotherapy, explores its current applications and potential in precision medicine, and aims to provide patients with more precise, efficient, and cost-effective treatment options.
PD-1/PD-L1 inhibitors have demonstrated significant efficacy in the treatment of malignant tumors. Clinical studies have shown that combination therapies with PD-1/PD-L1 inhibitors as the core can bring better survival benefits to patients through synergistic effects. Vascular endothelial growth factor (VEGF) is a glycoprotein with potent pro-angiogenic activity, which promoting endothelial cell proliferation and neovascularization. Overexpression of VEGF is observed in various solid tumors, and inhibition of VEGF signaling can enhance the therapeutic efficacy of PD-1/PD-L1 inhibitors by modulating the tumor immune microenvironment. Ivonescimab, developed by Akeso Biopharma, is a humanized bispecific antibody that simultaneously targets PD-1 and VEGF. This article provides an overview of the basic information, mechanism of action, preclinical studies, and clinical development of ivonescimab.
Objective To explore the impact of different eosinophil (EOS) levels on the therapeutic efficacy of budecortide (BGF) and budecortide and formoterol (BFF) in the treatment of asthma, and to provide basis for clinical individualized treatment. Methods From January 2022 to January 2025, 120 asthma patients from Huzhou Hospital of Traditional Chinese Medicine affiliated to Zhejiang Chinese Medical University were selected and randomly divided into BGF (n=60) and BFF (n=60) groups. The asthma control questionnaire-7(ACQ-7), improvement of lung function, inflammatory indicators and adverse events after 4 weeks of treatment were evaluated, and the change rate of lung function in different EOS subgroups was analyzed. Results After treatment, forced expiratory volume in the 1second(FEV1)、forced vital capacity(FVC)and FEV1/FVC in the two groups were increased, ACQ-7 score, FeNO and serum IgE were decreased, and the improvement in BGF group was more obvious than that in BFF group. There was no significant difference in the incidence of adverse events between the two groups. In patients with high EOS, the improvement rates of FEV1, FVC and FEV1/FVC in BGF group were significantly higher than those in BFF group, and the low EOS group also showed dose-dependent improvement. BGF significantly increased the improvement rate of FEV1 in patients with high EOS (β=8.213, P<0.001), and there was a positive interaction with baseline EOS (β=0.023, P=0.002). The improvement rate of FEV1 in BGF of high EOS subgroup was significantly higher than that in BFF (Δ=7.58%, P<0.001), while the dose effect difference in low EOS subgroup was smaller (Δ=3.71%, P=0.112). The stratified interaction of drug type×EOS was significant (P=0.007), indicating that baseline EOS levels are a key indicator for predicting efficacy and guiding drug selection. Conclusion BGF more effectively improves lung function and reduces the level of inflammation in patients with asthma, and the improvement of lung function is more significant in patients with high EOS.
Objective To analyse the prescribing patterns and trends of thrombolytic therapy for acute ischemic stroke (AIS) in China from 2019 to 2024. Methods Using data from the China Hospital Prescription Analysis Project, we analyzed thrombolytic prescriptions from 150 hospitals across 9 cities. Annual prescriptions volumes, costs, and regional utilization rates were systematically assessed. Results Among 10 194 thrombolytic prescriptions analyzed, 68.8% were for male patients (median age: 67 years; 58.3% aged ≥65). Prescriptions increased from 1 471 in 2019 to 2 145 in 2023, reaching 1 296 in the first half of 2024, with a statistically significant growth trend (P=0.024). In contrast, total costs rose from ¥5 605 179.7 in 2019 to ¥6 221 588.4 in 2023 and reached ¥3 744 311.5 in the first half of 2024, but the cost increase was not statistically significant (P=0.060). Alteplase dominated (69.7%), followed by urokinase (29.5%), and tenecteplase adoption surged post-2022 (1.6% to 7.3% in 2024; P<0.001). Prescription data showed that the proportion of AIS patients receiving thrombolytic drugs increased from 0.2% in 2019 to 0.3% in 2024 (P=0.008), with significant growth in Harbin, Hangzhou, Shenyang, and Tianjin (P<0.001). Conclusion Thrombolysis utilization for AIS shows sustained growth with marked regional variations in China. Alteplase remains the primary thrombolytic drug, while tenecteplase demonstrates promising clinical application prospects.
Objective To systematically evaluate the efficacy and safety of remimazolam for sedation in elderly patients undergoing painless gastroscopy, so as to provide evidence for clinical sedative drug selection. Methods A comprehensive search was conducted in both Chinese and English databases including PubMed, Scopus, Embase, Web of Science, Chinese Biomedical Literature Database, CNKI, and Wanfang Database up to October 31st 2024. Meta-analysis was performed to pool outcome measures. Publication bias was assessed using funnel plots and Egger's test. Results A total of 10 studies were included, comprising 1581 patients in the observation group and 1580 in the control group. The effective sedation rate was slightly lower in the observation group than in the control group (OR=0.63, 95%CI: 0.40-0.98, P=0.04), with low heterogeneity (I²=0%). The sedation onset time was significantly longer in the observation group (WMD=18.06, 95%CI: 11.34-24.78, P<0.05), with high heterogeneity (I²=98%). Subgroup analysis indicated that differences among centers were the main source of heterogeneity (I²=98%, P>0.05), and inter-subgroup differences were statistically significant (P<0.05). Combined results showed that adverse events including hypotension, respiratory depression, injection pain, body movement, and nausea/vomiting were all lower in the observation group than in the control group (P<0.05). The funnel plot for effective sedation rate showed a symmetric scatter distribution without points outside the confidence interval. Egger's test suggested no significant publication bias (t=1.45, P=0.32). Conclusion Remimazolam demonstrates superior safety compared to commonly used control sedatives in elderly patients undergoing gastroscopy, significantly reducing adverse events such as hypotension, respiratory depression, injection pain, and body movement. It offers reliable efficacy with a lower risk profile.
Objective To analyze the serum concentration monitoring results of levetiracetam and its influencing factors in adult patients with epilepsy, thus to provide evidence for individual administration of levetiracetam. Methods Epileptic patients who received levetiracetam treatment and therapeutic drug monitoring (TDM) of levetiracetam in our hospital from October 2020 to August 2024 were selected as study subjects. The steady-state trough concentration of serum levetiracetam was measured. Univariate analysis and multiple linear regression were used to analyze the effects of daily dose, body weight, age, gender, renal function, combined medication, and other factors on levetiracetam concentration. Results A total of 128 levetiracetam blood concentration samples from 89 patients were included in this study. Levetiracetam concentration ranged from 0.94 to 62.97 mg/L, and the target attainment rate was 41.41%. The coefficient of variation of levetiracetam concentration/dose (CDR) in the 89 patients was calculated to be 73.35%, suggesting significant inter-individual pharmacokinetic variability. Univariate analysis showed that age, creatinine, creatinine clearance (Ccr) and urea significantly affected levetiracetam CDR. Further multiple linear regression analysis showed that Ccr and combined use of sodium valproate were independent factors causing inter-individual variation of CDR. Conclusion Levetiracetam concentration is positively correlated with the dosage, but there is significant pharmacokinetic variation among individuals. For patients with epilepsy who have renal insufficiency or receiving valproate, optimization of the levetiracetam dosage can be achieved through TDM to reduce the impact of pharmacokinetic variation among individuals.
Objective To investigate the clinical value of omalizumab in patients with eosinophilic chronic sinusitis with nasal polyps (eCRSwNP) after nasal endoscopic surgery, and to provide evidence for optimizing postoperative management strategies. Methods Patients with eCRSwNP who underwent nasal endoscopic surgery between January and December 2023 were randomly assigned to either an observation group (receiving subcutaneous omalizumab) or a control group (receiving nasal spray of mometasone furoate). Treatment lasted for a total of 4 months. Efficacy and adverse reactions were compared between the two groups, and prognosis was assessed over a 12-month follow-up period. Results A total of 77 patients were enrolled, including 38 in the observation group and 39 in the control group. After 4 months of treatment, both groups showed reductions in VAS scores for nasal congestion, runny nose, and olfaction, as well as in SNOT-22, modified Lund-Kennedy (MLK), and nasal polyp score (NPS). Additionally, levels of eosinophil (Eos) absolute value, allergen specific immunoglobulin E (sIgE), matrix metalloproteinase-9 (MMP-9), and transforming growth factor-β1(TGF-β1) levels decreased in both groups, with more significant reductions observed in the observation group. At 12 months postoperatively, the observation group had significantly shorter nasal mucosal epithelialization time and fewer acute sinusitis episodes compared to the control group [(6.25±1.53)weeks vs (9.87±2.14)weeks,(0.52±0.21) vs (1.89±0.79) episodes, respectively]. There was no significant difference in the incidence of adverse drug reactions. During follow-up, 6 patients (15.79%) in the experimental group experienced recurrence, compared with 15 patients (38.46%) in the control group; this difference was statistically significant (χ²=4.856,P=0.028). Conclusion Omalizumab assisted nasal endoscopic surgery can effectively improve postoperative symptoms and endoscopic scores, reduce recurrence risk, and maintain a favorable safety profile. It represents an effective therapeutic option for early postoperative control of eosinophilic inflammation in patients with eCRSwNP.
Objective To analyze the clinical characteristics and patterns of drug-induced hypofibrinogenemia (HFIB) in hospitalized patients, providing references for prevention and management of HFIB. Methods Using the Adverse drug event active surveillance and assessment system-Ⅱ(ADE-ASAS-Ⅱ), data from hospitalized patients at the first medical center of a tertiary hospital between 2022 and 2023 were extracted retrospectively. System-alerted cases underwent manual causality assessment per WHO-UMC criteria. We evaluated drug profiles, demographics, time-to-onset, severity (per CTCAE v5.0), and clinical outcomes. Results Among 202 783 monitored hospitalizations, 2 777 cases triggered system alerts, with 1 533 confirmed HFIB cases (0.76%, 1 533/202 783). The male-to-female ratio was 1.36:1, and 57.66% of patients were aged ≥60 years. Mild to moderate HFIB cases accounted for 81.93% (1 256/1 533). HFIB involved 164 drugs across 11 classes, predominantly anti-infectives and hematologic agents. The top five suspected drugs causing hFIB were bothrops atrox (363 cases, 23.68%), tigecycline (260 cases, 16.96%), methylprednisolone (81 cases, 5.28%), hemocoagulase bothrops atrox (67 cases, 4.37%), and cefoperazone-sulbactam (66 cases, 4.31%). The median time-to-onset of HFIB was 2 days for batroxobin and snake venom-derived hemocoagulases, compared to 7 days for tigecycline. Severe or life-threatening HFIB occurred in 54.82% (199/363) of batroxobin cases, significantly higher than tigecycline (8.08%, 21/260) and snake venom hemocoagulases (10.61%, 19/179), with a statistically significant difference (P<0.01). Conclusion Drug-induced HFIB is associated with a wide range of medications, primarily including batroxobin, tigecycline, and snake venom hemocoagulases. Significant variations exist in time-to-onset and severity across drugs. Enhanced monitoring and early warning measures should be implemented for high-risk populations based on the characteristics of HFIB-inducing drugs.
Objective To evaluate the treatment effects of first or second-generation epidermal growth factor receptor (EGFR) tyrosine kinase inhibitors (TKIs) compared to third-generation TKIs in patients with EGFR-sensitive mutations and locally advanced or metastatic non-small cell lung cancer (NSCLC). Methods Patients diagnosed with locally advanced or metastatic non-small cell lung cancer and identified as EGFR-sensitive mutations through pathology and genetic testing in the Department of Respiratory and Critical Care Medicine at Peking University Shougang hospital between January 2018 and December 2023 were selected. Patients were categorized into 1st-generation and 3rd-generation EGFR TKI groups. After balancing confounding factors through inverse probability treatment weighting, we analyzed the efficacy and survival of each group. Results 52 patients with EGFR-sensitive mutations were enrolled. After inverse probability treatment weighting, there were no statistically significant differences in clinical characteristics between the two groups (|SMD|<0.1). The overall objective response rate (ORR), median real-world progression-free survival (rwPFS), and median overall survival (OS) were compared between the groups, with no significant differences observed (P>0.05). Conclusion In locally advanced or metastatic EGFR sensitive mutation NSCLC patients, 3rd-generation and 1st-generation EGFR TKIs show comparable efficacy and can be used as first-line treatments for EGFR-sensitive mutations.
Objective To evaluate the clinical comprehensive value of shenquxiaoshi oral liquid in treating pediatric functional dyspepsia (FD). Methods Based on literature and data analysis, a clinical comprehensive evaluation framework was applied to compare Shenquxiaoshi oral liquid with domperidone formulations in terms of effectiveness, safety, and cost-effectiveness, etc. Results Shenquxiaoshi oral liquid demonstrates efficacy in improving gastrointestinal motility. Compared to domperidone, it achieves a higher clinical response rate in children with FD, while exhibiting a comparable adverse reaction incidence. Economically, although the unit price of shenquxiaoshi oral liquid is higher than domperidone, the total course-of-treatment cost remains within an affordable range. In other aspects, the herbal preparation features a scientifically formulated composition, convenient storage and administration, and relatively clear usage principles, largely meeting the clinical needs for pediatric FD treatment. Conclusion Shenquxiaoshi oral liquid is suitable for children with FD and demonstrates broad clinical applicability.
Objective To conduct a clinical comprehensive evaluation of cefcapene pivoxil as an oral antimicrobial agent for the treatment of pediatric infectious diseases. Methods This study evaluated effectiveness, safety, economy, suitability, accessibility, and innovation of cefcapene pivoxil using qualitative and quantitative methods. Results Cefcapene pivoxil showed a lower eradication rate against Group A Streptococcus than cefteram pivoxil but higher than azithromycin. It demonstrated higher efficacy in treating moderate-to-severe infections compared to cefteram pivoxil and a significantly lower risk of treatment failure in urinary tract infections than amoxicillin. Its efficacy in preventing postoperative oral infections was higher than that of amoxicillin. Overall safety profiles were similar across agents. Cefcapene pivoxil had a lower incidence of digestive and cutaneous ADRs compared to amoxicillin. Signal detection from the database revealed that cutaneous ADRs had the highest frequency among children treated with cefcapene pivoxil. Cefcapene pivoxil was a pediatric-specific drug with a broad antibacterial spectrum and low protein binding rate. No significant differences were observed between cefcapene pivoxil and comparator drugs (cefteram pivoxil, cefdinir, cefaclor, azithromycin, amoxicillin) in terms of other pharmacokinetic parameters, dosage forms, taste, management costs, chemical structures, or formulation performance. Cefcapene pivoxil was covered under China's National Medical Insurance (Category B); imported versions were more expensive than domestic alternatives, but all options remained affordable. Conclusion Compared to cefteram pivoxil, cefdinir, cefaclor, azithromycin, and amoxicillin, cefcapene pivoxil demonstrates superior efficacy in the treatment of moderate to severe infections and urinary tract infections, with comparable safety and moderate cost-effectiveness.
This article reports a case of a patient with liver abscess infected by carbapenem-resistant Klebsiella Pneumoniae during midtrimester pregnancy. The clinical pharmacist reviewed relevant domestic and foreign literatures, and recommended anti-infective therapy with meropenem in combination with fosfomycin based on a comprehensive assessment of efficacy and safety considerations. The patient's condition improved significantly with no observed adverse reactions.. Clinical pharmacists play a critical role in the individualized treatment of pregnant patients by participating throughout the clinical decision-making process, including drug therapy selection, dosage regimen determination, and pharmaceutical care, thereby ensuring medication safety and efficacy.