To compare the clinical efficacy of daratumumab injection combined with lenalidomide capsules and dexamethasone injection in the treatment of multiple myeloma (MM).

Patients with MM were divided into control group and treatment group according to the treatment method. Patients in the control group received the bortezomib for injection, lenalidomide capsules and dexamethasone injection (VRd) regimen; patients in the treatment group received the daratumumab injection, lenalidomide capsules and dexamethasone injection (DRd) regimen. The VRd regimen: Within each 28-day cycle, bortezomib for injection at 1.3 mg·m^-2, lenalidomide capsules at 25 mg·d^-1 and dexamethasone injection at 20 mg·d^-1 were administered according to the standard protocol. The DRd regimen: Based on lenalidomide capsules and dexamethasone injection , daratumumab injection at 16 mg·kg^-1 was added (once weekly in cycles 1-2, once every 2 weeks in cycles 3-6, and once every 4 weeks thereafter). Efficacy was evaluated after 3 cycles of treatment in both groups. Clinical efficacy, MM-related biomarkers and adverse reactions were compared between the two groups.

The study enrolled 88 patients, with 45 cases in the control group and 43 cases in the treatment group. Post-treatment evaluation revealed objective response rates (ORR) of 77.78% (35 cases/45 cases) and 90.70% (39 cases/43 cases) for the control and treatment groups, respectively, demonstrating no statistically significant intergroup difference (P>0.05). After treatment, the levels of hemoglobin of control group and treatment group were (102.85±15.87) and (109.71±16.24) g·L^-1, respectivley; the levels of β2-microglobulin (β2-MG) were (4.29±1.12) and (3.67±1.03) mg·L^-1, respectivley; the levels of lactate dehydrogenase (LDH) were (167.29±30.92) and (152.32±29.67) U·L^-1, respectivley; the levels of monoclonal protein (M protein) were (17.42±3.87) and (15.39±3.42) g·L^-1, respectivley; bone marrow plasma cells were (10.25±2.13)% and (8.92±2.06)%, respectivley; cluster of differentiation 3 positive (CD3⁺) were (62.45±8.74)% and (67.06±9.12)%, respectivley; CD4⁺ levels were (34.26±5.12)% and (37.68±5.33)%, respectivley; CD4⁺/CD8⁺ ratios were 1.16±0.24 and 1.28±0.22, respectivley. Compared the control group with treatment group, the differences of above indexes were all statistically significant (all P<0.05). The total incidence of peripheral neuropathy of control group and treatment group were 22.22% (10 cases/45 cases) and 2.33% (1 case/43 cases), respectivley; myelosuppression were 15.56% (7 cases/45 cases) and 37.21% (16 cases/43 cases), respectivley; infection were15.56% (7 cases/45 cases) and 34.88% (15 cases/43 cases), respectivley, all with statistically significant differences (P<0.05, P<0.01).

While ensuring clinical efficacy, the DRd regimen exerts more significant effects in improving immune function, levels of MM-related biomarkers, and quality of life, and significantly reduces the incidence of peripheral neuropathy. However, it is associated with increased risks of myelosuppression and infection.

To evaluate the clinical efficacy and safety of intrathecal nusinersen combined with risdiplam in children with spinal muscular atrophy (SMA).

A total of 75 children with SMA were divided into a control group and an experimental group. The control group received intrathecal nusinersen (12 mg) at loading doses on days 0, 14, 28, and 63, followed by maintenance doses every 4 months for 12 months. The experimental group received additional oral risdiplam once daily, with the dose adjusted according to age and body weight, for 12 months. Clinical efficacy, motor function, pulmonary function, and adverse drug reactions were compared between the two groups.

Following treatment, the overall response rates in the experimental group and control group were 91.67% (33 cases/36 cases) and 82.05% (32 cases/39 cases), respectively, with no statistically significant difference between the two groups. (P>0.05). After treatment, the revised upper extremity module (RULM) scores in the experimental group and control group were 32.69±2.07 and 31.59±1.97, respectively; the Hammersmith Functional Motor Scale Expanded (HFMSE) scores for spinal muscular atrophy were 27.75±6.19 and 24.62±6.08, respectively; the 6-minute walking test (6MWT) results were (323.28±29.99) and (303.18±31.39) meters, respectively; the percentage of predicted forced vital capacity (FVC%pred) was (88.09±7.63)% and (83.85±7.79)%, respectively; the percentage of predicted forced expiratory volume in one second (FEV1%pred) was (97.54±8.69)% and (93.28±8.95)%, respectively; the percentage of predicted peak expiratory flow (PEF%pred) was (84.31±8.14)% and (79.64±8.05)%, respectively. In the PedsQL^TM-SMA Module, the scores for daily activities were 1.97±0.17 and 1.85±0.37, motor function scores were 2.14±0.35 and 2.03±0.28, fatigue/pain scores were 2.06±0.23 and 1.97±0.28, social interaction scores were 1.83±0.38 and 1.72±0.51, treatment worry scores were 1.94±0.23 and 1.85±0.37, and emotional functioning scores were 2.11±0.40 and 1.97±0.28, respectively. Serum neurofilament light chain (NfL) levels were (16.58±2.09) and (17.57±2.14) pg·mL^-1, and serum creatine kinase (CK) levels were (59.72±6.81) and (61.73±7.33) U·L^-1, respectively. Statistically significant differences were observed in the above indicators between the two groups (P<0.05). The overall incidence of adverse drug reactions in the experimental group and control group was 8.33% and 10.26%, respectively, with no statistically significant difference (P=0.775).

Intrathecal nusinersen combined with risdiplam significantly improves motor function in children with SMA compared with nusinersen alone, without increasing adverse drug reactions.

To explore the clinical efficacy of recombinant human brain natriuretic peptide (rhBNP) combined with amiodarone on patients with elderly heart failure (HF) complicated with ventricular arrhythmia (VA) and its impact on heart rate variability (HRV).

From January 2023 to January 2025, 220 patients with patients with elderly HF complicated with VA admitted to our hospital were prospectively selected and randomly separated into a control group (amiodarone) and an experimental group (rhBNP+amiodarone), with 110 patients in each group. The clinical outcomes, cardiac function, HRV indicators, serum N-terminal pro brain natriuretic peptide (NT-proBNP), QT interval dispersion (QTd), and adverse events were compared.

After treatment, the total effective rate in the experimental group was significantly higher than that in the control group (P<0.05). The experimental group had lower left ventricular end systolic diameter (LVESD), left ventricular end diastolic diameter (LVEDD), NT-proBNP, and QTd than the control group (P<0.05), and higher left ventricular ejection fraction (LVEF), cardiac output (CO), RMSSD, SDNN, and PNN50 than the control group (P<0.05).

The combination of rhBNP and amiodarone has a conspicuous effect on patients with elderly HF complicated with VA. It can optimize cardiac function and HRV, improve electrophysiological stability, and has good safety.

This study aimed to reassess the antibiotic treatment regimen for invasive community-associated methicillin-resistant Staphylococcus aureus(CA-MRSA) infections at a tertiary children’s hospital.

A retrospective analysis was conducted on cases of invasive clindamycin-resistant MRSA infections in children without underlying diseases between 2021 and 2025. A total of 34 children met the inclusion criteria, with the most common types of diagnosis being osteomyelitis (17 cases) and deep abscess (7 cases).

The median duration of intravenous (IV) antibiotic therapy was 11.5 days (interquartile range [IQR], 6-42 days), and the total treatment duration (IV + oral) was 32 days (IQR, 23-42 days). Overall, 50% of the children were subsequently switched to oral antibiotic therapy. The definitive antibiotics used for treatment included vancomycin (15 cases), TMP-SMX (trimethoprim-sulfamethoxazole, 9 cases), linezolid (7 cases), ceftaroline (2 cases), and doxycycline (1 case). The cure rates of different definitive antibiotic treatment regimens were similar: 73.3% in the vancomycin group, 88.9% in the TMP-SMX group, 50% in the ceftaroline group, and 100% in both the linezolid group and the doxycycline group. Three children died from MRSA infection, among whom 2 were treated with vancomycin and 1 with ceftaroline.

Vancomycin is the most commonly used drug for the treatment of invasive clindamycin-resistant CA-MRSA infections in children. However, in specific cases, TMP-SMX and linezolid may be considered as oral alternative options during the completion phase of treatment. Further research is still needed in the future to optimize the treatment regimen for such infections.

To explore the effect of the Mst1/Nrf2 pathway on myocardial autophagy function in type 2 diabetic mice, and to verify whether Mst1 gene knockout and Nrf2 activation can improve diabetic cardiomyopathy (DCM) by regulating autophagy.

The experimental animals were randomly divided into the following 4 groups, with 8 animals in each group: Control group (6 C57BL/6N mice, standard diet), DCM group (6 C57BL/6N mice), DCM+Mst1^-/- group (6 Mst1 gene knockout mice), high-sugar and high-fat diet, DCM+Mst1^-/-+Nrf2 group (6 diabetic mice with Mst1 gene knockout intervened by Tert-butylhydroquinone(tBHQ)(50 mg/kg/day)(PHR3512, Sigma-Aldrich). Cardiac function was detected by blood pressure, and autophagy was analyzed by Western blot.

The 4w systolic blood pressures of the control group, the high glucose control group, the high glucose Mst1 knockout group, and the high glucose Mst1^-/-±Nrf2 group were (103.6±1.43), (117.23±9.80), (111±7.43) and (109.21±3.22) mmHg, respectively, and the 8w systolic blood pressures were (99.09±1.46), (135.78±5.98), (123.57±8.90) and (119.97±6.97) mmHg, respectively. the 12w systolic blood pressures were (106.67±2.32), (138.90±2.00), (126.68±2.98) and (120.63±3.12) mmHg, respectively. The diastolic blood pressures at 4w were (79.50±1.12), (99.24±3.89), (85.03±1.89) and (82.90±3.22) mmHg, respectively. The diastolic blood pressures at 8w were (76.35±2.12), (106.78±5.98), (94.80±3.90) and (88.90±2.90) mmHg, respectively. The diastolic blood pressure at 12w were (78.79±1.70), (101.32±2.13), (93.62±3.98) and (89.02±1.92) mmHg, respectively. The 4-week mean arterial pressures were (87.51±0.41), (117.23±9.80), (85.56±1.46) and (83.91±2.23) mmHg, respectively. The 8-week mean arterial pressures were (84.24±2.41), (116.46±5.68), (104.80±3.91) and (98.90±2.90) mmHg, respectively. the 12-week mean arterial pressures were (86.09±1.74), (101.32±2.13), (103.60±3.37) and (99.02±1.55) mmHg, respectively. Compared with the mice in the Control group, the systolic blood pressure, diastolic blood pressure and mean arterial pressure of the mice in the DCM group, DCM+Mst1^-/- group and DCM+Mst1^-/-+Nrf2 group were significantly increased (P<0.000 01).

Improved cardiac diastolic function in mice with Mst1/Nrf2 activation. This effect is achieved by reducing the expression of p62 protein, further emphasizing that the Mst1/Nrf2 axis exerts its protective function by regulating autophagy-related proteins.

To investigate the effect of wogonin on hypoxia/reoxygenation (H/R)-induced neuronal injury based on the C-C chemokine ligand 2-C-C chemokine receptor 2 (CCL2-CCR2) pathway.

PC12 cells were cultured and divided into five groups, control group (normal culture), model group (H/R injury), experimental group (H/R+50 μmol·L^-1 wogonin pretreatment for 1 h), antagonist group (H/R+10 μmol·L^-1 CCR2 antagonist RS102895 pretreatment for 1 h), and agonist group (H/R+50 μmol·L^-1 wogonin+20 μmol·L^-1 CCL2 recombinant protein pretreatment for 1 h). Cell viability and cytotoxicity were assessed using the cell counting kit-8 (CCK-8) and lactate dehydrogenase (LDH) assay kits. Apoptosis was detected by Annexin V-fluorescein isothiocyanate/propidium iodide (Annexin V-FITC/PI) double staining and terminal deoxynucleotidyl transferase-mediated dUTP nick-end labeling (TUNEL) staining. Oxidative stress and inflammatory responses were evaluated by enzyme-linked immunosorbent assay (ELISA) and quantitative real-time polymerase chain reaction (RT-qPCR). Protein expression levels related to the CCL2-CCR2 signaling pathway were measured by Western blot.

The viability of PC12 cells in control group, model group, experimental group, antagonist group and agonist group were 0.89±0.06, 0.32±0.04, 0.69±0.05, 0.65±0.07 and 0.46±0.04, respectively; the SOD activities were (45.11±3.02), (10.05±1.05), (33.62±3.15), (30.15±2.87) and (21.05±2.17) U·mL^-1, respectively; the Annexin V FITC/PI double staining apoptosis rates were (3.21±0.68)%, (29.65±1.59)%, (8.99±0.82)%, (9.87±0.85)% and (18.65±1.06)%, respectively; the TUNEL positive rates were (5.66±0.89)%, (33.65±2.42)%, (13.25±1.05)%, (11.66±1.87)% and (24.06±1.16)%, respectively; the relative expression levels of tumor necrosis factor-alpha(TNF-α) mRNA were 1.02±0.15, 3.05±0.32, 1.59±0.16, 2.98±0.24 and 2.66±0.20, respectively; the relative expression levels of interleukin-6 (IL-6) mRNA were 1.05±0.12, 4.54±0.39, 1.87±0.15, 1.75±0.14 and 3.08±0.32, respectively; the relative expression levels of CCL2 mRNA were 1.01±0.07, 3.99±0.32, 1.64±0.15, 3.90±0.33 and 2.84±0.23, respectively; the relative expression levels of CCR2 mRNA were 1.03±0.10, 5.21±0.51, 2.03±0.26, 2.11±0.20 and 3.85±0.32, respectively; the relative expression levels of phosphorylation (p)p65/p65 were 0.12±0.02, 0.75±0.06, 0.24±0.03, 0.22±0.01 and 0.52±0.05, respectively; the leakage rates of LDH were (18.32±1.15)%, (58.64±5.16)%, (29.02±2.16)%, (26.98±3.02)% and (40.24±3.54)%, respectively; the malondialdehyde (MDA) contents were (50.16±5.24), (169.65±10.16), (91.32±7.12), (86.21±5.16) and (134.16±11.87) nmol·mL^-1, respectively. The differences of above indicators between model group and control group, between experimental group and model group, and between the antagonist, agonist group and experimental group were all statistically significant (all P<0.05).

Wogonin exerts a protective effect against H/R-induced neuronal injury by inhibiting the CCL2-CCR2 signaling pathway.

To establish a high-efficiency and sensitive liquid chromatography-tandem mass spectrometry (LC-MS/MS) method for the quantitative analysis of cyclosporine A in human whole blood.

Protein precipitation with methanol was employed for sample pretreatment, and cyclosporine A-d₄ was used as the internal standard. Chromatographic separation was performed on a Kinetex C₁₈ column (3.0 mm×50.0 mm, 2.6 μm, 100 Å) with a mobile phase consisting of 0.1% formic acid in water (phase A) and 0.1% formic acid in methanol (phase B) at a flow rate of 0.55 mL·min^-1. The column temperature was maintained at 40 ℃. Quantitative detection was carried out using electrospray ionization in positive ion mode and multiple reaction monitoring (MRM). The method was validated for specificity, calibration curve and lower limit of quantitation (LLOQ), accuracy and precision, matrix effect, and sample stability.

The retention time of cyclosporine A was 1.19 min, with a total run time of 2.0 min per sample. A good linear relationship was observed in the concentration range of 10-1 000 ng·mL^-1 (r=0.998 5), and the LLOQ was 10.0 ng·mL^-1. The recovery rates ranged from 99.53% to 102.83%, with intra-batch precision ≤3.58% and inter-batch precision ≤3.24%. The sample stability was satisfactory, and the matrix effect was negligible.

The established method features short analysis time, high sensitivity, and low sample volume requirement, making it suitable for therapeutic drug monitoring and accurate quantification of cyclosporine A in human whole blood samples.

Explore the correlation between tacrolimus trough concentration (C₀) and clinical efficacy in children with lupus nephritis (LN), and identify key factors affecting the efficacy of tacrolimus treatment in LN patients..

A total of 120 children with LN who took tacrolimus in our hospital from March 2021 to December 2024 and were monitored for blood drug concentration were retrospectively selected. According to the treatment effect, they were divided into effective group (n=78) and ineffective group (n=42). The basic information, laboratory indicators and other data of the children were collected to explore the influencing factors of the efficacy of the children.

The daily dose of tacrolimus, trough concentration C₀, 24 h urinary protein and albumin in the effective group were higher than those in the ineffective group (all P<0.001), while the glomerular filtration rate was significantly lower than that in the ineffective group (P<0.001). After collinearity diagnosis, the indexes were included in Logistic regression analysis. The daily dose of tacrolimus, trough concentration C₀, glomerular filtration rate and albumin were all protective factors affecting the efficacy of children with LN (P<0.05), while 24 h urine protein quantification was a risk factor (P<0.05). The receiver operating characteristic curve was drawn. The AUC of glomerular filtration rate, 24-hour urinary protein quantification, and albumin in predicting the efficacy of children with LN was 0.882 [95% CI: 0.809-0.956], 0.861 [ 95 % CI: 0.790-0.932], 0.897 [95% CI: 0.837-0.956]. The AUC and optimal cut-off value of tacrolimus daily dose and trough concentration C₀ in predicting the efficacy of children with LN were 0.750 [95% CI : 0.649-0.851 ], 1.45 mg and 0.772 [ 95 % CI: 0.691-0.853 ], 4.05 ng·mL^-1.

The trough concentration of tacrolimus (C₀) is significantly correlated with the clinical efficacy of LN in children, and the daily dose and trough concentration C₀ are key factors affecting the efficacy.

To establish a population pharmacokinetic (PPK) model of duloxetine (DLXT) in hospitalized patients with depression, to investigate its influencing factors, and to provide reference for individualized medication.

A total of 325 patients who were hospitalized and received oral DLXT treatment at Shenzhen Kangning Hospital from January 2022 to October 2025 were retrospectively included, and their medication information, plasma concentrations, and other clinically relevant data were collected. Nonlinear mixed-effects modeling (NONMEM) was used to establish a PPK model of DLXT concentrations. Goodness-of-fit (GOF) plots, visual predictive check (VPC) and Bootstrap were used to evaluate the stability and predictive performance of the model, respectively. Based on the significant covariates identified in the final model, the steady-state plasma concentrations (C_ss) of DLXT over time were simulated under different dosage regimens in various populations.

A one-compartment model with first-order absorption and elimination was developed using 450 plasma concentration data points from 325 inpatients. The inter-individual variability of apparent clearance (CL/F) was 42.2%, and the proportional residual variability was 27.1%. Gender was identified as a significant covariate affecting CL/F. The final model was expressed as: CL/F(L·h^-1)=64.6×(1-0.25×Gender), where Gender is an indicator variable (male=0, female=1). The apparent volume of distribution was 1 530 L, and the absorption rate constant was fixed at 0.168 h^-1. Monte Carlo simulations showed that the C_ss in females was higher than that in males at the same doses.

A PPK model for DLXT in hospitalized patients with depression was successfully established. The final model is stable and reliable, and it can be used for individualized dosing regimens in clinical practice.

Against the backdrop of the comprehensive promotion of the policy of centralized bulk procurement of drugs (referred to as "centralized procurement"), this study aims to systematically review and integrate relevant evidence on the effectiveness, safety, and cost-effectiveness of cephalosporin injectables under centralized procurement as presented in real-world studies, and compare their clinical performance with that of original research drugs/imported generic drugs. The goal is to provide an evidence-based basis for the rational use, clinical evaluation, and policy optimization of selected generic drugs in centralized procurement.

Retrieval of real-world research literature on centralized collection cephalosporin injections published from April 2021 to February 2026, comprehensive analysis was conducted from the dimensions of clinical efficacy, safety, and economy, and QATSM-RWS was used to evaluate the quality of the included studies.

After screening, a total of 17 studies were included. The investigated medications primarily comprised cefuroxime, ceftriaxone, and cefoperazone-sulbactam sodium, covering diverse infectious populations in both adults and children, as well as two key clinical scenarios: perioperative infection prevention and therapeutic anti-infective treatment. The results demonstrated that, in terms of efficacy, most studies indicated comparable overall effectiveness between centrally procured cephalosporin injections and original/imported generic drugs for perioperative infection prevention and therapeutic anti-infective therapy, with only individual studies showing differences in secondary endpoints. Regarding safety, no significant differences were observed between the two groups for perioperative prophylaxis; in therapeutic applications, most studies confirmed equivalent safety profiles across both groups, with only certain safety indicators differing in specific studies. In terms of economy, centrally procured medications significantly reduced treatment-related expenses, demonstrating notable economic advantages.

Overall, centrally procured cephalosporin injections are basically consistent with original drugs/imported generic drugs in terms of clinical effectiveness and safety, while also offering superior cost-effectiveness, which can reduce patients’ medical burden. Limited by factors such as the limited coverage of included research varieties, mostly single-center studies, and small sample sizes, relevant research evidence still needs to be improved. Large-sample, multi-center clinical studies need to be conducted in the future to further consolidate the evidence-based basis.

To analyze the characteristics of adverse drug reactions (adverse drug reaction, ADRs) of oral riluzole in patients with amyotrophic lateral sclerosis (amyotrophic lateral sclerosis, ALS), and to provide references for clinical safe medication.

Data of ALS patients treated with riluzole at Peking Union Medical College Hospital from May 2018 to August 2023 were collected. Patients with ADRs were retrospectively analyzed regarding demographic information, medication details, clinical manifestations, time of onset, management, and outcomes. Causality assessment was performed according to China’s Measures for Reporting and Monitoring of Adverse Drug Reactions, severity was evaluated using CTCAE V5.0, and ADRs were coded using MedDRA terms.

A total of 67 ALS patients were included, among whom 22 experienced ADRs (32.8%). The incidence of ADRs was higher in females (44.0%) than in males (26.2%). A total of 54 ADR episodes were recorded. The most common ADRs involved the nervous system (20 cases/54 cases, 37.0%), followed by gastrointestinal system disorders (13 cases/54 cases, 24.1%), general disorders and administration site conditions (7 cases/54 cases, 13.0%), and various laboratory investigations (6 cases/54 cases, 11.1%). One patient (1.5%) experienced a grade 3 serious ADR (interstitial lung disease). Seventeen patients (77.3%) developed ADRs within the first three months of treatment. Sixteen patients (72.7%) tolerated the ADRs without specific intervention, while six (27.3%) improved after drug discontinuation, dose reduction, or symptomatic treatment.

The most common ADRs of riluzole in ALS patients involve the nervous system, gastrointestinal tract, and elevated liver enzymes (all not higher than twice the normal value), mostly occurring early in the treatment course. Clinicians should remain vigilant for respiratory ADRs and potential heightened inflammatory responses, with close monitoring recommended during the first six months of treatment.

To investigate the current status of safety review by institutional review boards (IRBs) for clinical drug trials in China, analyze existing issues and needs for improvement, and provide a basis for enhancing the quality and efficiency of ethical review.

An online questionnaire survey was conducted from May 2025 to March 2026, targeting 391 IRBs for clinical drug trials across 29 provinces, municipalities, and autonomous regions nationwide. The questionnaire covered the basic profile of the ethics committees, requirements for submitting safety reports and review processes, as well as attitudes toward the need for improvements in the review process. SPSS AU was used to perform descriptive statistics, analysis of variance (ANOVA), correlation analysis, and post-hoc analysis. The results showed that the questionnaire had good reliability and validity (Cronbach’s α=0.932, KMO=0.946).

86.7% of the drug clinical trial ethics committees explicitly stipulated in their SOPs the selection criteria and decision-makers for the review method of safety reports; however, implementation was inconsistent at critical stages, with a portion (46.04%) of the committees not having the chairperson decide on the selection of the lead reviewer. Regarding serious adverse events (SAEs), 94.63% of drug clinical trial ethics committees required the principal investigator to submit reports; however, there were discrepancies in the interpretation of the provisions of the "Good Clinical Practice for Drug Clinical Trials" (2020) (48.85% believed that only suspected and unexpected adverse reactions needed to be reviewed). 59.34% of ethics committees updated the types of review conclusions in a timely manner to be consistent with the "Measures for Ethical Review of Life Sciences and Medical Research Involving Humans" (2023). The results showed that there was no significant difference in the recognition of all improvement suggestions among the levels of different health institutions, investigators’ working years, professional backgrounds and technical titles, proving that the industry has a common and consistent demand for improving ethical standardization review. Correlation analysis shows that various improvement measures are closely related. Among them, the "Standard Operating Procedures for the Establishment and Review of Ethics Committees" and the "Application Guidelines for Institutional Establishment" showed a strong positive correlation (r=0.716, P<0.01), and various measures were positively correlated with "supervision by superior authorities" (r=0.407~0.705, P<0.01), highlighting the need for systematic optimization.

Our country’s ethical review system for drug clinical trial safety reports has been initially established, but it needs to be strengthened in terms of uniformity of review standards, timely implementation of regulations and standardization of process operations. This survey results show that in the future, efforts should be made to build a four-in-one systematic optimization plan of "regulations-standards-capabilities-supervision" to unify industry standards, improve review efficiency, and effectively protect the rights and interests of research participants.

To analyze the characteristics of patient subgroups and temporal patterns of adverse events (AE) associated with efgartigimod treatment for generalized myasthenia gravis (gMG), based on the U.S. FDA Adverse Event Reporting System (FAERS) database, providing evidence for personalized clinical use and long-term drug safety monitoring.

Individual case safety reports (ICSRs) in which efgartigimod was the primary suspect drug were extracted from the FAERS database covering Q1 2020 to Q4 2023. After deduplication and MedDRA standardization, binary logistic regression was used to assess the impact of age, sex, and body weight on symptom recurrence risk; K-means clustering identified high-risk patient subgroups; and descriptive analysis characterized the distribution of AE onset times (TTO).

A total of 788 ICSRs were included, of which 321 had complete time information. Logistic regression revealed that low body weight (OR=0.97, P=0.021) and younger age (OR=0.96, P=0.014) were risk factors for symptom recurrence. Clustering analysis divided patients into three groups: older and heavier individuals (mainly cardiovascular or procedure related AEs), younger and lighter individuals (primarily immune or hypersensitivity related AEs), and a heterogeneous group. The overall TTO distribution of AEs was right-skewed, with 68% occurring within 30 days. Median TTO for serious AEs (73 days) was longer than for non-serious AEs (54 days), indicating a clear delayed risk (>180 days).

Efgartigimod related AE risks exhibit heterogeneity across populations and time dependency. Younger, lower weight patients require close monitoring for immune related AEs, while older, higher weight patients should be monitored for cardiovascular risks. A comprehensive, long-term monitoring strategy throughout the treatment cycle is warranted clinically. The integrated framework proposed here "risk stratification-subgroup identification-temporal monitoring" can be directly applied to other neonatal Fc receptor (FcRn) targeted drugs, offering an innovative model for post-marketing evaluation of biologics in China.

To explore the key elements in the evaluation and selection of sites for multicenter clinical trials, providing references for standardizing clinical trial site selection practices.

By integrating the practical experience of multiple sponsors, contract research organizations (CROs), and research sites, a standardized framework for site evaluation and selection was constructed.

A three-phase standardized process was proposed, encompassing pre-assessment preparation, formal evaluation, and outcome assessment. This study systematically categorized common and specific evaluation criteria, including 5 core dimensions and 17 key indicators covering site and personnel qualifications, hardware conditions, patient resources and quality systems, management processes, and special considerations.

This study constructed a comprehensive site evaluation system encompassing standardized procedures, structured dimensions, and core indicators. It achieves a transformation in evaluation practices from an experience-driven approach to a framework-guided methodology, providing an operable tool for the scientific selection of sites in multicenter clinical trials.

To analyze the current status and development trends of registered clinical trials of inhalation preparations in China, and to provide references for drug development and clinical evaluation.

Clinical trials of inhalation preparations registered on the NMPA Platform for Drug Clinical Trial Registration and Information Publicity from its inception to December 31, 2025 were retrieved. Descriptive statistical analysis was conducted on the basic information, design types, drug characteristics, and indications of the trials.

A total of 445 registered clinical trials of inhalation preparations were included, which had entered a rapid development phase in the past five years. The trials were predominantly domestic (94.61%) and bioequivalence (BE) studies (48.31%). The most common dosage forms were liquid inhalation formulations (44.04%) and dry powder inhalations (35.51%). Research and development were highly concentrated on asthma and chronic obstructive pulmonary disease, with inhaled corticosteroids and their combinations being the main directions. In contrast, trials involving childer and adolescents accounted for only 4.94%, and explorations in areas such as pulmonary fibrosis and pulmonary infections remained relatively limited. Sponsors and research resources were primarily concentrated in the more developed pharmaceutical regions such as Beijing, Shanghai, and Jiangsu.

The development of inhalation preparations in China has entered a phase of rapid growth, characterized by a dominance of generic drugs, a high concentration of therapeutic areas, and differentiated development of dosage forms. Future efforts should focus on unmet clinical needs and innovative research and development of high-technical-barrier dosage forms to promote high-quality development in this field.