To synthesize resveratrol analogues and investigate their anti-diabetic activity and related mechanism.

Using different substituted anilines and different substituted benzaldehydes as raw materials, resveratrol analogs were synthesized under ethanol reflux, and their protective effects on pancreatic β cells were tested under the stimulation of palmitic acid (PA). The anti-diabetic mechanism of the dominant compounds was explored.

Most of the compounds have protective effects on pancreatic β cells, and the compounds JA10 and JA12 have significant cytoprotective effects. Molecular mechanism studies showed that the compounds JA10 and JA12 reduced the generation of reactive oxygen, and inhibited the gene expression of inflammatory factors IL-1β and IL-6.

The resveratrol analogs JA10 and JA12 are potential antidiabetic new chemical entities.

To investigate the therapeutic effect and safety of the extract of Qingxin Lianzi Decoction (QLD) on nephritis in rats.

The antibacterial activity of QLD extract against Staphylococcus aureus, Escherichia coli and Pseudomonas aeruginosa in vitro was determined by disk diffusion and double dilution methods. Pyelonephritis model induced by bacterial, adriamycin-induced glomerulonephritis model and IgA nephropathy model in rats were used to evaluate the therapeutic effect on nephritis in rats. The safety of QLD extract was preliminarily evaluated using the acute toxicity test in mice.

QLD extract had no obvious antibacterial activity against the three pathogenic bacteria in vitro, but it significantly reduced the contents of creatinine and urea nitrogen in serum, decreased the levels of urinary occult blood and urine protein in pyelonephritis rats. At the same time, the 8 g·kg^-1 dose of QLD significantly reduced the contents of creatinine and urea nitrogen in serum, but had no significant effect on the kidney index and urine protein in glomerulonephritis rats. Moreover, the 8 g·kg^-1 dose of QLD significantly reduced the level of urine protein in IgA nephropathy rats, but had no significant effect on creatinine, urea nitrogen and kidney index. The results of acute toxicity showed that the maximum dose of QLD extract is 48 g·kg^-1, and no obvious toxicity and death were observed.

QLD extract has therapeutic effects on three experimental nephritis in rats.

The present study explored the active ingredients and potential mechanism of Wuwei Ganlu yaoyu decoction powder in the treatment of gouty arthritis(GA).

The main chemical components of Wuwei Ganlu yaoyu decoction powder were identified by UPLC-Q-Exactive-Orbitrap-MS. The target compounds were screened from the identified compounds by Swiss Target database. The databases genecards, OMIM and others were used to screen disease targets, STRING to build PPI network and select key targets, DAVID to enrich GO and KEGG pathways of common targets of drug diseases. Based on these, we discussed the mechanism of Wuwei Ganlu yaoyu decoction powder in the treatment of GA. In addition, the "drug-component-target-disease" network was constructed by using Cytoscape 3.9.1 software, and Maestro software was used to conduct molecular docking between components and core targets to screen effective components.

A total of 52 compounds were identified, including 24 flavonoids, 5 alkaloids, 10 organic acids, 5 phenols, 5 phenylpropanoids, 2 terpenoids and 1 alkane. According to the analysis of network pharmacology, there were 520 component targets and 141 intersection targets of Wuwei Ganlu yaoyu decoction powder, and 5 core targets were screened (SRC, STST3, PIK3CA, MAPK1, HSP90AA1). Molecular docking showed that six components such as Luteolin had good binding ability with five core targets.

The active components of Wuwei Ganlu yaoyu decoction powder in the treatment of GA are luteolin, hyperoxin-7-O-β-D-glucopyranoside, Hyprolatine-7-O-β-D-xylopyranoside, quercetin, naringin and luteolin, which may be related to the action on targets like SRC, STAT3, PIK3CA and others to regulate the signal pathways of PI3K Akt, Ras, MAPK, etc.

To evaluate the impact of budget on the total health care costs of using ferric carboxymaltose to correct iron deficiency anaemia compared with usual care in perioperative anaemia management in general surgery, and to provide a decision-making basis to promote the implementation of patient blood management (PBM) in China, particularly anaemia management focused on rapid and adequate iron supplementation in the perioperative period.

Based on Chinese epidemiological data, the differences in the reduction of allogeneic blood transfusion rate and length of stay in the perioperative period between the PBM group and the conventional treatment group were compared, and an analysis model of impact on budget was constructed combined with domestic cost data. Clinical outcomes were from data of foreign randomized clinical trials, and cost data were from public prices in the domestic market, including iron drug (oral iron, ferric carboxymaltose), injection, EPO use, blood transfusion, and hospitalization.

Based on 3.758 million elective general surgery patients nationwide, the use of ferric carboxymaltose for anaemia management in the perioperative period as a complete substitute for routine care would have prevented 704,690 blood transfusions and saved a total of ￥139 million in treatment costs, with an average saving of ￥42 per patient. A sensitivity analysis showed total treatment cost savings of ￥1.086 billion, ￥2.033 billion, ￥2.981 billion and ￥3.928 billion assuming a 10%, 20%, 30% and 40% reduction in the price of ferric carboxymaltose, with average cost savings per patient being ￥289, ￥541, ￥793 and ￥1 045, respectively.

It is recommended that the patients with clinical perioperative iron deficiency anaemia undergo implementation of patient blood management and that ferric carboxymaltose be used for anaemia management.

To establish a scientific applicability evaluation index system of real-world data (RWD), and provide a quantitative analysis basis for the transformation of RWD into real-world evidence (RWE).

Hierarchical extraction analysis of factors affecting the applicability of RWD was carried out by studying domestic and international guides, documents and literature. Analytic hierarchy process (AHP) and group eigenvalue method (GEM) were used in combination to establish the hierarchical structures of the selected indicators, expert scoring matrix was constructed, and MATLAB software for index system was used to compute the weights.

Applicability evaluation index was constructed from two aspects: relevance and reliability. Relevance included five sub-criteria level indexes and 26 specific evaluation indicators: data source and sample, exposure/intervention and event outcome definition, integration of multi-source data, regulatory requirements, ethics. Reliability consisted of three sub-criteria level indexes and 27 specific evaluation indicators: data validity, quality control, and quality assurance.

When gathering and applying RWD, attention should be paid to the data source, validity, and quality control, to ensure that the data is closely related to the demand and is reliable, and then to upgrade the applicability from RWD to RWE.

To investigate the status of clinical trials registered on ClinicalTrials.gov on diseases in the list of rare diseases in China and analyze the registration characteristics.

All clinical trials involved in the list of rare diseases in China were searched on ClinicalTrials.gov from inception to April 5th, 2022. The relevant information of clinical trial registration was collected, and WPS Office was used for data analysis.

A total of 12 904 rare disease-related clinical trials were screened out, and the overall trend was increasing year by year. The information of clinical trial stages was missing a lot, and the sample size was mainly 0~100 cases. The types of trials were mainly interventional studies (9 042 trials, 70.1%) and observational studies (3 760 trials, 29.1%). There were three main statuses of trials: completed (6 467 trials, 50.1%), recruiting (2 388 trials, 18.5%), and unknown status (1 207 trials, 9.4%). The number of drug therapy trials was the largest (5 992 trials, 46.3%). The countries/regions participating in clinical trials were mainly developed countries in Europe and the United States. All of the top three single/multi-center rare disease-related clinical trials were conducted in European and American countries. There were a small percentage of clinical trials conducted in China (460 trials, 3.6%), but the proportion of single center-clinical trials was the highest (260 trials, 56.5%). The research institutions were mainly universities and medical institutions, and in China they were mainly medical institutions (358 trials, 77.8%). The sponsoring institutions were mainly European and American countries. The design of rare disease-related clinical trials was mainly non-randomized, open label and parallel allocation of therapeutic regimens. The outcome indicators were mainly related to safety and efficacy.

The development of clinical trials of rare diseases in China is on the rise, but there is a big gap with European and American countries. We should establish a systematic research and development incentive mechanism and introduce relevant laws and regulations as soon as possible to promote the high-quality development of clinical trials of rare diseases.

To develop a highly sensitive, efficient, and stable cell-based platform for detection of anti-adeno-associated virus 8 (AAV8) neutralizing antibody titer in monkey plasma and perform preliminary validation of the relevant performance of the method.

In vitro, an equal volume of diluted monkey plasma was incubated with AAV8-Luciferase and then the mixture was added to a 96-well plate lined with HEK293 cells and incubated overnight to infect HEK293 cells; after cell lysis, the cells were added to a firefly luciferase reporter gene assay and the fluorescence signal (RLU) was detected using ELISA to assess the plasma levels of anti-AAV8 neutralising antibodies and subsequently obtain the corresponding antibody titres. The assay's sensitivity (LOD) and titer reproducibility, precision, method robustness and sample stability were preliminary were preliminary validated.

A highly sensitive, efficient and stable method for the detection of anti-AAV8 neutralizing antibody titer in monkey plasma at the cellular level was developed and preliminarily validated. The LOD value of the method was 10.36 ng·mL^-1. The repeatability, precision and method robustness of the titer assay were good, and the positive control samples were stable after being placed at room temperature and 2 ℃~8 ℃ for 24 h and after freeze-thawing for 6 times at -65 ℃~-90 ℃/room temperature.

It has been preliminarily validated that the method can be used for the analysis and detection of anti-AAV8 neutralizing antibody titer in monkey plasma, which is useful for grasping the process of drug treatment and adjusting the treatment protocol in time and is suggestive for the development of AAV8 serotype gene therapy drugs and individualized treatment.