Progressive pulmonary fibrosis (PPF) is a progressive and lethal condition with few effective treatment options. Improvements in quality of life for patients with PPF remain limited even while receiving treatment with approved antifibrotic drugs. Traditional Chinese medicine (TCM) has the potential to improve cough, dyspnea and fatigue symptoms of patients with PPF. TCM treatments are typically diverse and individualized, requiring urgent development of efficient and precise design strategies to identify effective treatment options. We designed an innovative Bayesian adaptive two-stage trial, hoping to provide new ideas for the rapid evaluation of the effectiveness of TCM in PPF. An open-label, two-stage, adaptive Bayesian randomized controlled trial will be conducted in China. Based on Bayesian methods, the trial will employ response-adaptive randomization to allocate patients to study groups based on data collected over the course of the trial. The adaptive Bayesian trial design will employ a Bayesian hierarchical model with "stopping" and "continuation" criteria once a predetermined posterior probability of superiority or futility and a decision threshold are reached. The trial can be implemented more efficiently by sharing the master protocol and organizational management mechanisms of the sub-trial we have implemented. The primary patient-reported outcome is a change in the Leicester Cough Questionnaire score, reflecting an improvement in cough-specific quality of life. The adaptive Bayesian trial design may be a promising method to facilitate the rapid clinical evaluation of TCM effectiveness for PPF, and will provide an example for how to evaluate TCM effectiveness in rare and refractory diseases. However, due to the complexity of the trial implementation, sufficient simulation analysis by professional statistical analysts is required to construct a Bayesian response-adaptive randomization procedure for timely response. Moreover, detailed standard operating procedures need to be developed to ensure the feasibility of the trial implementation. Please cite this article as: Zhang C, Nie YS, Zhang CT, Yang HJ, Zhang HR, Xiao W, Cui GF, Li J, Li SJ, Huang QS, Yan SY. An adaptive Bayesian randomized controlled trial of traditional Chinese medicine in progressive pulmonary fibrosis: Rationale and study design. J Integr Med. 2025; 23(2): 138-145.
Female ; Humans ; Male ; Bayes Theorem ; Disease Progression ; Drugs, Chinese Herbal/therapeutic use* ; Medicine, Chinese Traditional/methods* ; Pulmonary Fibrosis/therapy* ; Quality of Life ; Randomized Controlled Trials as Topic ; Research Design ; Adaptive Clinical Trials as Topic

Relevant literature on TCM diagnostic and treatment equipment from January 1994 to May 2023 was collected with three Chinese databases,namely,China National Knowledge Infrastructure,Wanfang and Wipu.CiteSpace software was used for the analyses of the trend of annual publication volume,co-occurrence of publication institutions,co-occurrence of keywords,cluster and burst and the generation of corresponding knowledge graphs.It's pointed out TCM diagnostic and treatment equipment had problems in low publication volume and collaboration between research institutions,hotspots of Chinese medicine diagnosis and treatment,diagnosis and treatment equipment,diagnosis device,Chinese medicine diagnosis,diagnosis and treatment technology and traditional Chinese medicine,and research frontiers of artificial intelligence,medical alliance,curriculum design and innovation and entrepreneurship.References were provided for relevant research in China.[Chinese Medical Equipment Journal,2024,45(3):76-80]

Aim To analyze the genotype-phenotype characteristics of voltage-gated potassium channels (Kv) associated genetic epilepsy and evaluate the efficacy of anti-seizure medications(ASMs). Methods PubMed database was searched and patients meeting the inclusion criteria were included for analysis. We divided the patients into “benign”, “encephalopathic” and other phenotypes according to the clinical characteristics. We performed descriptive statistical analysis of patients' mutated genes, clinical phenotype and drug efficacy, and used logistic regression to explore the influencing factors of treatment outcome. Results Data of 474 children were included for analysis. There were significant differences among different phenotypes in mutated genes, source of mutations and so on. In terms of clinical characteristics, there were also significant differences between patients with different phenotypes in age of onset, combined developmental delay and so on. In terms of monotherapy, phenobarbital was the most common treatment choice for children with “benign” phenotype, and sodium channel blockers (SCBs) were the most common treatment choice for children with “encephalopathy” phenotype, and the efficacy of SCBs monotherapy was superior to that of other ASMs. Multivariate Logistic analysis of the children receiving monotherapy showed that whether the children were combined with developmental delay and whether SCBs were used were significant factors influencing the efficacy of drug therapy. Conclusions Patients with the “benign” and “encephalopathic” phenotypes differ in several aspects of genetic variation, clinical characteristics, and drug selection. These results suggest that SCBs may be one of the recommended options for monotherapy.

A case series report was conducted to analyze the cases of children with multiple sclerosis(MS)who received fingolimod for the first time.Four Chinese children with MS were reported.A 9-year-old girl weighted 50 kg with low basal heart rate developed asymptomatic bradycardia.The other 3 children did not experience cardiac adverse reactions.Cardiac safety of the first dose of fingolimod in children with MS was also evaluated.We suggest that special attention should be paid to patients with slow basal heart rate,young age and low body weight.In order to promote its safe use at initiation,the monitoring time can be extended appropriately and night monitoring can be strengthened.

Ultra-performance liquid chromatography-quadrupole-electrostatic field Orbitrap mass spectrometry(UHPLC-Q-Exactive Orbitrap MS/MS) was used for rapid identification of the chemical components in Kaixin San substance benchmark. The gradient elution was performed through a Waters ACQUITY~(TM) BEH C_(18) column(2.1 mm×150 mm, 1.7 μm) with water-acetonitrile as mobile phase, a column temperature of 30 ℃, a flow rate of 0.3 mL·min~(-1), and a sample size of 1 μL. The scanning was performed in the negative ion mode. The complex component groups in Kaixin San substance benchmark were quickly and accurately identified and clearly assigned based on the comparison of the retention time and MS data with those of the reference substance as well as the relative molecular weight of the same or similar components in the mass spectrum database and literature. A total of 77 compounds were identified, including 26 saponins, 13 triterpenoid acids, 20 oligosaccharide esters, 5 xanthones, and 13 other compounds. The qualitative method established in this study can systematically, accurately, and quickly identify the chemical components in Kaixin San substance benchmark, which can provide a basis for the further analysis of its active components in vivo and the establishment of its quality control system.
Benchmarking ; Chromatography, High Pressure Liquid/methods* ; Drugs, Chinese Herbal/chemistry* ; Tandem Mass Spectrometry/methods*

Objective: To investigate the detection types and aggregation of high-risk population of cardiovascular disease (CVD) in Jiangsu province and the related influencing factors to provide reference for the prevention and control of cardiovascular disease. Methods: A total of 120 211 participants were included in the investigation. Information was collected by questionnaire based survey, physical examination and biochemical tests. χ² test and multivariate logistic regression were used for statistical analysis. Results: The detection rate of CVD high risk was 25.03%. The detection rates were 19.01%, 4.85%, 3.18% and 5.31% for hypertension, dyslipidemia, cardiovascular history and WHO assessed risk ≥20% types, respectively. Male, rural, old age, low education level, low family income, drinking, waist circumference exceeding standard, overweight and obesity were risk factors of CVD (all P<0.01). The composition ratios of aggregation of 1, 2 and ≥3 high risk types of CVD were 74.01%, 22.91% and 3.08%, respectively. With the increase of aggregation types, the correlation strength increased with age, rural residents, education level and annual family income. Conclusion: Targeted measures should be carried out according to different influencing factors for the prevention and control of CVD in Jiangsu province in order to achieve the maximum prevention and control effect with the minimum cost.
Cardiovascular Diseases/epidemiology* ; China/epidemiology* ; Humans ; Hypertension/epidemiology* ; Male ; Overweight ; Prevalence ; Risk Factors ; Waist Circumference

Objective: To explore the heterogeneity and correlation of clinical phenotypes and genotypes in children with disorders of sex development (DSD). Methods: A retrospective study of 1 235 patients with clinically proposed DSD in 36 pediatric medical institutions across the country from January 2017 to May 2021. After capturing 277 DSD-related candidate genes, second-generation sequencing was performed to analyzed the heterogeneity and correlation combined with clinical phenotypes. Results: Among 1 235 children with clinically proposed DSD, 980 were males and 255 were females of social gender at the time of initial diagnosis with the age ranged from 1 day of age to 17.92 years. A total of 443 children with pathogenic variants were detected through molecular genetic studies, with a positive detection rate of 35.9%. The most common clinical phenotypes were micropenis (455 cases), hypospadias (321 cases), and cryptorchidism (172 cases) and common mutations detected were in SRD5A2 gene (80 cases), AR gene (53 cases) and CYP21A2 gene (44 cases). Among them, the SRD5A2 mutation is the most common in children with simple micropenis and simple hypospadias, while the AMH mutation is the most common in children with simple cryptorchidism. Conclusions: The SRD5A2 mutation is the most common genetic variant in Chinese children with DSD, and micropenis, cryptorchidism, and hypospadias are the most common clinical phenotypes. Molecular diagnosis can provide clues about the biological basis of DSD, and can also guide clinicians to perform specific clinical examinations. Target sequence capture probes and next-generation sequencing technology can provide effective and economical genetic diagnosis for children with DSD.
3-Oxo-5-alpha-Steroid 4-Dehydrogenase/genetics* ; Child ; China/epidemiology* ; Cryptorchidism/genetics* ; Disorders of Sex Development/genetics* ; Female ; Genital Diseases, Male ; Genotype ; Humans ; Hypospadias/genetics* ; Male ; Membrane Proteins/genetics* ; Penis/abnormalities* ; Phenotype ; Retrospective Studies ; Steroid 21-Hydroxylase/genetics*

Idiopathic asthenozoospermia, a common factor in male infertility, is characterized by altered sperm motility function in fresh ejaculate. Although the β-defensin 126 (DEFB126) protein is associated with asthenozoospermia, DEFB126 gene polymorphisms have not been extensively studied. Therefore, the association between DEFB126 gene polymorphisms and asthenozoospermia requires further investigation. Screening was performed by semen analysis, karyotype analysis, and Y microdeletion detection, and 102 fertile men and 106 men with asthenozoospermia in Chengdu, China, were selected for DEFB126 gene sequence analyses. Seven nucleotide mutations and two nucleotide deletions in the DEFB126 gene were detected. rs11467417 (317-318 del/del), rs11467497 (163-166 wt/del), c.152T>C, and c.227A>G were significantly different between the control and asthenozoospermia groups, likely representing high-risk genetic factors for asthenozoospermia among males. DEFB126 expression was not observed in sperm with rs11467497 homozygous deletion and was unstable in sperm with rs11467417 homozygous deletion. The rs11467497 four-nucleotide deletion leads to truncation of DEFB126 at the carboxy-terminus, and the rs11467417 binucleotide deletion produces a non-stop messenger RNA (mRNA). The above deletions may be responsible for male hypofertility and infertility by reducing DEFB126 affinity to sperm surfaces. Based on in silico analysis, the amino acids 51M and 76K are located in the highly conserved domain; c.152T>C (M51T) and c.227A>G (K76R) are predicted to be damaging and capable of changing alternative splice, structural and posttranslational modification sites of the RNA, as well as the secondary structure, structural stability, and hydrophobicity of the protein, suggesting that these mutations are associated with asthenozoospermia.
Male ; Humans ; Asthenozoospermia/metabolism* ; Sperm Motility/genetics* ; Homozygote ; Polymorphism, Single Nucleotide ; Semen ; Sequence Deletion/genetics* ; Spermatozoa/metabolism* ; Nucleotides/metabolism* ; beta-Defensins/metabolism*

Objective To analyze the status and trends for the prevalence，morbidity，mortality， and DALY of tuberculosis ( TB) in China from 1990 to 2017，so as to provide scientific basis for TB pre- vention and control． Methods From 1990 to 2017 in China，the United States，India，Japan，and the global，the TB ( standardized) prevalence，( standardized) morbidity，( standardized) mortality，and ( standardized) DALY rates were obtained from Institute for Health Metrics and Evaluation ( IHME) web- site，and comparative analysis of changes in these indicators with years were carried out． Ｒesults In 2017， the prevalence，morbidity，mortality，and DALY rate of TB in China were higher in the high-age group than in the low-age group． The prevalence，morbidity，mortality，and DALY rates were all higher for males than females． The prevalence was 29 641. 59/100 000 and 28 803. 26/100 000 for male and female，respectively． The male and female mortality rates were 72. 23/100 000 and 44. 85/100 000． The male and female mortali- ty rates were 3. 85/100 000 and 1. 67/100 000，and the DALY rate of male and female were 156. 95/100 000 and 84. 73/100 000． From 1990 to 2017，the prevalence of TB decreased from 32 178. 39/100 000 in 1990 to 29 231. 80/100 000 in 2017，the morbidity rate of TB decreased from 141. 83/100 000 in 1990 to 58. 83/100 000 in 2017，the mortality rate of TB decreased from 13. 82/100 000 in1990 to 2. 78/100 000 in 2017，and the DALY rate of TB decreased from 582. 87/100 000 in 1990 to 121. 60/100 000 in 2017． Conclusions The burden of TB in China declined markedly from 1990 to 2017． Although some progress has been made in the prevention and treatment of TB in China，but there is still a certain gap compared with de- veloped countries． It is still necessary to further reduce the morbidity rate and mortality rate of TB．

OBJECTIVETo investigate the prognostic value of F-FDG PET/CT scan quantization parameters, max standardized uptake value （SUVmax）, metabolic tumor volume （MTV）, total lesion glycolysis （TLG） and other clinical factors for prognostic evaluation of paticnts with diffuse large B-cell lymphoma （DLBCL）.

METHODSPET/CT scan and clinical data of a total of 65 newly diagnosed DLBCL patients who received Rituximab plus cyclophosphamide, doxorubicin, vincristine and prednisone （R-CHOP） chemotherapy as first-line treatment were analyzed. All patients received a PET/CT scan at diagnosis and an interim PET/CT after 2-4 circles of chemotherapies. The related parameters of SUVmax, MTV and TLG were acquired by analyzing and calculating the scan results. The receiver operating characteristic （ROC） curve was used to determine the optimal cut-off of parameters. Pearson chi-square test, Kaplan-Meier method and COX proportional hazard model were performed to analyze the prognostic value of PET/CT related parameters and clinical factors in progression-free survival （PFS）.

RESULTSAge, B symptom, Ann Arbor stage and extra-nodal involvement in major organs significantly related with PFS （P<0.05）, but the SUVmax didn't relalt with the prognosis. The cut-off values of MTV0, MTV1, TLG0 and TLG1 for disease recurrence or progression were 172.20cm , 4.32cm , 1043.33g and 14.07g. The lower MTV and TLG groups showed longer PFS significantly. In the multivariate Cox regression model, B symptoms, MTV1 and TLG1 were the independent prognostic risk factors.

CONCLUSIONMTV and TLG at baseline and in the interim and NCCN-IPI correlate with disease prognosis. SUVmax related parameters hare no significant relationship with prognosis. Besides MTV and TLG during treatment are the independent prognostic risk factors suggesting more predictive value than NCCN-IPI.