OBJECTIVE: To introduce and compare research support for rare disease and orphan drug innovation in China and United States, and provide reference for relevant policies in China. METHODS: Data of main source of funding for rare disease research in two countries ie. National Institutes of Health, Food and Drug Administration in the United States and National Natural Science Foundation of China were analyzed and compared. RESULTS: US NIH gives substantial support for rare disease research every year with funded capital growing. FDA Orphan Products Grants program provides incentives for sponsors to develop products for rare diseases. In China, however, there is no specific support project for rare disease research, and there is a huge gap in funding efforts for rare disease research between China and the United States. CONCLUSION: China should establish rare disease research center to promote rare disease research and set up specific funding for rare diseases research, increase efforts to support research and innovation for rare diseases and orphan drugs, in order to protect the health interests of patients with rare diseases.

OBJECTIVE: To introduce international reimbursement policies on drugs for pulmonary arterial hypertension (PAH), and provide a reference for establishing relevant policies in China. METHODS: Reimbursement policies on PAH drugs of Australia, Canada, America and some EU countries were analyzed and compared with the status quo in China. RESULTS: Although expensive, PAH drugs are reimbursed by medical insurance in most countries included in this study. However, the accessibility of PAH drugs in China remains very poor with only a few cities including these drugs in the medical insurance. Patients with PAH face with poverty caused by disease, even abandoning treatment. CONCLUSION: Learning the experience from countries with rare disease act and good medical insurance system and analysing the earliest attempts to reimburse PAH drugs in China will help promote the reimbursement policy on PAH drugs in China.

OBJECTIVETo investigate the incidence of late onset of hypogonadism (LOH) in males as well as the sensitivity, specificity and applicability of the androgen deficiency in aging males (ADAM) questionnaire and the aging males' symptoms (AMS) scale in Chinese males based on the community population data.

METHODSA total of 1 498 old and middle-aged healthy males attended the ADAM and AMS investigations, of whom 434 received the measurement of the concentration of serum reproductive hormones, the positive rate of LOH screening, the rate of androgen deficiency, the clinical incidence of LOH, and the sensitivity and specificity of ADAM and AMS by tT and fT cut-off. The symptom evaluation cut-off value of the AMS score was optimized using the ROC curve.

RESULTSAlthough 5 of the subjects had previously received irregular testosterone supplement, none of them was given or actively sought for androgen therapy at the time of the investigation. Among the 40 - 69 years old males, the mean positive rates of LOH screening by ADAM and AMS were 80.77% and 32.34%, and the mean androgen deficiency rates obtained by tT and fT cut-off were 14.02% and 43.69%. The mean clinical incidences of LOH in the ADMA- and AMS-positive subjects were 37.85% and 15.42%. According to the fT cut-off, the sensitivities of ADAM and AMS were 86.63% and 35.29%, and their specificities were 24.48% and 63.49%, respectively. The symptom evaluation cut-off value of the AMS score optimized by the ROC curve was 19.5.

CONCLUSIONThe sensitivity and specificity of ADAM and AMS in the Chinese population are basically consistent with the results of most studies abroad, while the positive rate of LOH screening, the rate of androgen deficiency and the clinical incidence of LOH obviously higher in the former than those reported in other studies. Both ADAM and AMS are applicable to the Chinese population. The former is advantageous for its high sensitivity, time saving and easy operation, and therefore suitable for screening LOH, while the latter can be used for monitoring therapeutic efficacy.

Adult ; Aged ; Asian Continental Ancestry Group ; Humans ; Hypogonadism ; diagnosis ; Male ; Mass Screening ; methods ; Middle Aged ; Sensitivity and Specificity ; Surveys and Questionnaires

OBJECTIVETo evaluate the results of video-assisted thoracoscopic extended thymectomy for myasthenia gravis.

METHODSWe retrospectively reviewed data from 107 patients received thoracoscopic extended thymectomy from June 1995 to June 2004. All patients had confirmed diagnosis of myasthenia gravis by clinical manifestation and electromyogram. Thoracoscopic extended thymectomy as well as dissection of all fatty tissue anterior to the pericardium was performed.

RESULTSDuring a follow-up of 1-98 months, symptom was significantly improved in 83% of patients, including 34 patients experienced complete remission. There was no postoperative mortality.

CONCLUSIONFavorable results of video-assisted thoracoscopic extended thymectomy can be achieved in patients with myasthenia gravis. The technique is safe and minimally invasive.

Adolescent ; Adult ; Aged ; Female ; Follow-Up Studies ; Humans ; Male ; Middle Aged ; Myasthenia Gravis ; surgery ; Retrospective Studies ; Thoracic Surgery, Video-Assisted ; Thymectomy ; methods ; Treatment Outcome

Objective:To analyze the equity of Traditional Chinese medicine(TCM)human resource allocation across 31 provinces in China and explore its influencing pathways,aiming to provide scientific reference for optimizing the allocation of TCM human resources.Methods:The Health Resource Density Index(HRDI)was employed to measure the equity of TCM human resource allocation in China,and the fuzzy-set qualitative comparative analysis(fsQCA)was utilized to explore the configurational pathways influencing this equity.Results:Based on the data from 2021,the HRDI of TCM human resources in China exhibited significant regional disparities,manifesting as a distribution pattern of"high in the east and low in the west."Three pathways promoting high equity were identified:the internal-external balance-driven pathway(H1),the economy-demand co-driven pathway(H2),and the government-led driving pathway(H3).Meanwhile,three pathways leading to low equity were also recognized:the economy-demand constraint pathway(L1)and the internal-external constraint pathways(L2、L3).Conclusion:There are notable regional disparities in the equity of TCM human resource allocation in China,with multiple factors jointly influencing this equity,among which population density serves as a core factor.In subsequent efforts to enhance equity,it is advisable to consider optimizing the synergies among multiple factors and implementing precise policies for different regions to promote efficient allocation and balanced development of TCM human resources.

OBJECTIVETo investigate the prognostic factors related to triple-negative breast cancer (TNBC) by analyzing clinicopathologic characteristics, treatment and prognosis.

METHODSThree hundred and five TNBC patients treated between January 2004 and December 2011 were enrolled and retrospectively reviewed with Kaplan-Meier and Cox proportional hazards models. All patients were females and the age onset were 24-82 years old (the median were 50 years old).

RESULTSThe follow-up period was 1 to 114 months, with median 38 months. The 5-year disease free survival (DFS) rate was 68% and overall survival (OS) rate 75%. The peak risk of recurrence occurs within the first 2-3 years after initial treatment of the disease, but distant relapse after this time is much less common. Survival analysis showed that surgery type (χ(2) = 4.030, P = 0.045), tumor grade (χ(2) = 8.000, P = 0.046), lymph-vascular invasion (χ(2) = 10.386, P = 0.001) and lymph node stage (χ(2) = 119.36, P = 0.000), TNM stage (χ(2) = 65.961, P = 0.000) and treatment plan (χ(2) = 28.371, 21.874, 32.163, all P = 0.000) were statistically related to DFS; while age (χ(2) = 10.226, P = 0.006), lymph-vascular invasion (χ(2) = 18.881, P = 0.000), lymph node stage (χ(2) = 98.958, P = 0.000), TNM stage (χ(2) = 65.342, P = 0.000) and type of treatment (χ(2) = 17.862, 18.708, 31.921, all P = 0.000) were related to OS. The lymph nodes stage was prognostic factor related to both DFS and OS.

CONCLUSIONSTNBC was characterized by poor prognosis and rapid progression. The lymph nodes metastatic status was the most important prognostic factor of TNBC.

Adult ; Aged ; Aged, 80 and over ; Female ; Follow-Up Studies ; Humans ; Kaplan-Meier Estimate ; Lymph Nodes ; pathology ; Lymphatic Metastasis ; pathology ; Middle Aged ; Prognosis ; Proportional Hazards Models ; Retrospective Studies ; Survival Rate ; Triple Negative Breast Neoplasms ; diagnosis ; pathology

OBJECTIVETo describe the epidemiological characteristics and related factors of SARS in Shanxi in order to provide scientific basis for prevention and control of severe acute respiratory syndrome (SARS).

METHODSData on clinically-diagnosed SARS cases reported to Shanxi Center for Disease Control and Prevention through SARS reporting system of Shanxi province and epidemiological reports were collected from early March to 20 May, 2003. The characteristics of SARS distribution in time, place and population in Shanxi were described. The epidemiological characteristics and related influential factors were analyzed with EPI info 6.0 software.

RESULTSSince the first imported SARS case was diagnosed clinically on 7 March and till 20 May in Shanxi province, the number of cumulative clinically-diagnosed SARS cases were 445 with an attack rate of 1.34/10,000. 20 deaths occurred in that period with the mortality rate 4.49%. The number of cases increased from 28 March and formed the first peak. However, the number continued to increase until 18 April to have formed the second peak. Since then, the number of cases has gradually decreased gradually. Since 19 May, there has been no clinically-diagnosed cases being reported. SARS cases were mostly seen in urban areas of the city (83.82% of the total SARS cases) with sporadic cases found in rural areas. Students and medical staff and people from 20 - 59 years of age occupied the large part of the cases. Age specific mortality rate increased with age and the male/female ratio was 1:0.87.

CONCLUSIONIn Shanxi province, the SARS epidemic seemed to have had the following stages: importation of the first case, gradual increase of the number of cases to reach the peak and decreasing. Case identification at early stage as well as taking measures to decrease the chance of transmission were strategically crucial for controlling the spread of SARS virus in the community.

China ; epidemiology ; Female ; Humans ; Male ; Occupations ; Severe Acute Respiratory Syndrome ; diagnosis ; epidemiology ; mortality

In this research, the effect of novel retinoid SX-116 on acute promyelocytic leukemia cell line NB4 was studied in vitro. Cell proliferation, cell morphological characters, flow cytometry, DNA electrophoresis and RT-PCR were observational parameters. The results showed that treated with SX-116 at 10(-6) mol/L, the growth and survival of NB4 cells were markedly inhibited, morphological changes of apoptosis, including membrane blebbing, chromosome condensation and fragmentation of nuclei were observed in NB4 cells after 24 hours exposure of SX-116. Further studies showed "DNA ladder" in genomic DNA electrophoresis, as well as a typical apoptotic peak below G(1) phase presented in flow cytometry. The expression of apoptosis - related gene bcl-2 and p53 were examined. The level of bcl-2 mRNA was downregulated by 6-hour treatment of SX-116, while the gene restored to the normal level by following 12-, 24- and 48-hour exposure. However, p53 mRNA was unchanged during the treatment. The results demonstrated that SX-116 could induce apoptosis of NB4 cells while the mechanism remains to be studied.

Jumonji domain-containing protein D3(JMJD3)is a 2-oxoglutarate-dependent dioxygenase that specif-ically removes transcriptional repression marks di-and tri-methylated groups from lysine 27 on histone 3(H3K27me2/3).The erasure of these marks leads to the activation of some associated genes,thereby influencing various biological processes,such as development,differentiation,and immune response.However,comprehensive descriptions regarding the relationship between JMJD3 and inflammation are lacking.Here,we provide a comprehensive overview of JMJD3,including its structure,functions,and involvement in inflammatory pathways.In addition,we summarize the evidence supporting JMJD3's role in several inflammatory diseases,as well as the potential therapeutic applications of JMJD3 inhibitors.Additionally,we also discuss the challenges and opportunities associated with investigating the functions of JMJD3 and developing targeted inhibitors and propose feasible solutions to provide valuable insights into the functional exploration and discovery of potential drugs targeting JMJD3 for inflammatory diseases.

Aim To explore the effect and mechanism of the artesunate(ART)on hepatocellular carcinoma(HCC).Methods The cell lines MHCC-97H and HCC-LM3 were used to be detected.MTT and clone formation were used to determine the cell proliferation;Wound healing was used to detect the cell migration;Transwell was used to test the cell invasion.Flow-cy-tometry was used to detect cell apoptosis and cell cy-cle.RNA-seq and qRT-PCR was used to detect the genes expression.Results The proliferation,migra-tion and invasion of treated cells were obviously inhibi-ted(P＜0.01).Moreover,the apoptosis rate in-creased significantly,so did the proportion of G2/M cells.Transcriptomic analysis identified GADD45A as a potential target of ART through RNA-sequencing da-ta,and suggested that ART might induce apoptosis and cell cycle arrest through regulating the expression of GADD45A.In addition,the results of mechanism studies and signaling analysis suggested that GADD45A had interaction with its upstream gene NACC1(nucle-us accumbens associated 1).Moreover,after ART treatment,the expressions of GADD45A and NACC1 were changed significantly.Conclusion ART may be a potential drug to resist HCC by affecting the expres-sion of GADD45A and its upstream gene NACC1,which provides a new drug,a new direction and a new method for the clinical treatment of HCC.