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

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.

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

Aim To clarify the regulatory effect of Artesunate(ART) on tumor cell function and cell cycle in the pathological process of esophageal squamous cell carcinoma(ESCC). Methods KYSE450 and TE14 cells were treated with different concentrations of ART. The cells treated with 0 mg •L

OBJECTIVE: To observe the difference for vascular dementia among flipping moxibustion of medicine at acupoints in governor vessel combined with acupuncture, simple medicine and simple acupuncture. METHODS: A total of 120 patients with vascular dementia were randomly assigned into a combination group, a flipping moxibustion group and an acupuncture group, 40 cases in each one, with 2 patients dropping respectively. Flipping moxibustion was used at the acupoints of group A on Monday and at the acupoints of group B on Friday in the flipping moxibustion group. The acupoints of group A were Baihui (GV 20), Dazhui (GV 14), Shenzhu (GV 12), Zhiyang (GV 9), Jizhong (GV 6), Mingmen (GV 4), Yaoyangguan (GV 3), and the acupoints of group B were Fengfu (GV 16), Taodao (GV 13), Shendao (GV 11), Jinsuo (GV 8), Xuanshu (GV 5), Changqiang (GV 1). Acupuncture was used in the acupuncture group at the main acupoints of Baihui (GV 20), Sishencong (EX-HN 1), Neiguan (PC 6), Zusanli (ST 36), Taixi (KI 3) and Xuanzhong (GB 39), matched with the acupoints based on syndrome differentiation, 30 min a time, once a day, continuous 5 times a week. The above two methods were applied in the combination group. All the treaments were for 4 weeks. The indexes were observed before and after treatment, including syndrome differentiationof TCM scale for vascular dementia (SDSVD), simple mental state scale (MMSE), self-care ability of daily life scale (ADL). The clinical effects and safety were evaluated. RESULTS: After treatment, the total effective rate in the combination group was 89.5% (34/38); the rate in the flipping moxibustion group was 65.8% (25/38); the rate in the acupuncture group was 63.2% (24/38). The difference among groups was statistically significant (<0.05). The effect in the combination group was better than those in the flipping moxibustion group and in the acupuncture group (both <0.05). There was no statistically significant difference between the flipping moxibustion group and the acupuncture group (>0.05). The SDSVD scores after treatment were lower and the MMSE and ADL scores after treatment were higher than those before treatment in the three groups (all <0.01), with better results on the above three scores in the combination group than those in the other two groups (<0.05, <0.01), and the differences on the three scores between the flipping moxibustion group and the acupuncture group were not statiatically significant (all >0.05). The treatment in the three groups was safe, without stastical significance (>0.05). CONCLUSION The effect of flipping moxibustion combined with acupuncture for vascular dementia is better than those of simple flipping moxibustion and simple acupuncture. The combination treatment achieves better effect on TCM syndrome, cognitive function and daily activity ability than the other two simple treatment.
Activities of Daily Living ; Acupuncture Points ; Dementia, Vascular ; Humans ; Moxibustion

In this study, we cloned a monoterpene synthases, TwMS from Tripterygium wilfordii suspension cells. TwMS gene contained a 1 797 bp open reading frame (ORF), encoding a polypeptide of 579 amino acids, which deduced isoelectric point (pI) was 6.10 and the calculated molecular weight was 69.75 kDa. Bioinformation analysis showed that the sequence of TwMS was consistent with the feature of monoterpene synthases. Differential expression analysis revealed that the relative expression level of TwMS increased significantly after being induced by methyl jasmonate (MeJA). The highest expression level occurred at 24 h. TwMS protein was successfully expressed in Escherichia coli BL21 (DE3), which laid the foundation for identifying the function of T. wilfordii monoterpene synthases.