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OBJECTIVE To investigate the marketing status, general characteristics, and time trends of rare disease drugs in China. METHODS Based on 121 kinds of rare diseases included in the First Batch of Rare Disease Catalog, the names and marketing approval information of corresponding drugs with indications were obtained from the databases of the Center for Drug Evaluation, National Medical Products Administration and Yaozhi.com, and the relevant characteristic variables were extracted for descriptive statistical analysis. RESULTS As of December 31, 2022, only 32 of 121 rare diseases have therapeutic drugs available for treatment on the market in China, and 79 rare disease drugs have been approved. Among them, 46.84% of the drugs are domestic drugs, 88.61% of the drugs are approved for use in both adults and children; 67.09% are chemicals and 59.49% are injections. According to the ATC classification, Category A (digestive system drugs) is the most, accounting for 20.25%. The number of rare disease drugs on the market each year is the highest in 2021, with an overall upward trend from 2018 to 2021 and a downward trend in 2022. Among rare disease drugs on the market each year, according to the ATC classification, the number of Category L (antineoplastics and immune inhibitors) will be the largest in 2021, being 5. By dosage form, oral medicines were marketed in the largest number in 2022, and injectable medicines in 2021. CONCLUSIONS In recent years, the number of approved rare disease drugs in China has been continuously increasing, but it is still far from meeting the needs of patients, and there is still a lack of domestically approved rare disease drugs. We should further accelerate the research and development of rare disease drugs, and promote the import and replication of rare disease drugs.
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OBJECTIVE To investigate the marketing status, general characteristics, and time trends of rare disease drugs in China. METHODS Based on 121 kinds of rare diseases included in the First Batch of Rare Disease Catalog, the names and marketing approval information of corresponding drugs with indications were obtained from the databases of the Center for Drug Evaluation, National Medical Products Administration and Yaozhi.com, and the relevant characteristic variables were extracted for descriptive statistical analysis. RESULTS As of December 31, 2022, only 32 of 121 rare diseases have therapeutic drugs available for treatment on the market in China, and 79 rare disease drugs have been approved. Among them, 46.84% of the drugs are domestic drugs, 88.61% of the drugs are approved for use in both adults and children; 67.09% are chemicals and 59.49% are injections. According to the ATC classification, Category A (digestive system drugs) is the most, accounting for 20.25%. The number of rare disease drugs on the market each year is the highest in 2021, with an overall upward trend from 2018 to 2021 and a downward trend in 2022. Among rare disease drugs on the market each year, according to the ATC classification, the number of Category L (antineoplastics and immune inhibitors) will be the largest in 2021, being 5. By dosage form, oral medicines were marketed in the largest number in 2022, and injectable medicines in 2021. CONCLUSIONS In recent years, the number of approved rare disease drugs in China has been continuously increasing, but it is still far from meeting the needs of patients, and there is still a lack of domestically approved rare disease drugs. We should further accelerate the research and development of rare disease drugs, and promote the import and replication of rare disease drugs.
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Objective:To investigate the safety and efficacy of excimer laser ablation (ELA) combined with drug-coated balloon (DCB) for the below-the-knee (BTK) artery lesions.Methods:From Jun 2019 to Nov 2022, 22 patients receiving ELA combined with DCB in atherosclerotic lesions of BTK artery at these two centers were retrospectively analyzed.Results:In these 22 patients there were 3 (13.6%) suffering from stenosis and 19 (86.4%) from chronic total occlusion (CTO) lesions. The mean length of lesion was (25.6±5.7) cm. The technical success rate was 95.5%. Flow-limiting dissection was found in 1 (4.5%) patient and a bailout stent was deployed. The mean ankle-brachial index (ABI) significantly improved during the follow-up period compared with that before the treatment. The median follow-up time was 21.5 months. The 1-year primary patency rate was 80.3%Conclusion:ELA combined with DCB was safe and effective in the treatment of atherosclerotic lesions of BTK artery, improving the primary patency.
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OBJECTIVES@#Stroke has become the leading cause of death and disability among adults in China. This study aims to analyze the disease burden based on gender and age and the risk factors for stroke subtypes in China 2019, and to provide reference for targeted stroke prevention and control.@*METHODS@#Based on 2019 data of the Global Burden of Disease (GBD), the gender and age in patients with different stroke subtypes (ischemic stroke, intracranial hemorrhage, subarachnoid hemorrhage) in China 2019 was described by using disability-adjusted life years (DALY), and attributable burden of related risk factors was analyzed.@*RESULTS@#In 2019, the burden of intracranial hemorrhage was the heaviest one in China, resulting in 22.210 6 million person years of DALY, following by ischemic stroke and subarachnoid hemorrhage, resulting in 21.393 9 and 2.344 7 million person years of DALY, respectively. Among them, except the 0-14 age group, the disease burden of different subtypes of stroke in men was higher than that in women. The disease burden of ischemic stroke was increased with age in both men and women, with the heaviest disease burden in ≥70 years group. The disease burden of intracranial hemorrhage and subarachnoid hemorrhage was the heaviest in males aged 50-69 years old, and in females aged ≥70 years and 50-69 years, respectively. Metabolic factors were the main risk factors in all ages of different stroke subtypes, and the most important risk factor was high systolic blood pressure. Other risk factors were different between men and women. Smoking, high body mass index, high low-density lipoprotein, and outdoor particulate matter pollution were the main risk factors for stroke in men, while high body mass index, outdoor particulate matter pollution, and high fasting blood glucose were the main risk factors of stroke in women. The main risk were different among different age groups.@*CONCLUSIONS@#The burden and attributable risk factors for different stroke subtypes are discrepancy in different gender and age groups. Targeted interventions should be conducted in the future to reduce the burden of stroke.
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Male , Adult , Humans , Female , Infant, Newborn , Infant , Child, Preschool , Child , Adolescent , Middle Aged , Aged , Subarachnoid Hemorrhage/epidemiology , Quality-Adjusted Life Years , Cost of Illness , Stroke/etiology , Risk Factors , China/epidemiology , Particulate Matter , Ischemic Stroke , Intracranial Hemorrhages/etiologyABSTRACT
Objective:To discuss the strategies of distal embolic filter protection(DEFP) during excimer laser ablation (ELA) or percutaneous mechanical thrombectomy (PMT) in treatment of peripheral artery disease.Methods:Clinical data of 29 patients undergoing ELA or PMT under the DEFP from Oct 2019 to Aug 2021 were retrospectively collected to analyze the strategies of DEFP and high-risk factors of capturing clinically significant macrodebris.Results:There were 21 males and 8 females, aged (70.3±11.0) years with 32 lesions (29 limbs) including 5 in-stent restenosis (ISR), 10 thrombosis and 17 chronic total occlusion (CTO). The technical success rate of DEFP device release and recovery was 100%. The overall debris capture rate was 77.3% and the macrodebris capture rate was 36.4%. Even with DEFP the distal embolization (DE) incidence was 3.4%. When ELA for CTO with severe calcification or long-segment ISR lesions, the capture rate of macrodebris was as high as 60.0%, and the former was significantly higher than ELA for CTO without high calcification lesions ( P<0.05). Conclusion:ELA or PMT under the DEFP in treatment of peripheral artery disease appears to be of great significance in preventing DE.
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Objective:To evaluate the safety and efficacy of a Zenith fenestrated aortic stent-graft (ZFEN) system in the treatment of short-necked aneurysms.Methods:The clinical data of 5 patients receiving F-EVAR in Zhongshan Hospital, Fudan University from Mar 2018 to Sep 2019 according to the standards of short-necked abdominal aortic aneurysm and COOK ZFEN custom stent were retrospectively analyzed.Results:Patients' average age was (69±11) years old. The time of stent customization was 4-6 weeks. The average maximum diameter of AAA was (60.8±14.0) mm. The average proximal neck diameter was (26.0±2.6) mm and the average length anchoring zone was (6.4±2.2) mm. The rate of technical success was 100%. There was no intraoperative loss of visceral vessels and stent displacement. The meane follow-up time was 6.8 months. The perioperative mortality was 0, and there were no other major complications. There were 3 patients with simple type Ⅱ endoleak and 1 patient with type Ⅲ endoleak. The endoleaks disappeared in 4 patients during follow-up of 3-12 months. 1 patient suffered with type Ⅱ and type Ⅲ endoleak, and the visceral vessels and branching stents were patent in all patients.Conclusion:The short term result of COOK ZFEN stent-graft system was safe and feasible for the treatment of short-necked AAA.
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Objective:To evaluate the effect of Rotarex in peripheral arterial disease (PAD).Methods:The clinical data of 90 PAD patients treated with Rotarex from Aug 2018 to Feb 2020 were retrospectively analyzed.Results:Among the 90 patients, 45 patients had atherosclerotic obliterans complicated with acute thrombosis (ASOCAT), 27 patients had graft restenosis or reocclusion, 16 patients had primary or embolism-induced thrombosis, 2 patients had traumatic or iatrogenic arterial occlusion. Except for 2 patients undergoing hybrid surgery, 88 patients underwent endovascular treatment. Two patients died perioperatively. Within 12 months follow-up, 2 patients died, 4 patients underwent major amputation, target arteries of 10 patients were re-stenosed or re-occluded and 5 patients were lost to follow-up. Compared with the preoperative ankle-branchial index (ABI), significant increase was observed in the 12-month ABI (0.80±0.22 vs. 0.43±0.16, P<0.01). The 12-month restenosis/re-occlusion-free rate was 82.7%, and the 12-month major amputation-free survival (MAFS) was 91.6%. Conclusion:For PAD patients, acceptable outcomes can be achieved with reasonable use of Rotarex for debulking, combined with balloon, stent and other techniques to correct the residual lesions.
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Objective:To summarize the early and intermediate outcomes of second-grade false-lumen endovascular occlusive repair (FLEVOR) for aortic dissection.Methods:The clinical data of 12 patients undergoing second-grade FLEVOR after proximal repair of aortic dissection at our center from Aug 2016 to Aug 2019 was retrospectively analyzed.Results:The mean age was (51.3±14.9) years old. Four patients received open repair due to Stanford type A aortic dissection previously, the other 8 patients underwent thoracic endovascular aortic repair for Stanford type B aortic dissection. The time to the proximal repair varied from 3 months to 16 years. The technical success rate was 100%. Visceral ischemia, early spinal cord ischemia and in-hospital death did not occur perioperatively. The mean follow-up time was 16.7 months. Persistent false lumen perfusion was found in 3 patients, complete thrombosis of false lumen was achieved in the other 8 patients. The maximum diameter of abdominal aorta decreased in 8 (72.7%) patients.Conclusions:FLEVOR could block the blood flow from the false lumen and induce the thrombosis of false lumen, which promotes the aortic remodeling. Meanwhile, FLEVOR could protect the blood supply of spinal cord and viscera, and reduce the risk of type Ⅱ endoleaks.
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Objective:To evaluate the correlation between tumor size and surgical outcomes of Shamblin Ⅱ/Ⅲ carotid body tumor (CBT) and the prognosis.Methods:From Apr 2011 to Dec 2017, 88 patients with Shamblin Ⅱ and Ⅲ CBTs undergoing tumor resection were retrospectively analyzed. The long-term prognosis of surgical resection was also evaluated.Results:One patient died of multi-organ failure postoperatively, two patients abandoned surgery. The perioperative mortality was 1%. 85 patients having 87 lesions successfully resected were analyzed. There were 13 Shamblin Ⅱ and 74 Shamblin Ⅲ CBTs resection. Two patients had bilateral CBTs resection. The median of tumor size, procedural blood loss, procedural time, and length of hospital stay was 3.0 (1.2-5.5) cm, 200 (0-2 500) ml, 162 (85-430) min, and 10 (6-28) d, respectively. Seventy-two patients had carotid vascular intervention during the procedure, and thirty-three patients had neurological complications after the procedure. Correlation analysis showed that the tumor size had positive relationship with blood loss (Spearman R=0.35, P<0.01) and procedural time (Spearman R= 0.54, P<0.01). Tumor size was positively associated with the risk of cranial neurological complication ( OR=1.72, 95% CI 1.08-2.73, P=0.02) and carotid vascular intervention ( OR=2.69, 95% CI 1.27-5.69, P=0.01). There were four patients who were diagnosed with malignant CBT due to the metastasis. Three suffered CBT recurrence after 1, 5, and 7 years during the follow up. Conclusions:Shamblin Ⅱ or Ⅲ CBTs should be intervened as early as possible to facilitate the surgical resection, as well as to reduce the risk of carotid vascular and neurological complications.
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Objective:To discuss the clinical treatment for arteriosclerosis obliterans combined with acute thrombosis (ASOCAT) of lower extremities.Methods:The treatment methods and results of 30 patients with ASOCAT admitted to our center from Jan 2009 to May 2019 were retrospectively analyzed.Results:The course of acute thrombosis in 30 patients was (9.5±5.2) days, and the average course of ASO was (2.1±1.4) years. Among 30 patients, 13 patients had aortoiliac occlusion (type Ⅰ), and 17 patients had femoropopliteal lesion (type Ⅱ). Twenty-eight patients underwent endovascular treatment, 1 had hybrid operation, and 1 was given aorto-bilateral femoral bypass. One patient died perioperatively. 24 patients were followed up for (16.3±16.1) months. One died during follow-up.Two patients underwent above-knee amputation within 6 months. Two patients had distal superficial femoral artery reocclusion within 12 months. The restenosis/reocclusion rates within 12 months of type Ⅰ and Ⅱ patients were 12.5% and 21.4%, respectively. The 6/12-month amputation-free survival rates for type Ⅰ and type Ⅱ were 87.5%/87.5% and 92.8%/85.7%, respectively.Conclusion:Reasonable and active use of open surgery, endovascular treatment or hybrid operation could achieve acceptable outcomes in patients with ASOCAT.
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Based on career cycle theory, this paper mainly discussed the training system of teachers in colleges and universities. Considering the teachers' teaching and scientific research development, tachers' career in colleges and universities can be divided into four stages: adapting transition stage, growth stage, stable contribution stage and regression stage. From the perspective of the above-mentioned analysis of the characteristics in different stages, well-directed training schemes should be set up. The practice results show that the teacher' training system has promoted the growth of teachers, strengthened the competitive power of teachers, and achieved good training results.
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In this study, the necessity of professional master's degree training in clinical pharmacy was analyzed by means of literature research and practice summary, and the existing problems in current professional curriculum, tutor team and practice teaching were discussed. In view of the existing problems, this paper puts forward that medical colleges and universities should give full play to their medical resources, draw lessons from the talents training experience of clinical pharmacists training base, adopt measures such as optimizing curriculum system, selecting tutors, attaching importance to practical teaching, so as to improve the quality of postgraduate training in clinical pharmacy, train high-level clinical pharmacists, and promote the development of clinical pharmacy in China.
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In this study,the necessity of professional master's degree training in clinical pharmacy was analyzed by means of literature research and practice summary,and the existing problems in current professional curriculum,tutor team and practice teaching were discussed.In view of the existing problems,this paper puts forward that medical colleges and universities should give full play to their medical resources,draw lessons from the talents training experience of clinical pharmacists training base,adopt measures such as optimizing curriculum system,selecting tutors,attaching importance to practical teaching,so as to improve the quality of postgraduate training in clinical pharmacy,train high-level clinical pharmacists,and promote the development of clinical pharmacy in China.
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OBJECTIVE@#To analyze clinical features, surgical treatment and outcomes of neurilemmoma and carotid body tumors in bifurcation of carotid artery.@*METHODS@#The clinical data of 17 patients with neurilemmomas and 76 patients with carotid body tumors at the bifurcation of carotid artery, who were surgically treated in Zhongshan Hospital of Fudan University from March 2012 to November 2016, were retrospectively analyzed. The clinicopathological characteristics, surgical procedures and outcomes were compared between two groups.@*RESULTS@#No difference of preoperative clinical demographic data was found between two groups. Operation time of the neurilemmoma group was significantly shorter than that of the carotid body tumor group[(93.9±30.8) min vs. (159.3±52.9) min, 0.05).@*CONCLUSIONS@#The clinical manifestations of neurilemmoma and carotid body tumors at carotid artery bifurcation are similar. The carotid body tumor group has a longer operating time, larger intra-operative blood loss, higher external carotid resection rate and relative higher incidence of malignancy. More cautions should be given when carotid body tumors at carotid artery bifurcation are treated.
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Humans , Carotid Arteries , General Surgery , Carotid Body Tumor , Pathology , General Surgery , Neurilemmoma , Pathology , General Surgery , Retrospective Studies , Treatment OutcomeABSTRACT
In order to cultivate clinical pharmacy undergraduates to have better quality, Chongqing Medical University collaborated with The University of Chicago and University of Cincinnati in the reform of the course of pharmacotherapeutics. We build pharmacotherapeutics series curriculum with the center of disease, construct department of clinical pharmacy for transnational departments, build course leader and teaching team of pharmacotherapeutics series curriculum , compile teaching program and its material of pharmacotherapeutics series curriculum, build pharmacotherapeutics series curriculum and teaching model in line with the current direction of China's education system of clinical pharmacy training, reform teaching methods, and strengthen clinical pharmacy practice and community clinical pharmacy education.
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Objective To evaluate the safety and efficacy of transplantation of purified CD34 + cells (PCCs) in treatment of critical limb ischemia (CLI) caused by thromboangiitis obliterans (TAO).Methods From May 2009 to June 2015,34 TAO-induced-CLI cases underwent PCCs transplantation.None of these patients were eligible for surgical or endovascular revascularization.G-CSF was subcutaneously injected for 5 days before peripheral CD34 + cells were isolated,purified and intramuscularly injected in the limbs.Patients were regularly follow-up.Results Technical success was achieved in all cases.The mean number of transplanted cells was (7.5 ± 2.4) × 105/kg.The follow-up was accomplished in 32 cases,ranging from 6 to 79 months (mean 45 ±24 months),and two patients were lost.Wong-Baker FACES pain rating scale score significantly decreased from 8.0 ±2.0(4-10)to 2.2 ±3.1 (P <0.05) at 1 month.The Peak pain-free walking time improved from (4.0 ± 2.0) min to (13.5 ± 5.3) min (P < 0.05) at 3 months and (19.0 ± 3.1) min (P < 0.05) at 6 months.The ankle-brachial index increased from 0.42 ± 0.20 to 0.50 ± 0.10 (P < 0.05) at 3 months and 0.52 ± 0.11 (P < 0.05) at 6 months,respectively.Transcutaneous partial oxygen pressure rose from (25 ± 11) mmHg to (48 ± 11) mmHg(P < 0.05) at 3 months and (58 ± 10) mmHg (P < 0.001) at 6 months,respectively.Ulcers healed in 21 out of 22 patients at (5 ± 4) months.The overall amputation-free survival rate was 94.1% at 6 months and 91.2% at 48 months.No major adverse events were observed perioperatively or postoperatively.Conclusions Transplantation of PCCs could yield safe,satisfactory and durable treatment outcomes in patients with TAO-induced-CLI.
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Objective@#To evaluate the safety and effectiveness of the Endurant stent graft for abdominal aortic aneurysm cases with proximal neck length <10 mm.@*Methods@#This study was a retrospective analysis. From January 2010 to May 2015, 22 consecutive abdominal aortic aneurysm patients with proximal neck length <10 mm were treated with endovascular aortic repair by Endurant stent graft in Department of Vascular Surgery, Zhongshan Hospital, Fudan University. There were 19 (86.3%) male cases, aging from 57 to 84 years. All patients underwent preoperative CT angiography and the anatomic parameters of abdominal aortic aneurysm were measured. All patients performed standard endovascular aortic repair first and if there was obvious proximal typeⅠ endoleak, the CUFF or the chimney technology were applied to observed the perioperative technical and the clinical success rate. During follow-up, the incidence of adverse events and the reintervention rate were observed.@*Results@#These 22 cases had proximal neck length 5 to 9 mm with the average of (7.2±1.4) mm. Immediate endoleak occurred in 5 patients with 4 cases of proximal typeⅠ endoleak, 3 cases were implanted proximal CUFF, 1 case implanted CUFF and left renal artery chimney. One case died perioperatively, the clinical success rate was 95.4%, the technical success rate was 77.3%. During the follow-up of 6 to 54 months, there was 1 case with delayed proximal type-1 endoleak, during operation the patient had no endoleak, but disappeared 6 months later without further intervention. So the incidence of adverse event was 4.5% and reintervention rate was 0.@*Conclusion@#The Endurant stent graft for abdominal aortic aneurysm cases with proximal neck length < 10 mm is safe and effective.
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<p><b>OBJECTIVE</b>To evaluate the efficacy of endovascular repair of popliteal artery aneurysm (PAA) and to summarize the clinical experience and lessons.</p><p><b>METHODS</b>A clinical data of 9 PAA cases (males 8, females 1, age range 55-84) undergoing endovascular repair from October 2006 to December 2014 at Zhongshan Hospital Fudan University were retrospectively analyzed. CT angiography was made in all of the 9 cases preoperatively, and 10 PAA were included in the analysis. Of the 9 cases, 6 cases were symptomatic. The mean diameter of PAA was 3.9 cm (range, 2.1-8.2).</p><p><b>RESULTS</b>The technique successful rate of endovascular repair was 10/10. The preoperative complications were 2/10, including one case suffered hematoma in the arterial puncture site and 1 case with distal artery embolization. The mean follow-up was 33 months (range, 3-87). During the follow-up, 2 cases received the secondary intervention and drug thrombolysis because of the stent distal thrombosis. The stent-graft primary patency at 1 year was 78.8% (95% CI: 48%-91%). One case suffered stent fracture, and one case died of brainstem hemorrhage 15 months post endovascular repair of PAA.</p><p><b>CONCLUSIONS</b>Endovascular treatment of PAA is safe and effective in perioperative and midterm follow-up. However, individual endovascular strategy and postoperative complications in the long-term follow-up should be payed more attention.</p>
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Aged , Aged, 80 and over , Female , Humans , Male , Middle Aged , Aneurysm , General Surgery , Angiography , Embolization, Therapeutic , Endovascular Procedures , Popliteal Artery , Pathology , General Surgery , Postoperative Complications , Retrospective Studies , Stents , Treatment Outcome , Vascular PatencyABSTRACT
Clinical internship is for bachelor students to learn how to integrate theories in textbooks with clinical practice.Clinical pharmacist training is for pharmacists to obtain and enforce the capability of rational use of drugs.These two are at the different stages of clinical pharmacist cultivation mode.They are different in teaching nature,goal and students' background.The necessity of clinical internship bridged to clinical pharmacist training is discussed in this paper and after the convergence,the teaching design of chnical internship program bridged to the training is characterized by basic education which focuses on basic theories,knowledge and skills of clinical medicine and clinical pharmacy relevant to a certain common disease.The optimized internship program will work as a good foundation for students to face the challenges of clinical pharmacist training after graduation from school.
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<p><b>OBJECTIVE</b>To evaluate the effect of extensive aortic diseases (EAD) after hybrid repair with supra-arch branches or visceral arterial debranching and endovascular repair.</p><p><b>METHODS</b>A total of 24 EAD patients (19 male and 5 female) underwent hybrid repair in Department of Vascular Surgery, Zhongshan Hospital, Fudan University between March 2005 and April 2015. The clinical data was analyzed retrospectively. The mean age was (49±12) years (from 29 to 69 years). The high-risk candidates for open surgery were enrolled in the study. The patients, including 14 cases with thoracic aortic aneurysms and 10 cases with aortic dissection, were treated by one or two stages. Post-operative follow-up with CT angiography was performed at 3, 6 and 12 months and then annually thereafter.</p><p><b>RESULTS</b>A total of 74 branches, including 63 with visceral arteries and 11 with supra-arch arteries, were recanalized. Nine patients were treated in two-stage hybrid procedure and fifteen were repaired by one stage. The perioperative mortality was 12.5% (3/24), whereas the aneurysm-related mortality was 8.3% (2/24). The 30-day patency of the grafts was 95.9% (71/74). There was no endoleak, paralysis, and intestinal ischemia at 30 days post-hybrid procedures. Four cases (16.6%) suffered acute renal dysfunction. During the follow-up (3 to 123 months), there were two deaths and one endoleak (type Ia and III) which emerged at 4 months post-procedure. The 1-year and 3-year survival rates were 81.7% and 73.5%, respectively.</p><p><b>CONCLUSIONS</b>Hybrid treatment is safe and effective for complex EAD patients in the midterm follow-up. It is especially suitable for the high-risk patients with comorbidities, re-intervention, or little tolerance to open surgery repair.</p>