OBJECTIVE To assess the efficacy， safety and cost-effectiveness of tenecteplase in the treatment of acute ischemic stroke， and to provide a basis for clinical rational drug use and related decision-making. METHODS The related literature in the PubMed， the Cochrane Library， CNKI， Wanfang data and health technology assessment （HTA） databases were searched from the establishment of the database to June 30th， 2024. Systematic reviews/meta-analyses， pharmacoeconomic studies and HTA reports on tenecteplase in the treatment of acute ischemic stroke were collected. After data extraction and quality assessment， descriptive analysis of the included studies was carried out. RESULTS A total of 31 articles were included， involving 28 systematic reviews/ meta-analysis and 3 pharmacoeconomic studies. In terms of effectiveness， compared with alteplase， tenecteplase （0.25 mg/kg） could significantly increase the early neurological improvement； the 90 d excellent neurological recovery rate， 90 d good neurological recovery rate， and recanalization were not inferior to alteplase. For safety， compared with alteplase， tenecteplase did not increase the incidence of hemorrhage， symptomatic intracranial hemorrhage， 3-month mortality， or intracranial hemorrhage. In terms of cost-effectiveness， foreign research results showed that tenecteplase had economic advantages over alteplase. CONCLUSIONS Compared with alteplase， tenecteplase is effective and safe in the treatment of acute ischemic stroke， and it is cost-effective.

Objective To compare and explore the differences between the eight batches of drugs in the centralized procurement list and the 2018 edition of the national essential medicine list,and to provide reference for updating and improving the national essential medicine list and the national centralized procurement list of drugs.Methods The category,generic name variety,specification,and other information of drugs included in the centralized drug procurement were collected and compared with the 2018 edition of the national essential medicine list,and the reasons for differences were analyzed.Results A proportion of 39%of centralized procurement drugs were listed in national essential medicines.Forty pharmacological classifications were not involved in the drugs of centralized procurement.Only anticoagulant and thrombolytic drugs with dual attributes accounted for a smaller variety proportion than the specification proportion.Conclusion There are some differences between the centralized procurement list and the 2018 edition of the national essential medicine list,which have some rationality,but also some problems to be solved.

Drugs under special management include narcotic drugs,psychotropic drugs,toxic drugs for medical use,radiopharmaceuticals,and pharmaceutical precursor chemicals.Supervising and guiding the clinical use of drugs under special management is one of the important responsibilities of the Pharmaceutical Management and Drug Therapy Committee(Group)of medical institutions.The standard for drugs under special management is led by the Pharmaceutical Professional Committee of the China Hospital Association,which standardizes 16 key elements of organizational management,process management,and quality control management drugs under special management in medical institutions.It can guide the standardized implementation of Pharmaceuticals under special control work in various levels and types of medical institutions.This article elaborates on the methods and contents of formulating standards for Pharmaceuticals under special management,to provide reference and inspiration for medical institutions to carry out special drug drug management and daily related work.

Objective:To evaluate the safety of a novel nanoparticle neurotrophic factor complex for intraocular application in non-human primates.Methods:Nanoparticles incorporated with ciliary neurotrophic factor (NP-CNTFs) were produced utilizing nanotechnology.Three adult male cynomolgus macaques were included and intravitreally injected with 10 μl NP-CNTFs at a concentration of 1 μg/μl into one of the two eyes, and these three eyes were designated as the NP-CNTFs group.The contralateral eyes received the same volume of phosphate buffered saline and were designated as the control group.Before the injection and on days 3 and 7 after the injection, routine clinical examinations of the anterior segment were performed to evaluate the ocular clinical symptoms such as conjunctival congestion, anterior chamber flare and cells.The fundus condition was observed by fundus photography.The morphological structure and thickness of retinas were detected by spectral domain-optical coherence tomography (SD-OCT).The use and care of animals were in accordance with the Guide for the Care and Use of Laboratory Animals issued by the National Institutes of Health and the standards of Association for Assessment and Accreditation of Laboratory Animal Care.The study protocol complied with the ethics of laboratory animal welfare and was approved by Hubei Topgene Biotechnology Co., Ltd.(No.IACUC-2019-012).Results:The NP-CNTFs prepared in this study had a particle size of (317±3)nm, a polydispersity index of 0.042±0.015, and a zeta potential of (-38.9±0.7)mV, and exhibited relatively good stability, bioavailability, and biocompatibility.Clinical examinations revealed that the clinical manifestations of conjunctival congestion, anterior chamber flare and cells were slightly more obvious in the NP-CNTFs group at 3 days after injection compared to the control group, but basically returned to normal at 7 days after injection.The scores of anterior-segment clinical symptoms of the NP-CNTFs and control group were (2.67±0.88) and (1.00±0.58) at 3 days after injection, and (0.67±0.33) and (0.33±0.33) at 7 days after injection, respectively, with no statistical differences between them ( t=2.50, 1.00; both at P>0.05).Fundus photography showed normal fundus in both groups at 7 days after injection with no abnormal changes including vitreous opacity, vitreous hemorrhage, retinal hemorrhage or papilloedema.SD-OCT showed no significant histological changes in the retinas at 7 days after injection in both groups.The retinal nerve fiber layer thickness of the NP-CNTFs and control group were (107.67±0.88) and (111.00±3.22)μm, respectively, and the macular foveal thickness of the two groups were (255.67±2.03) and (254.67±3.84)μm, respectively, with no statistical differences between them ( t=1.43, 0.50; both at P>0.05). Conclusions:The complex NP-CNTFs shows good safety for intraocular application in cynomolgus macaques.

Objective:To assess the value of double-balloon enteroscopy(DBE)in the treatment of Peutz-Jeghers syndrome(PJS)polyp with intussusception in children.Methods:A total of 14 cases with PJS polyp with intussusception were collected in Gastroenterology Department of Beijing Children′s Hospital Affiliated to Capital Medical University from July 2019 to January 2023.The general information, clinical manifestations, history of surgeries, abdominal ultrasound, enteroscopy and postoperative outcomes were retrospectively analyzed.Results:Fourteen cases with intussusception of PJS(including nine boys and five girls) were enrolled, and the average age was(10.25±2.52)years.There were 21 small intestinal intussusceptions in 14 cases, including eight cases of single intussusception and six cases of multiple intussusceptions.All the patients underwent DBE examination and treatment.A total of 215 small intestinal polyps were removed, no direct complications such as bleeding or perforation were found during the operation.Twelve cases were confirmed intussusception relieved by DBE, and the average maximum diameter of polyps causing intussusception was(4.38±1.43)cm.In addition, DBE did not reach the site of intussusception polyp in one case, and another case developed intestinal obstruction after DBE operation, which were treated by surgery.Conclusion:DBE provides a relatively effective method for the treatment of PJS polyps with intussusception without complete ileus in children.

Purpose/Significance Based on big data,a cardiovascular and cerebrovascular electronic medical record(EMR)analy-sis platform is developed.By utilizing imaging data analysis techniques and clinical document analysis techniques,the platform provides patients with precise diagnosis,treatment plans,scientific administration,prognosis prediction,smart health education prescriptions and other precise services.Method/Process The medical ontology,knowledge rules and knowledge graph for cardiovascular and cerebrovas-cular diseases are developed and constructed by using Protégé.On the basis of constructing a knowledge graph,a knowledge base for clinical diagnosis,treatment,pathological analysis and prognosis judgment of cardiovascular and cerebrovascular diseases is formed.A EMR analysis platform for cardiovascular and cerebrovascular diseases is designed based on the knowledge base.Result/Conclusion The designed cardiovascular and cerebrovascular EMR analysis platform is conducive to providing personalized diagnosis and treatment plans for different populations,and providing patients with various precise diagnosis and treatment services.

Objective:To compare the efficacy of combination therapy on cyclic vomiting syndrome(CVS)in children, and improve the efficacy of CVS treatment in the future.Methods:This study retrospectively analyzed patients′ medical records of CVS, which were admitted to Digestive Department of Beijing Children′s Hospital from 2012 to 2019.The treatment regimen was A(Cyproheptadine+ Doxepin+ Valproate), B(Propranolol+ Cyproheptadine), or C(Propranolol+ Amitriptyline). Meanwhile, the patients should take drugs more than three months.The clinical data of 42 cases were analyzed retrospectively, and the treatment effect after discharge was followed up by telephone until October, 2020.Results:Among the 42 cases, 17 were male and 25 were female, whose mean age of onset was (4.65±3.23) years, and the age of diagnosis was (6.79±3.58) years.The main accompanied symptoms were abdominal pain and upper gastrointestinal bleeding.Forty-two patients were moderate/severe CVS.The regimens A, B and C were observed in 7, 11, and 24 patients, respectively.The age at improvement was(8.17±4.12)years.The course of treatment was(1.37±0.96)years.The age at follow-up was(10.32±4.03)years.During the 1-year follow-up, 35 cases were effective, and the efficiency was 83.3%.Among them, 23 cases had no paroxysmal vomiting and 7 cases had no effect.There was no significant difference in therapy effects among group A, B and C. Between the effective group and non-effective group, there were statistical differences in the personal history of hiatus hernia( P=0.024), the weight at follow-up ( P=0.042), and the course of medication( P=0.020). Conclusion:The combination regimen has a higher effective rate in the treatment of CVS.There was no significant difference among the three regimens in the treatment of CVS.For children with refractory CVS, who can not be treated with combination therapy, individualized therapy should be further developed.

Objective:To investigate endoscopic and clinical characteristics of intestinal Behcet′s disease (BD) in children.Methods:General information, clinical manifestations, laboratory tests, imaging examinations and endoscopic characteristics of 14 children with intestinal BD treated in the Department of Gastroenterology, Beijing Children′s Hospital Affiliated to Capital Medical University from January 2016 to March 2020 were retrospectively analyzed.Results:Fourteen children with intestinal BD were recruited, involving 5 males and 9 females with the age of 1 month to 12 years and 11 months [(6.68±3.73) years old], and a median disease course of 6 months.All of them had gastrointestinal symptoms, including 11 cases with abdominal pain, 7 cases with diarrhea and 2 cases with vomiting.Complications of children with intestinal BD included gastrointestinal bleeding, intestinal perforation and stenosis.Extragastrointestinal symptoms included recurrent oral aphthosis in all the 14 cases, fever in 11 cases, skin lesions in 9 cases, vascular manifestations in 8 cases, positive pathergy test in 6 cases, joint manifestations in 5 cases, neurological manifestations in 1 case, and malnutrition in 6 cases.Observed by the endoscopy, lesions were mainly distributed in the distal ileum [61.5%(8/13 cases)]and ileocecal part[53.8%(7/13 cases)], with the main manifestation of ulcer.There were 3 cases with single ulcer and 10 cases with multiple ulcers, including 7 cases with deep and large ulcers.Pathological examinations of endoscopic lesions showed that the main features were mucosa chronic nonspecific inflammation, mucosa chronic active inflammation with ulcer and vasculitis.Conclusions:Clinical manifestations of intestinal BD vary a lot and are non-specific.Some children with intestinal BD may develop severe complications.Endoscopic lesions of intestinal BD have certain characteristics, which contribute to the diagnosis.Gastroenterologists need to be fully aware of intestinal BD.

Objective:To evaluate the clinical efficacy and safety of Infliximab (IFX) in pediatric Crohn′s disease (CD).Methods:The efficacy of IFX therapy in 30 patients suffering from active CD who were not completely improved with traditional medicine and enteral nutrition or had intolerance to the medicine in Beijing Children′s Hospital Affiliated to Capital Medical University from December 2014 to December 2019 were retrospectively analyzed.Pediatric Crohn′s Disease Activity Index (PCDAI), blood biochemistry indices, mucosal healing, nutritional status, and adverse reactions were compared and evaluated.Results:Thirty active CD cases, with 18 males and 12 females, were enrolled, and the average age was (8.63±4.76) years old.Three cases who didn′t complete 3 times of IFX injection and 1 case who lost to be followed up were excluded.A total of 26 cases of CD in active period were enrolled in this study on efficacy.The clinical remission and response rate of 26 cases were 61.5% and 84.6%, respectively, at 14-week of IFX therapy.The clinical remission and response rate of 21 cases were 71.4% and 85.7%, respectively, at 30-week.The clinical remission and response rate of 15 cases were 86.7% and 93.3%, respectively, at 54-week.At week 14 th, PCDAI score [(9.56±8.05) scores vs.(29.02±10.86) scores] decreased compared with before treatment ( t=7.339, P<0.05). The levels of erythrocyte sedimentation rate [(15.54±10.26) mm/1 h vs.(33.77±21.30) mm/1 h] and C-reactive protein [(4.79±12.94 ) mg/L vs.(16.33±23.43) mg/L] were obviously decreased, and the hemoglobin [(126.27±16.51) g/L vs.(110.58±16.45) g/L], hematocrit [(37.03±3.95)% vs.(33.52±4.32)%], and albumin levels [(42.30±3.03) g/L vs.(37.13±5.68) g/L] were remarkably increased compared with those before treatment ( t=3.932, 1.993, -3.398, -3.060, -4.009, all P<0.05). Height for age Z score and body mass index Z score were increased after IFX treatment, without statistically significant differences (all P>0.05). Conclusions:IFX therapy had good clinical efficacy in controlling inflammatorys and inducing clinical remission in pediatric CD.

X-linked juvenile retinoschisis (XLRS) is a rare X-linked inherited retinal disorder, mainly affects bilateral retina. Patients often present with visual deterioration accompanied by a spoke-wheel pattern in the macula due to splitting of inner retinal layers and a disproportionate decline in the b-wave relative to a-wave of electroretinogram. The current therapy is mainly directed toward treatment of complications with no effective clinical management yet. In recent years, with the deepening understanding of XLRS, adeno-associated virus(AAV)-mediated gene therapy has become a potential new approach for the treatment. Two clinical trials on XLRS gene therapy are currently underway. These two clinical trials assess the ocular safety and tolerability of recombinant AAV- RS1 vector and explore its safe dose in XLRS patients. However, the recovery of retinal structure and function in XLRS patients is unsatisfactory. Following the in-depth research and progress of clinical trials, it is expected that more accurate and effective treatments for XLRS patients will be provided in the future.