Inherited retinal diseases (IRDs) are a group of severe retinal degenerative diseases leading to permanent visual impairment. IRDs are the major cause of irreversible blindness in children and working age groups. Gene therapy is a new clinical treatment method and currently the only clear and effective treatment for IRDs, while, there are still risks in clinical research and application. How to standardize perioperative management and reduce the potential risks of treatment is one of the keys to ensure the safety and effectiveness of treatment. However, there is no systematic and standardized guidance on the perioperative management for IRDs gene therapy. Therefore, in order to standardize the perioperative management, the Fundus Disease Group of Ophthalmology Society of Chinese Medical Association and Chinese Medical Doctor Association organized domestic experts to put forward standardized opinions on the perioperative management of IRDs gene therapy in China after repeated discussion and combined with domestic and foreign research experience, so as to provide clinicians with reference and application in clinical research and practice.
Rapid development of recently emerging precision medicine techniques represented by gene therapy has brought hope for the treatment of rare blinding eye diseases such as inherited retinal diseases (IRDs) for which there was no effective treatment previously. Although the globally growth of clinical trials for IRDs has increased rapidly over the past decade, due to the highly genetic and clinical phenotypic heterogeneity, as well as limited data on epidemiology and natural history of the disease, along with severe loss of vision function of majority patients for which the established measurements may not be appropriate, such studies lack standard outcome measurements and endpoints to asses clinical meaningful effectiveness, posing great challenges in terms of study design and evaluation of treatment efficacy, as well as clinical practice application. At present, there is no systematic nor standardized guidance on safety measures, clinical outcomes and endpoints of visual function for clinical trial design in IRDs. Therefore, in order to standardize the validated evaluation of IRDs clinical efficacy outcome measurements and endpoints, the Fundus Disease Group of Chinese Medical Association Ophthalmology Branch and Fundus Disease Committee of Chinese Medical Doctor Association Ophthalmology Branch organized domestic experts to put forward consensus and recommendations on standardizing outcome measurements and endpoints for clinical study design in IRDs, aiming to advance the study design of IRDs natural history research and clinical trials and to effectively evaluate disease progression and intervention efficacy. Along with the development of medical science and clinical trials, relevant content will be improved and updated accordingly.
An 89 years old male patient was admitted to the First Medical Center of Chinese PLA General Hospital due to chest tightness and shortness of breath for half a month. Severe aortic valve stenosis was found in post admission assessment. We proposed to perform transcatheter aortic valve replacement surgery. Preoperative evaluation showed severe distortion of the descending aorta. We used the double guide wire technique and a 14F long sheath to assist the stepwise balloon expansion, and successfully completed the valve implantation. For patients with severe tortuous aorta, how to successfully complete transcatheter aortic valve replacement, this case may provide some reference.
ObjectiveTo compare the similarities and differences in procedures between Western medicine (WM) and traditional Chinese medicine (TCM) guidelines. MethodsWe systematically searched China National Knowledge Infrastructure, WanFang Data, China Biology Medicine disc and PubMed from inception to May 17, 2022. Supplementary search of reputable medical institutions and organizations, as well as retrospective references were conducted and screened. We extracted and analyzed the basic information and guideline development procedure of the included handbooks. ResultsA total of 10 handbooks published from 2013 to 2022 were included, with four WM handbooks and six TCM handbooks. The median completeness of handbooks was 53.3%, with a maximum of 93.3% and a minimum of 43.3%. There are six handbooks with less than 60.0% completeness. The median reporting rate of the key steps was 65.0%, and the reporting rate of 5/8 of the key steps was more than 80.0%. Among the key reporting steps, role of funders, update methods, and conflict of interest management were reported at a low rate. Compared with WM handbooks, TCM handbooks reflected TCM characteristics in the procedure of topic selection, working groups, conflict of interest declaration and management, clinical questions, evidence, recommendations, report guideline, and external review. ConclusionThe completeness and reporting rate of the key steps need to be improved. TCM characteristics need to be further integrated when developing TCM guideline handbooks. We highly recommend that guideline stakeholders actively participate in handbook development to promote the quality of handbooks.
In the formulation of the clinical question of traditional Chinese medicine clinical practice guidelines, even if the intervention elements (intervention or control) have an appropriate scope, guideline developers are still faced with a variety of interventions. By analyzing the difficulty and necessity of priority selection of intervention interventions, we propose the approach of extending expert evidence to the process of priority selection of intervention interventions, and further provide the methodology of expert evidence data collection table design, application, data presentation and expert decision-making method to provide references and guidance for guideline developers.
ObjectiveTo evaluate the efficacy and safety of a novel wire for echo-guiding percutaneous atrial septal defect (ASD) closure.MethodsTwenty sheep were randomly divided into two groups, an experimental group and a control group (n=10 in each group). In both groups, an animal model of ASD was established by transthoracic balloon dilatation, and percutaneous closure of atrial septal defect was finished only by echography guiding. The total procedure time, passing time (time needed for the guide wire to enter the left atrium), frequency of delivery sheath dropping into right atrium, frequency of arrhythmias and rate of complications were compared between the two groups.ResultsASD model was uneventfully established in all animals. ASD devices were successfully implanted in all sheep. Compared with the control group, the experimental group had significantly less mean procedure time and mean passing time, lower frequency of guide wire dropping into right atrium and frequency of atrial and ventricular premature contractions, respectively (15.36±4.86 min vs. 25.82±7.85 min, 2.69±0.82 min vs. 5.58±3.34 min, 0% vs. 40.0%, 4.41±2.61 vs. 9.60±3.68, 0.75±0.36 vs. 1.34±0.68; all P<0.05) . Two groups did not have serious complications within postoperative one week.ConclusionThe novel wire system is not only safe and effective in the treatment of ASD occlusion, but also easy to operate.
The diagnosis and management of congenital heart disease (CHD), the most common inborn defect, has been a tremendous success of modern medicine. With the development of diagnostic techniques, surgical procedures and interventional techniques, more than 90% of CHD children can survive to adulthood. Consequently, the prevalence of patients with CHD has shifted away from infancy and childhood towards adulthood. Adult CHD cardiology is now encompassing not only young or middle-aged adults but also patients aged above 60 years. Standardized guidelines can provide good theoretical support for the comprehensive management of adult CHD. Ten years after the European Society of Cardiology guidelines for the management of grown-up CHD released in 2010, the new version was officially released in August 2020. The new version of guidelines updated the classification and stratification of diseases, comprehensive intervention methods and intervention timing, and put forward some new concepts, new intervention standards and methods. For adult CHD that has not been repaired or needs to be repaired again, the indication and mode of surgical intervention and perioperative management have a great impact on the prognosis. The new version of the guidelines provides a detailed description of the surgical and intervention indications and methods for different diseases, and clarifies the management methods for high-risk groups. This article attempts to interpret this newly updated guideline from the perspective of a surgeon, sort out several key diseases introduced by the guideline, and strives to provide a concise and actionable guideline for domestic counterparts.
There is an increase in published studies relevant to the development of patient version of guidelines (PVGs). We reviewed, summarised and analysed the current developments in this field, with the aim of informing domestic scholars of the factors to consider when developing quality PVGs. We proposed three recommendations: (1) the concept of patient guidelines needs to be better defined; (2) a platform for the dissemination of PVGs should be established to support their implementation; and (3) a standardised PVGs development methodology should be developed to ensure the quality of the PVGs.
Based on previous evidence-based researches and teaching experience, our team conducted literature and book review, and summarized 4 requirements, 1) effect measure calculation and conversion, 2) registration of evidence-based research, 3) evidence-based research database and 4) quality evaluation tools and reporting guidelines. We developed an online platform of evidence-based medicine research helper using the front-end and back-end technology, which can be accessed using www.ebm-helper.cn. Currently, the online tool has included 46 scenarios for effect measure calculation and conversion, introduction of 7 evidence-based research registration platforms, 26 commonly used databases for evidence-based research and 29 quality evaluation tools and reporting guidelines. This online tool can help researchers to solve specific problems encountered in different stages of evidence-based medicine research. Promoting the application of this platform in evidence-based medicine will help researchers to use the tool scientifically and improve research efficiency.