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.
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.
Diffuse large B-cell lymphoma is highly heterogeneous and is diagnosed according to the 2016 World Health Organization Classification of Tumours of Haematopoietic and Lymphoid Tissues. The decision of treatment should be upon age, International Prognostic Index score and the tolerability of chemotherapy. High-dose chemotherapy and autologous stem cell transplantation is the standard care for relapsed, chemotherapy sensitive patients. Clinical trials are recommended in specific conditions.
Neuromuscular disease (NMD) encompasses a group of disorders that affect motor neurons, peripheral nerves, neuromuscular junctions, and skeletal muscles, potentially leading to respiratory muscle impairment and decline in respiratory function, significantly impacting patients' quality of life. In March 2023, clinical practice guideline titled Respiratory Management of Patients with Neuromuscular Weakness was released by the American College of Chest Physicians. This article summarizes, categorizes, and interprets the contents and key points of the guideline, aiming to provide more targeted guidance for clinical healthcare professionals and ultimately enhance the effectiveness of respiratory management for patients with NMD.
Objective To evaluate the methodological quality and reporting quality of clinical guidelines and consensus on central venous catheters. Methods The PubMed, EMbase, Web of Science, CBM, WanFang Data, CNKI databases and Guidelines International Network, National Institute for Health and Clinical Excellence, National Guideline Clearinghouse, Medive.cn websites were searched to collect clinical guidelines and consensus related to central venous catheters. The retrieval time was from the establishment of the database to October 2022. Two researchers independently screened the literature, extracted data and used evaluation tools AGREE Ⅱ and RIGHT to evaluate the quality of the included studies. Results A total of 34 central venous catheter guidelines and consensus were included. The average score for each field of AGREE II was 53.73% for scope and purpose, 39.26% for participants, 39.57% for rigor, 46.76% for clarity, 30.23% for application and 49.18% for editorial independence. Items 1a, 1b, 3, and 4 (100.00%) had the highest reporting rate in the RIGHT evaluation items, followed by items 19a (97.05%), 2/19b (94.11%), 20 (91.17%), 7b/11a (88.23%), and 7a (85.29%). The reporting rate of the remaining items was below 60%. Subgroup analysis results showed that the average score and RIGHT score of the guidance class in the four fields of AGREE Ⅱ (rigor, clarity, application and editorial independence) were higher than those of the consensus class. Guidelines and consensus formulated based on evidence-based medicine methods were higher than those formulated based on expert opinions or reviews in the three fields of AGREE II (rigor, application and editorial independence). The average scores of foreign guidelines and consensus in 6 fields and RIGHT scores of AGREE Ⅱ were higher than those of domestic guidelines and consensus. Conclusion The AGREE Ⅱ of 6 fields average score and RIGHT score in foreign guidelines are higher than those in domestic guidelines.
ObjectiveTo evaluate the reliability and validity of the instrument of clinical applicability of guidelines (version 2.0). MethodsThe experts of domestic medical institutions were investigated by questionnaire, and the instrument of clinical applicability of guidelines (version 2.0) were evaluated the guidelines for the diagnosis and treatment of tinea mantis and tinea pedis (revised edition 2017) and the guidelines for the diagnosis and treatment of cerebral hemorrhage in China (2019). Using Cronbach's α coefficient and Spearman-Brown coefficient to evaluate the inherent reliability and split-half reliability. The content validity was evaluated by calculating the content validity index of the item level and the adjusted Kappa value. The correlation coefficient between each item and the dimension and the hypothesis test were used to evaluate the convergent and discriminant validity. The structural validity was evaluated by using structural equation model to evaluate the structural validity of the tool. ResultsThe Cronbach's α coefficient and Spearman-Brown coefficient of the instrument of clinical applicability of guidelines (version 2.0) were both greater than 0.7, the content validity index (S-CVI/Ave) were more than 0.8, the success rates of convergent were 100%, and the success rates of discriminant validity calibration were 100% and 96%. In the second-order confirmatory factor analysis model, the χ2/ df were less than 3, the fitting index (CFI), the goodness of fit index (GFI) and the adjustment goodness of fit index (AGFI) were all greater than 0.9. The root mean square residual (RMR) were all less than 0.05, and approximate error root mean square (RMSEA) were less than 0.09. The P value of RESEA hypothesis test were more than 0.05. ConclusionThe instrument of clinical applicability of guidelines (version 2.0) has good reliability and validity, which can be further verified in practical application in the future.
Objective To explore the methods used for developing evidence-based clinical practice guidelines for acupuncture. Methods Based on the characteristics of acupuncture in traditional Chinese medicine,and principles of evidence-based medicine, this article introduces and summarizes the processes and methods for developing an evidence-based clinical practice guideline for acupuncture. We analyzed similarities and differences between clinical practice guidelines for acupuncture and for other interventions. We used an evidence-based clinical practice guideline of acupuncture for depression as an example to illustrate the methods of literature search, grading of evidence and recommendations, evidence evaluation and consensus formation. Results Preliminary recommendations on the methods for developing evidence-based clinical practice guidelines for acupuncture were made. Conclusion Based on the optimized rational methodology for developing clinical guidelines, evidence-based high-quality clinical practice guidelines for acupuncture could be established.
Glucocorticoids are the first-line therapy of many neurological disorders, with the treatment regimen varying across types and characteristics of neurological disorders. This article reviews the national and international guidelines and expert consensuses in the past decade on glucocorticoids treatment for neurological disorders, and summarizes recommendations from the latest Chinese guidelines and consensuses. In summary, the most frequently used pulse therapy of glucocorticoids in China is intravenous infusion of high-dose (usually 1000 mg/d) methylprednisolone in a short period (often <5 d), followed by gradual tapering, bridging with oral prednisone or direct discontinuation. The treatment regimen for children and juveniles is similar to that for adults but the dose is adjusted by body weight. Pharmacodynamics of glucocorticoids should be considered for the treatment of perinatal women. To provide appropriate glucocorticoids treatment for patients with neurological disorders, clinicians should fully understand features of each neurological disorder and clinical characteristics of individual patient.
Objective To improve care and outcomes for all migraine suffers, the USHC created these evidence-based guidelines for migraine headache. Methods Firstly, 5 relative Technical reviews were done according to the Methods used in the AHCPR Technical Reviews. Secondly, based on the results of the 5 technical reviews, the 4 treatment guidelines were developed in direction of the USHC’S Methods used in developing clinical guidelines. Results Evidence supporting the acute treatment and preventive treatment were exclusively Class 1 studies, evidence supporting the diagnostic testing were either Class 2 or Class 3 studies , only very few expert judgment was given on some compelling issues without evidence. The recommendations they supporting were high-qualified, middle-qualified, and poor-qualified respectively. Conclusion This Evidence-Based Guidelines is one of the first and most extensive cooperative projects available for creating guidelines. The guideline was developed with systematical and scientific methods and stroven to base all of its recommendations on evidence.