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.
Peripheral artery disease (PAD) of the lower extremities, which poses a major challenge in the field of global public health, has seen a rising trend in its incidence and disability rate year by year. With the continuous innovation of new diagnostic techniques, imaging evaluation methods, and treatment strategies, profound changes have taken place in the diagnosis and treatment paradigm in this field. Based on the “European Society for Vascular Surgery (ESVS) 2024 Clinical Practice Guidelines on the Management of Asymptomatic Lower Limb Peripheral Arterial Disease and Intermittent Claudication” issued by the European Society for Vascular Surgery in 2024, we systematically reviewed the relevant international guidelines in recent years and conducted horizontal comparisons. Combining with the latest clinical research evidence, we conducted an in-depth analysis from the perspective of evidence-based medicine on the strategic evolution, technical key point updates, and clinical evidence levels of endovascular treatment for lower extremity PAD. The aim is to provide an evidence-based medical basis for clinical decision-making.
ObjectivesTo evaluate the reporting quality of clinical practice guidelines published in Chinese journals in 2017.MethodsCBM, CNKI and WanFang Data databases were searched for articles published in 2017. Two reviewers independently screened literature, extracted data, and evaluated the reporting quality of clinical practice guidelines using the Reporting Items for Practice Guidelines in Healthcare (RIGHT).ResultsOne hundred and seven clinical practice guidelines were included and a total reporting rate of 34.8%±0.1% in RIGHT. Among the seven domains of RIGHT, field on basic information had the highest reporting rate (56.8%) and fields on review and quality assurance had the lowest reporting rate (9.3%).The average reporting rate of RIGHT items of Chinese Science Citation Database (CSCD) articles was lower than non-CSCD [MD=?0.73, 95%CI (?0.78, ?0.68)] articles. The average reporting rates of RIGHT items differed between Chinese Medical Association (CMA) journal articles and non-CMA journal articles [MD=2.30, 95%CI (2.26, 2.34)]. The average reporting rates of RIGHT items was lower in guidelines established by associations or institutes [MD=?3.78, 95%CI (?3.83, ?3.73)], and was higher reported in Chinese medicine guidelines [MD=21.94, 95%CI (21.91, 21.97)].ConclusionsThe reporting quality of clinical practice guidelines published in journals of mainland China in 2017 is low in general, especially in fields such as review and quality assurance, funding and declaration and management of interests and other information. To improve this phenomena, it is suggested that guideline developers report the guidelines rigorously with international standard.
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.
Peritoneal dialysis (PD) is widely used to treat acute kidney injury (AKI) in low-resource and higher income countries. This paper summarizes the key points and improvements of the 2020 International Society for Peritoneal Dialysis guidelines in five aspects of outcomes for AKI treatment, peritoneal access, dialysis solutions, prescription of dialysis with targets of solute clearance and complications, so as to provide references for AKI in clinical practice.
The utilisation of statistical analysis plan (SAP) has the potential to enhance the reliability, transparency, and impartiality of statistical analysis procedures in the context of clinical studies. These plans are primarily designed for late phase clinical studies, namely phase Ⅱ and phase Ⅲ randomised controlled trials. The extended SAP reporting guidelines for early phase clinical studies, i.e., phase Ⅰ clinical studies and phase Ⅱ non-randomised controlled trials, have been expanded from the original reporting guidelines in six key areas: trial purpose, design, Bayesian statistics, data simulation, sample size, and the application of ICH E9 (R1). The expanded reporting guidelines facilitate the standardisation of SAP for early phase clinical trials, enhance the transparency and reproducibility of early phase clinical studies, and thereby improve the quality of early phase clinical studies. This, in turn, plays a pivotal role in later phase clinical studies.
ObjectiveTo evaluate the quality of traditional Chinese medicine (TCM) guidelines, so as to provide evidence for developing similar guidelines. MethodsWe systematically searched TCM guidelines published in domestic medical journals from WanFang Data, VIP, CNKI and CBM. We also searched Google, Amazon.cn and dangdang.com by hand in order to obtain TCM guidelines published in monographs. And we used AGREE II instrument to assess the methodological quality of included guidelines. ResultsA total of 115 guidelines (87 published in domestic medical journals and 28 published in monographs) were included. Scaled domain percentages from highest to lowest were:scope and purpose (41%), stakeholder involvement (28%), clarity of presentation (33%), rigour of development (20%), editorial independence (9%) and applicability (7%). ConclusionThere is still a large gap about methodological quality between TCM guidelines and international guidelines. So the guideline developers should systematically search, evaluate and synthesize the evidence based on structured problems. At the same time, they also need to consider the costs of recommendations, the implementation environment and the patients' preference and values. On this basis, the developer could further increase the transparency and independence during developing TCM guidelines.
This study was an interpretation study based on the standard of AGREEⅡ. It analyzed methodological perspective of the International Evidence-Based Recommendations for Focused Cardiac Ultrasound determined by the International Conference on Focused Cardiac UltraSound (IC-FoCUS).