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.
The National Comprehensive Cancer Network (NCCN) released the latest version 1, 2022 of "NCCN guidelines for the clinical diagnosis and treatment of small cell lung cancer" (hereinafter referred to as "guideline"). Based on high-quality evidence-based medicine, this guideline provides references of clinical diagnosis and treatment for clinicians around the world. Compared with the version 3, 2021 of the "guideline", updates and revisions mainly focused on the progress of radiotherapy and systemic treatment. This article will interpret the updated therapy content in this new version of the "guideline".
By employing the nominal group technique, as per the process standard of the EQUATOR Collaboration Network, experts were selected through purposeful sampling. Two rounds of nominal group discussions were conducted, and the essential information of the utilization of the consensus method was extracted from the literature. After comparison, discussion, evaluation, and optimization, a list of 3 fields, 11 themes, 63 necessary items, and 28 supplementary items was eventually constructed to upgrade the standardization and rigor of the application of the consensus method in the future, assisting guideline developers to plan the consensus process.
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.
Strengthening the management and evaluation of clinical pathways is one of the most important strategies of "Healthy China 2030" Strategic Plan. Evidence-based assessment and clinical guidelines can provide the best relevant evidence to develop clinical pathways. We planned to analyze the current situation of clinical pathways in China and explore how to apply evidence-based assessment on clinical pathway management. We searched PubMed, EMbase, ISI, CNKI, WanFang Data, VIP and the The Cochrane Library using "critical pathways" and "clinical guidelines" as key words or subject terms. And we conducted a comparison of their published volume, definitions, differences and connections. The management system of clinical pathway in China is fundamentally flawed, it is still a challenge to implement the clinical pathways effectively without scientific methodologies and standardized evidence-based evaluation system. In order to improve the management quality of clinical pathway in China, we should develop clinical pathways based on national situation and innovate the evaluation system to standardize the clinical pathway management according to WHO recommendations of clinical guideline and appraisal.
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.
The UK's National Institute for Health and Care Excellence (NICE) published guideline of mental wellbeing at work on March 2, 2022. The guideline covers how to create the right conditions for mental wellbeing in the workplace, with the aim of promoting supportive and inclusive workplace environments and helping people with or at potential risk of mental health problems. This review will interpret the guidelines in detail.
With the recent domestic adoption of novel formulations such as high-dose anti-vascular endothelial growth factor agents and gene therapy drugs, sterile intraocular inflammation (SIOI) following intravitreal injection has gradually come into public awareness and garnered widespread attention. Concurrently, multiple ophthalmic medications with established clinical histories, including prophylactic antibiotics (e.g., vancomycin) and corticosteroids (e.g., triamcinolone acetonide), have also been reported to induce SIOI. To enhance Chinese ophthalmologists’ understanding of SIOI and standardize its diagnostic and therapeutic protocols, the Fundus Disease Group of Ophthalmological Society of Chinese Medical Association, Fundus Disease Group of Ophthalmologist Branch of Chinese Medical Doctor Association, adhering to evidence-based medicine principles and integrating international guidelines and consensus documents with China’s socio-economic context, have spearheaded the development of the Expert consensus for the diagnosis and treatment of sterile intraocular inflammation after intraocular drug delivery (2025) through rigorous consensus-building processes. This consensus systematically presents diagnosis and treatment recommendations with Chinese characteristics, addressing seven key clinical issues such as the epidemiological features, clinical manifestations, identification of high-risk factors, perioperative management, and prognosis evaluation of SIOI. It focuses on constructing a hierarchical intervention system based on the severity of the disease. An in-depth understanding of the core content of this consensus can, on the one hand, help avoid diagnostic and therapeutic deviations caused by insufficient understanding, and on the other hand, assist in establishing a standardized SIOI management process, thereby effectively reducing the risk of visual impairment and optimizing the visual prognosis of patients. The introduction of this consensus marks the further improvement of the quality management system for intraocular injection therapy in our country and holds significant guiding importance for enhancing the diagnostic and therapeutic level of retinal diseases.
Working Group on Extracorporeal Life Support, National Center for Cardiovascular Quality Improvement developed guidelines on patient blood management for adult cardiovascular surgery under cardiopulmonary bypass, aiming to standardize patient blood management in adult cardiovascular surgery under cardiopulmonary bypass, reduce blood resource consumption, and improve patients outcomes. Forty-eight domestic experts participated in the development of the guidelines. Based on prior investigation and the PICO (patient, intervention, control, outcome) principles, thirteen clinical questions from four aspects were selected, including priming and fluid management during cardiopulmonary bypass, anticoagulation and monitoring during cardiopulmonary bypass, peri-cardiopulmonary bypass blood product infusion, and autologous blood infusion. Systemic reviews to the thirteen questions were performed through literature search. Recommendations were drafted using the GRADE (Grading of Recommendations Assessment, Development and Evaluation) system. After five rounds of experts discussions between 2023 and 2024, 19 recommendations were finally formed.