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.
Based on new clinical evidence, the National Comprehensive Cancer Network (NCCN) annually updates and releases the "NCCN Guidelines for the Clinical Diagnosis and Treatment of Non-Small Cell Lung Cancer" which has become the reference for clinical diagnosis and treatment approved and complied by clinicians worldwide. On November 25, 2020, the latest 2021 V1 version of "NCCN Clinical Diagnosis and Treatment Guidelines for Non-Small Cell Lung Cancer" (hereinafter referred to as "Guidelines") was released. Compared with the 8th edition of the "Guidelines" in 2020, many updates focused on the progress of targeted and immunotherapy. This article will provide the interpretations of the updated therapy content of this edition of the guidelines.
Breast cancer, the most common malignancy in the world, also causes the most death cases of women among malignancies. Breast cancer risk reduction guidelines (version 2023) was updated by National Comprehensive Cancer Network (NCCN). Based on high-level evidences from evidence-based medicine and the latest research progress, the guidelines provided standardized guidance for breast cancer risk assessment and risk reduction strategies for individuals without a history of invasive breast cancer or ductal carcinoma in situ, which has attracted widespread attention from clinicians worldwide. Breast cancer is also the most common malignancy in Chinese women, and the number of newly diagnosed breast cancer cases each year in China ranks first in the world due to the large population, so the breast cancer prevention has become a major public health challenge in China. Aimed to provide reference for breast cancer prevention in China, this article interpreted the guidelines (the new version) based on the characteristics of breast structure in Asian women and the epidemiological characteristics of breast cancer in China.
Surrogate endpoints, defined as biomarkers or intermediate outcomes utilized in clinical trials to replace the ultimate targeted outcomes, have witnessed a growing prevalence in both clinical trials and drug-device approvals in recent years. To standardize the application and reporting of surrogate endpoints in clinical trial protocols and associated studies, relevant scholars published the SPIRIT-Surrogate and CONSORT-Surrogate reporting guidelines in the BMJ in July 2024. This article provides an interpretation of these guidelines in conjunction with published case studies, with the aim of offering references for domestic researchers, elevating the overall quality of related clinical trials, and eventually facilitating the enhancement of domestic healthcare level.
In order to help Chinese guideline developers, clinicians, health policy makers and other relevant researchers fully understand and make appropriate use of World Health Organization (WHO) guidelines, Chinese GRADE Center and Guidelines Review Committee of World Health Organization (WHO-GRC) have written a series of papers about development methods, review principles and the structure and content of WHO guidelines. This is the third (also last) paper which interpreted Consolidated Guidelines on the Use of Antiretroviral Drugs for Treating and Preventing HIV Infection and Guidelines for the Screening, Care and Treatment of Persons with Hepatitis C Infection as examples with the process and steps of WHO guideline development.
Hip osteoarthritis (OA) is a significant disease worldwide that causes functional impairment and reduced quality of life. With the aging of the population and the increasing prevalence of obesity, its disease burden is becoming increasingly heavy. In December 2023, the American Academy of Orthopaedic Surgeons (AAOS) released the updated Clinical Practice Guideline for the Management of Osteoarthritis of the Hip (summary). Based on evidence-based medicine, this guideline provides systematic recommendations for the diagnosis and treatment of hip OA. This article will comprehensively interpret the guideline, compare it with relevant domestic expert consensus in China, analyze the consistency and differences in disease management strategies between the two, and provide references for clinical practice.
Breast cancer is one of the most common malignant tumors in women, and its treatment and management strategies are crucial for improving patients’ prognosis and quality of life. Early breast cancer refers to lesions confined to the breast and regional lymph nodes (N1 stage) without distant metastasis. Thanks to the improvement of screening techniques, the detection rate of early breast cancer has increased. The “early diagnosis and early treatment” model has led to a decrease in breast cancer mortality, especially among young women. The 2024 European Society for Medical Oncology (ESMO) clinical practice guideline for the diagnosis, treatment, and follow-up of early breast cancer aims to provide comprehensive and standardized recommendations for the diagnosis and treatment of early breast cancer, helping clinicians make optimal decisions. This article interprets the main content of the guideline in order to provide references and assistance for the current clinical diagnosis and treatment of early breast cancer in China.
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.
