基于文献研究与专家论证探讨儿科风湿免疫疾病临床未被满足的需求

The Clinical Unmet Needs of Pediatric Rheumatic Immune Diseases Based on Literature Research and Expert Demonstration

目的:系统梳理儿科风湿免疫疾病临床未被满足的需求,制定《儿科风湿免疫疾病临床未被满足的需求清单》,指明当前儿科风湿免疫疾病临床存在的迫切需求,为未来开展相关研究提供理论依据。方法:(1)文献研究。检索儿科风湿免疫疾病相关文献,依据文献纳入标准获得合格文献,提炼出儿科风湿免疫疾病临床存在的需求相关问题条目。(2)专家问卷调查。制定两轮专家问卷调查表,向专家咨询关于儿科风湿免疫疾病临床需求的意见,并对回收的问卷进行汇总处理,对数据进行统计分析,形成《儿科风湿免疫疾病临床未被满足的需求清单》初稿。(3)专家论证。提交《儿科风湿免疫疾病临床未被满足的需求清单》初稿,召开专家论证会,商议、讨论初稿并提出相关问题、意见,修改后形成清单终稿。结果:(1)文献研究。纳入系统性红斑狼疮、抗磷脂综合征、幼年特发性关节炎、强直性脊柱炎、系统性血管炎等24种儿科风湿免疫疾病,数据库检索共获得中文、英文文献198012篇,经筛选后最终纳入35038篇文献。(2)问卷调查。两轮专家问卷调查中,专家认为川崎病、过敏性紫癜、幼年特发性关节炎、系统性红斑狼疮及糖皮质激素诱导的骨质疏松症为儿童最为常见的5种风湿免疫疾病。在儿科风湿免疫性疾病常见临床问题中,形成13个临床需求清单条目及5个疾病临床需求条目。(3)专家论证。专家经过商讨,最终形成了《儿科风湿免疫疾病临床未被满足的需求清单》终稿。结论:形成《儿科风湿免疫疾病临床未被满足的需求清单》。中医药治疗儿科风湿免疫疾病相关指南/专家共识、儿科流行病学研究、高级别RCT研究缺乏;中医治疗的优势环节、切入点、应用时机不明确;儿科专用剂型缺乏、剂型规格单一,中成药临床应用指征不明确。

Objective:The unmet needs of pediatric rheumatic immune diseases were systematically reviewed,and the List of Clinical Unmet Needs of Pediatric Rheumatic Immune Diseases was formulated,indicating the urgent clinical needs in the field of pediatric rheumatic immune diseases,so as to provide theoretical basis for future relevant research.Methods:In the iiterature study,we searched the literature related to pediatric rheumatic immune diseases,got qualified documents according to the literature exclusion standard and extracted the items related to the clinical needs of pediatric rheumatic immune diseases.In the expert questionnaire survey,we developed two rounds of expert questionnaire,and got experts expert advice on the clinical needs of pediatric rheumatic immune diseases,to perform a summary processing of the collected questionnaires.According to statistical analysis of the data,a draft List of Clinically Unmet Needs for Pediatric Rheumatic Immune Diseases was formed.In the expert argumentation,we submit the first draft of the List of Clinical Unmet Needs of Pediatric Rheumatic Immune Diseases,held an expert discussion meeting to discuss the first draft,and proposed relevant issues and opinions.The final draft of the list was formed after modification.Results:In the literature study,24 kinds of pediatric rheumatic immune diseases such as systemic lupus erythematosus,antiphospholipid syndrome,juvenile idiopathic arthritis,ankylosing spondylitis,systemic vasculitis,etc.The database search obtained 198012 articles in Chinese and English,and after screening,35038 articles were finally included.In the questionnaire survey,experts believed that pediatric Kawasaki disease,allergic purpura,juvenile idiopathic arthritis,systemic lupus erythematosus and glucocorticoid induced osteoporosis were the most common rheumatic immune diseases in children,after two rounds of expert questionnaire survey.Among the common clinical problems of pediatric rheumatic immune diseases,13 clinical demand list items and 5 disease clinical demand items were formed.In the expert demonstration,experts finally formed the List of Clinical Unmet Needs of Pediatric Rheumatic Immune Diseases after discussion.Conclusion:The List of Clinical Unmet Needs of Pediatric Rheumatic Immune Diseases was formed.There is a lack of guidelines/expert consensus for the treatment of pediatric rheumatic immune diseases,pediatric epidemiological research and high-level RCT research.The advantages,entry points,and application timing of TCM are unclear.There is a lack of special dosage form for pediatrics,with single dosage form and specification,and unclear indication for clinical application of Chinese patent medicine.