中医药治疗重症肌无力临床研究核心结局指标集构建研究

Study on a Core Outcome Set(COS)of Myasthenia Gravis in Clinical Trials of Chinese Medicine

目的规范选择临床研究结局指标,可以客观评价中医药治疗重症肌无力的临床疗效或效果,本研究旨在规范构建中医药治疗重症肌无力的临床研究核心指标集。方法遵循核心结局指标集研制规范(COS-STAD)开展研究,设立研究工作组,进行文献研究、患者调查,建立结局指标条目池、德尔菲法咨询小组,开展德尔菲法专家咨询及共识会议。结果中医药治疗重症肌无力临床研究核心结局指标集,包括5个指标域:终点指标、重症肌无力症状评价、用药情况评价、生活质量评价、安全性指标;9个结局指标:复发率、激素并发症发生率、危象发生率、QMGS量表(MGFA定量重症肌无力评分)、MG患者日常活动量表(ADL)、免疫抑制剂用量的分析、糖皮质激素用量的分析、胆碱酯酶抑制剂用量的分析、不良事件发生率。结论核心结局指标集包括的5个指标域和9个结局指标可作为重症肌无力临床研究疗效评价结局选项指标。

Objective To standardize the selection of clinical research outcome indicators,which can objectively evaluate the clinical efficacy or effect of traditional Chinese medicine in the treatment of myasthenia gravis.This study aims to standardize the construction of the core outcome set of clinical research of traditional Chinese medicine in the treatment of myasthenia gravis.Methods We followed the core outcome set development specification(COS-STAD)to carry out research,established a research working group,which set up a Delphi-method advisory group.Two graduate students of working group conducted a document research and meetings of patients to establishe an outcome set item pool of myasthenia gravis in clinical trials of Chinese medicine under the instruction of other members.With the questionnaire based on the content of item pool,we then carried out Delphi-method expert consultations and a consensus meeting.Results The core outcome set of clinical research on myasthenia gravis treated with traditional Chinese medicine included five outcome domains:endpoint outcome,myasthenia gravis symptom evaluation,medication evaluation,quality of life evaluation and safety outcome;Nine outcome measures:recurrence rate,incidence of hormone complications,incidence of crisis,QMGS scale(MGFA quantitative myasthenia gravis score),daily activity scale of MG patients(ADL),analysis of immunosuppressant dosage,analysis of glucocorticoid dosage,analysis of cholinesterase inhibitor dosage,and incidence of adverse events.Conclusion The five outcome domains and nine outcome measures included in the core outcome set can be used as outcome options for the efficacy evaluation of myasthenia gravis clinical research.