Bergmann J F, Chassany O
Therapeutics Research Unit, Lariboisière Hospital, Paris, France.
Eur J Surg Suppl. 1998(583):87-91. doi: 10.1080/11024159850191319.
Generic questionnaire such as SF36 and functional gastrointestinal disorder (FGD)-specific questionnaires (i.e. IBSQOL or FGDQL) are now available for the evaluation of new drugs for the treatment of irritable bowel syndrome or functional dyspepsia. For regulatory issues it is necessary to describe in detail the scale of the QOL questionnaire, its psychometric properties and reason for its choice, as well as validation studies, populations concerned, means of administration and methods for analysis. Preselection of few dimensions of the QOL questionnaire, if necessary, has to be explained. QOL results have to be compared with symptom scores. Trials have to be double-blind placebo- controlled studies with a sample size calculation taking account of the high placebo-response. No official guidelines are available in FGD and more validation studies are needed to help in the choice of the specific QOL questionnaire, in the metrologic analysis and in the interpretation of QOL changes during FGD treatment. Then QOL questionnaires could become a primary end point in clinical trials if efficient drugs for FGD treatment are developed.
现在有通用问卷（如SF36）和功能性胃肠疾病（FGD）特异性问卷（即IBSQOL或FGDQL）可用于评估治疗肠易激综合征或功能性消化不良的新药。出于监管问题的考虑，有必要详细描述生活质量问卷的量表、其心理测量特性及其选择理由，以及验证研究、相关人群、施测方式和分析方法。如有必要，必须解释对生活质量问卷少数维度的预筛选。生活质量结果必须与症状评分进行比较。试验必须是双盲安慰剂对照研究，样本量计算要考虑到高安慰剂反应率。FGD领域尚无官方指南，需要更多验证研究来帮助选择特定的生活质量问卷、进行计量分析以及解释FGD治疗期间生活质量的变化。如果开发出有效的FGD治疗药物，那么生活质量问卷可能会成为临床试验的主要终点。
