Validating endpoints for therapeutic trials in fecal incontinence.
Validating endpoints for therapeutic trials in fecal incontinence.
复制标题
验证粪便失禁治疗试验的终点。
DOI：
10.1111/nmo.12809
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发表时间：
2016-08
影响因子：
3.5
通讯作者：
Bharucha AE
中科院分区：
文献类型：
--
作者：
Noelting J;Zinsmeister AR;Bharucha AE
每天的大便日记记录的大便失禁(FI)频率减少50%或更多是FI临床试验的主要终点。这种差异在临床上是否重要尚不得而知。用每日仪器和每周仪器记录的FI症状之间的关系尚不清楚。心理因素对FI患者生活质量(QOL)的影响尚不清楚。对44例FI妇女在接受安慰剂或可乐定双盲随机治疗前和期间，用每日肠道日记和定期问卷(大便失禁严重程度评分[FISS]、FIQOL、36项简明健康调查[SF-36]和医院焦虑和抑郁量表)评估FI的严重程度。FI频率的减少与FISS计算的最小临床重要差异(MCID)进行了比较。比较每天和每周日记中FI的终点。在FI频率下降50%~74%和≥下降75%的患者中，FISS超过MCID的比例分别为75%和83%。每日仪器和每周仪器测量的FI参数之间存在显著相关性。每日参数可解释FISS患者间差异的71%。SF-36健康评分，而不是FISS评级，解释了FIQOL中主体间差异的大部分。报告≥使FI频率减少50%的大多数患者都经历了临床上重要的改善。每周问卷调查准确评估FI的严重程度。自我报告的身心健康状况解释了FIQOL的差异，而不是FI症状的严重性。
A 50% or greater reduction in the frequency of fecal incontinence (FI) recorded with daily bowel diaries is the primary endpoint in clinical trials of FI. Whether this difference is clinically important is unknown. The relationship between FI symptoms recorded with daily and weekly instruments is unknown. The contribution of psychological factors to quality of life (QOL) in FI is unclear. FI severity was assessed with daily bowel diaries and periodic questionnaires (Fecal Incontinence Severity score [FISS], FIQOL, 36-Item Short Form Health Survey [SF-36], and Hospital Anxiety and Depression scales) for 4 weeks before and during double-blind randomization to placebo or clonidine in 44 women with FI. The reduction in FI frequency was compared to the minimal clinically-important difference (MCID) computed from the FISS. Endpoints of FI were compared between daily and weekly diaries. The FISS exceeded the MCID in 75% and 83% of patients in whom the FI frequency declined by 50%-74% and ≥75% respectively. Parameters of FI measured with daily and weekly instruments were significantly correlated. The daily parameters explained 71% of the inter-patient variation in the FISS. The SF-36 health scores, rather than the FISS rating, explained a majority of the inter-subject variation in FIQOL. Most patients who report a ≥50% reduction in FI frequency experience a clinically-important improvement. Weekly questionnaires accurately assess the severity of FI. Self-reported physical and mental health explained a greater proportion of the variance in FIQOL than FI symptom severity.