BACKGROUND Patients with chronic constipation (CC) or with irritable bowel syndrome with constipation are often dissatisfied about their medical therapy, but their condition remains poorly defined. AIM To evaluate the patients' satisfaction rates and which factors predict favourable outcomes through the aggregate analysis of N-of-1 trials. METHODS Eighty-one outpatients with CC or with irritable bowel syndrome with constipation underwent N-of-1 trials with at least a one-month cycle of effective treatment. Three primary endpoints (satisfaction with therapy, improvement after treatment and an extended satisfaction criterion including both endpoints) were adopted to define satisfaction with therapy. Dyssynergia, resting anal pressure, colonic transit time and somatisation were assessed. The Patient Assessment of Constipation-Symptoms (PAC-SYM) questionnaire and its Modified version (M-PAC-SYM) measured constipation severity. Straining at defecation, stool frequency and form were daily recorded. K statistics for agreement and logistic regression were used at statistical analysis. RESULTS Satisfaction with therapy was not achieved by 43% of patients, who had a significantly lower Body Mass Index (BMI) and more severe constipation at baseline. Only the change in constipation severity according to M-PAC-SYM remained significantly associated with satisfaction with therapy (OR = 4.3; P < 0.001) at multivariate analysis. CONCLUSIONS Satisfaction with therapy is often an unmet need for patients with CC or with irritable bowel syndrome with constipation. Lower BMI and more severe constipation are associated with worse outcome. Changes in M-PAC-SYM reflect satisfaction with therapy. ClinicalTrials.gov no. NCT02813616.

中文翻译：

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慢性便秘或肠易激综合症伴便秘的患者对药物治疗的不满：对81位患者进行的N-of-1前瞻性研究分析。

背景技术患有便秘的慢性便秘（CC）或肠易激综合征的患者通常对他们的药物治疗不满意，但是他们的状况仍然不清楚。目的通过对N-of-1试验的汇总分析来评估患者的满意率以及哪些因素可以预测良好的结果。方法对81例CC或便秘性肠易激综合征的门诊患者进行N-of-1试验，其有效治疗周期至少为一个月。通过三个主要终点（对治疗的满意度，治疗后的改善以及包括两个终点的扩展满意度标准）来定义对治疗的满意度。评估了神经痛，静息的肛门压力，结肠的转运时间和躯体化。便秘症状患者评估（PAC-SYM）调查表及其修改版本（M-PAC-SYM）测量了便秘严重程度。每天记录排便时的劳损，大便次数和形式。统计分析使用K一致性和logistic回归的统计量。结果43％的患者体重指数（BMI）显着降低，基线时便秘更为严重，患者对治疗的满意度未达标。在多变量分析中，仅根据M-PAC-SYM进行的便秘严重程度变化仍与治疗满意度显着相关（OR = 4.3； P <0.001）。结论对CC或便秘性肠易激综合征患者而言，对治疗的满意往往是未满足的需求。较低的BMI和较严重的便秘与较差的预后相关。M-PAC-SYM的变化反映了对治疗的满意度。ClinicalTrials.gov号。NCT02813616。