确定阿片类药物诱导性便秘的生活质量测量中的最小临床重要差异。
Establishing Minimal Clinically Important Differences in Quality of Life Measures in Opioid-Induced Constipation.
机构信息
Translational Research Center for Gastrointestinal Disorders, University of Leuven, Leuven, Belgium.
Clinical Enteric Neuroscience Translational and Epidemiological Research, Mayo Clinic, Rochester, Minnesota.
出版信息
Clin Gastroenterol Hepatol. 2022 Apr;20(4):855-863. doi: 10.1016/j.cgh.2021.05.004. Epub 2021 Aug 5.
BACKGROUND & AIMS: Opioids have a role in chronic pain management. However, opioid-induced constipation may cause patients to skip or reduce opioid doses, leading to inadequate pain relief and negatively impacting quality of life. We sought to establish a minimal clinically important difference to understand whether changes in quality of life scores are of value to patients.
METHODS
Integrated data from the double-blind, controlled, phase 3 COMPOSE-1 and COMPOSE-2 trials of naldemedine in chronic noncancer pain and opioid-induced constipation were used to determine minimal clinically important differences using Patient Assessment of Constipation Symptoms (PAC-SYM) and Patient Assessment of Constipation Quality of Life (PAC-QOL) questionnaires. Patients completed the questionnaires (5-point Likert scale; predose, Weeks 2, 4, and 12), kept a daily log of Bowel Movement and Constipation Assessment, and rated satisfaction at end of study. Minimal clinically important differences were computed using an anchor-based method with 6 anchors: 5 from the Bowel Movement and Constipation Assessment and 1 from patient satisfaction. Threshold values for each anchor were set to define responders versus nonresponders based on score definitions. Clinically meaningful cutoff values for changes in PAC-SYM and PAC-QOL scores were determined using receiver operating characteristic curves.
RESULTS
Data from 1095 patients (549, naldemedine; 546, placebo) were analyzed. The area under the curve for the receiver operating characteristic curves (ranges, 0.719 to 0.798 for PAC-SYM and 0.734 to 0.833 for PAC-QOL) indicated that both instruments can discriminate responders and nonresponders for each anchor. PAC-SYM cutoff values ranged from -1.04 to -0.83; PAC-QOL cutoff values ranged from -0.93 to -0.82.
CONCLUSIONS
Based on data derived from the anchor method, reductions in PAC-SYM and PAC-QOL scores of >1.0 in patients with chronic noncancer pain and opioid-induced constipation are clinically meaningful.
CLINICALTRIALS
gov Registration: NCT01965158; NCT01993940.
背景与目的
阿片类药物在慢性疼痛管理中具有一定作用。然而，阿片类药物引起的便秘可能导致患者减少或停用阿片类药物剂量，导致疼痛缓解不足，影响生活质量。我们旨在确定最小临床有意义差异，以了解生活质量评分的变化对患者是否有价值。
方法
采用纳美丁胺治疗慢性非癌痛和阿片类药物诱导性便秘的双盲、对照、3 期 COMPOSE-1 和 COMPOSE-2 试验的综合数据，使用患者便秘症状评估量表（PAC-SYM）和患者便秘生活质量评估量表（PAC-QOL）来确定最小临床有意义差异。患者完成问卷（5 分李克特量表；预治疗、第 2、4 和 12 周），每天记录排便和便秘评估情况，并在研究结束时评估满意度。最小临床有意义差异采用基于锚定的方法计算，有 6 个锚定物：5 个来自排便和便秘评估，1 个来自患者满意度。根据评分定义，为每个锚定点设定阈值，以定义反应者与非反应者。使用受试者工作特征曲线确定 PAC-SYM 和 PAC-QOL 评分变化的临床有意义的截断值。
结果
共分析了 1095 例患者（纳美丁胺组 549 例，安慰剂组 546 例）的数据。受试者工作特征曲线下面积（范围为 PAC-SYM 为 0.719 至 0.798，PAC-QOL 为 0.734 至 0.833）表明，两种工具均可为每个锚定点区分反应者和非反应者。PAC-SYM 截断值范围为-1.04 至-0.83；PAC-QOL 截断值范围为-0.93 至-0.82。
结论
基于锚定方法得出的数据，慢性非癌痛和阿片类药物诱导性便秘患者的 PAC-SYM 和 PAC-QOL 评分降低>1.0 具有临床意义。
临床试验
gov 注册号：NCT01965158；NCT01993940。
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