寻找用于化疗引起的周围神经病变临床试验的金标准患者报告结局指标。

In Search of a Gold Standard Patient-Reported Outcome Measure for Use in Chemotherapy- Induced Peripheral Neuropathy Clinical Trials.

作者信息

Smith Ellen M Lavoie, Knoerl Robert, Yang James J, Kanzawa-Lee Grace, Lee Deborah, Bridges Celia M

机构信息

1 University of Michigan School of Nursing, Ann Arbor, MI, USA.

2 Phylllis F. Cantor Center for Research in Nursing and Patient Care Services, Dana Farber Cancer Institute, Boston, MA, USA.

出版信息

Cancer Control. 2018 Jan-Mar;25(1):1073274818756608. doi: 10.1177/1073274818756608.

DOI:10.1177/1073274818756608

PMID:29480026

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC5925747/

Abstract

PURPOSE

To test a reduced version-CIPN15-of the European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire Chemotherapy-Induced Peripheral Neuropathy scale (QLQ-CIPN20) to establish a possible gold-standard patient-reported outcome measure for chemotherapy-induced peripheral neuropathy (CIPN).

METHODS

Using a prospective, longitudinal, case-control design, patients (n = 121) receiving neurotoxic chemotherapy completed the CIPN15 at baseline and 12 weeks and underwent objective neurological assessment using the 5-item Total Neuropathy Score-Clinical (TNSc). Healthy controls (n = 30) completed the CIPN15 once. Structural validity was evaluated using factor analysis. Because a stable factor structure was not found, a sum score was used to evaluate measures of the CIPN15's psychometric properties-reliability, validity, sensitivity, and responsiveness-as follows: internal consistency via Cronbach's α and item-item correlations; test-retest reliability via correlation between 2 CIPN15 scores from each patient; concurrent validity via correlation between CIPN15 and 5-item TNSc scores; contrasting group validity via comparison of CIPN15 scores from patients and healthy controls; sensitivity via descriptive statistics (means, standard deviation, ranges); and responsiveness via Cohen's d effect size.

RESULTS

Most patients received single agent oxaliplatin (33.7%), paclitaxel (21.2%), or more than 1 neurotoxic drug concurrently (29.8%). Factor analysis revealed no stable factor structure. Cronbach's α for the CIPN15 sum score was 0.91 (confidence interval [CI] = 0.89-0.93). Test-retest reliability was demonstrated based on strong correlations between the 2 scores obtained at the 12-week time point ( r = 0.86; CI = 0.80-0.90). The CIPN15 and 5-item TNSc items reflecting symptoms (not signs) were moderately correlated ( r range 0.57-0.72): concurrent validity. Statistically significant differences were found between patient and healthy control CIPN15 mean scores ( P < .0001): contrasting group validity. All items encompassed the full score range but the CIPN15 linearly converted sum score did not: sensitivity. The CIPN15 was responsive based on a Cohen's d of 0.52 (CI = 0.25-0.79).

CONCLUSION

The sum-scored CIPN15 is reliable, valid, sensitive, and responsive when used to assess taxane- and platinum-induced CIPN.

摘要

目的

测试欧洲癌症研究与治疗组织（EORTC）生活质量问卷化疗所致周围神经病变量表（QLQ-CIPN20）的简化版CIPN15，以建立一种可能的化疗所致周围神经病变（CIPN）的金标准患者报告结局指标。

方法

采用前瞻性、纵向、病例对照设计，接受神经毒性化疗的患者（n = 121）在基线和12周时完成CIPN15，并使用5项总神经病变评分-临床版（TNSc）进行客观神经学评估。健康对照者（n = 30）完成一次CIPN15。使用因子分析评估结构效度。由于未发现稳定的因子结构，因此使用总分来评估CIPN15的心理测量学特性——信度、效度、敏感性和反应性，具体如下：通过Cronbach's α和项目间相关性评估内部一致性；通过每位患者两次CIPN15评分之间的相关性评估重测信度；通过CIPN15与5项TNSc评分之间的相关性评估同时效度；通过比较患者和健康对照者的CIPN15评分评估对比组效度；通过描述性统计（均值、标准差、范围）评估敏感性；通过Cohen's d效应量评估反应性。

结果

大多数患者接受单药奥沙利铂（33.7%）、紫杉醇（21.2%）或同时接受超过1种神经毒性药物（29.8%）。因子分析未发现稳定的因子结构。CIPN15总分的Cronbach's α为0.91（置信区间[CI] = 0.89 - 0.93）。基于在12周时间点获得的两次评分之间的强相关性（r = 0.86；CI = 0.80 - 0.90）证明了重测信度。CIPN15与反映症状（而非体征）的5项TNSc项目中度相关（r范围为0.57 - 0.72）：同时效度。患者和健康对照者的CIPN15平均得分之间存在统计学显著差异（P <.0001）：对比组效度。所有项目涵盖了整个评分范围，但CIPN15线性转换后的总分未涵盖：敏感性。基于Cohen's d为0.52（CI = 0.25 - 0.79），CIPN15具有反应性。