多中心临床试验中患者报告结局的依从性：方法学与实践方法

Compliance with patient-reported outcomes in multicenter clinical trials: methodologic and practical approaches.

作者信息

Land Stephanie R, Ritter Marcie W, Costantino Joseph P, Julian Thomas B, Cronin Walter M, Haile Sarah R, Wolmark Norman, Ganz Patricia A

机构信息

National Surgical Adjuvant Breast and Bowel Project Operations and Biostatistical Centers, Pittsburgh, PA 15213, USA.

出版信息

J Clin Oncol. 2007 Nov 10;25(32):5113-20. doi: 10.1200/JCO.2007.12.1749.

DOI:10.1200/JCO.2007.12.1749

PMID:17991930

Abstract

PURPOSE

This report describes interventions undertaken by the National Surgical Adjuvant Breast and Bowel Project (NSABP) to improve compliance with patient-reported outcome (PRO) assessments in the setting of multicenter cancer clinical trials. We describe the effectiveness of several interventions and of observational factors.

METHODS

PRO submission rates were analyzed for the following three NSABP protocols: the Study of Raloxifene and Tamoxifen (STAR), B-32, and B-35. Institutions participating in protocol B-35 were randomly assigned to receive automated reminders of upcoming assessments or not. Compliance was analyzed with a logistic repeated measures mixed modeling.

RESULTS

Compliance was high in the three protocols, with rates greater than 80% for nearly all time points. Institutions were a significant source of variability (P < .01). The largest institutions had the highest compliance in STAR (odds ratio [OR] = 0.68 for < 50 participants enrolled and OR = 0.82 for 50 to 99 participants enrolled v larger institutions; P < .001). Midsized institutions had highest compliance in B-32 (OR = 4.63 for 31 to 50 patients enrolled and OR = 3.12 for > 50 patients enrolled v small institutions; P = .007). Compliance increased with participant age in STAR (OR = 0.57, 0.89, and 1.01 for ages < 50, 50 to 60, and 60 to 70 years, respectively, v > 70 years; P < .001). Race was significant in B-32 (OR = 2.63 for white v nonwhite; P < .001) and in STAR (OR = 1.41 for white v nonwhite; P < .001). Treatment group was significant in B-32 (OR = 0.74; P = .006). The B-35 prospective reminder did not improve compliance significantly (P = .30), but in B-32, delinquency sanctions were significant (OR = 1.56; P = .007).

CONCLUSION

Compliance in NSABP PRO studies is higher now than a decade ago. Results for compliance initiatives were mixed. Age and race are important factors, but institutional variation remains significant and largely unexplained.

摘要

目的

本报告描述了美国国家乳腺与肠道外科辅助治疗项目（NSABP）为提高多中心癌症临床试验中患者报告结局（PRO）评估的依从性而采取的干预措施。我们描述了几种干预措施和观察因素的有效性。

方法

分析了NSABP的以下三项方案的PRO提交率：雷洛昔芬与他莫昔芬研究（STAR）、B-32和B-35。参与方案B-35的机构被随机分配接受即将进行评估的自动提醒或不接受。采用逻辑重复测量混合模型分析依从性。

结果

三项方案的依从性都很高，几乎所有时间点的比率都超过80%。机构是变异性的一个重要来源（P < 0.01）。在STAR中，规模最大的机构依从性最高（入组<50名参与者的优势比[OR]=0.68，入组50至99名参与者的OR=0.82，与规模较大的机构相比；P < 0.001）。在B-32中，中等规模机构的依从性最高（入组31至50名患者的OR=4.63，入组>50名患者的OR=3.12，与小机构相比；P = 0.007）。在STAR中，依从性随参与者年龄增加而提高（年龄<50岁、50至60岁、60至70岁和>70岁的OR分别为0.57、0.89和1.01，与>70岁相比；P < 0.001）。种族在B-32（白人对非白人的OR=2.63；P < 0.001）和STAR（白人对非白人的OR=1.41；P < 0.001）中具有显著意义。治疗组在B-32中具有显著意义（OR=0.74；P = 0.006）。B-35的前瞻性提醒并未显著提高依从性（P = 0.30），但在B-32中，违规制裁具有显著意义（OR=1.56；P = 0.007）。