Basch Ethan, Jia Xiaoyu, Heller Glenn, Barz Allison, Sit Laura, Fruscione Michael, Appawu Mark, Iasonos Alexia, Atkinson Thomas, Goldfarb Shari, Culkin Ann, Kris Mark G, Schrag Deborah
Health Outcomes Group, Department of Epidemiology and Biostatistics, Memorial Sloan-Kettering Cancer Center, 307 East 63rd St, New York, NY 10065, USA.
J Natl Cancer Inst. 2009 Dec 2;101(23):1624-32. doi: 10.1093/jnci/djp386. Epub 2009 Nov 17.
In cancer treatment trials, the standard source of adverse symptom data is clinician reporting by use of items from the National Cancer Institute's Common Terminology Criteria for Adverse Events (CTCAE). Patient self-reporting has been proposed as an additional data source, but the implications of such a shift are not understood.
Patients with lung cancer receiving chemotherapy and their clinicians independently reported six CTCAE symptoms and Karnofsky Performance Status longitudinally at sequential office visits. To compare how patient's vs clinician's reports relate to sentinel clinical events, a time-dependent Cox regression model was used to measure associations between reaching particular CTCAE grade severity thresholds with the risk of death and emergency room visits. To measure concordance of CTCAE reports with indices of daily health status, Kendall tau rank correlation coefficients were calculated for each symptom with EuroQoL EQ-5D questionnaire and global question scores. Statistical tests were two-sided.
A total of 163 patients were enrolled for an average of 12 months (range = 1-28 months), with a mean of 11 visits and 67 (41%) deaths. CTCAE reports were submitted by clinicians at 95% of visits and by patients at 80% of visits. Patients generally reported symptoms earlier and more frequently than clinicians. Statistically significant associations with death and emergency room admissions were seen for clinician reports of fatigue (P < .001), nausea (P = .01), constipation (P = .038), and Karnofsky Performance Status (P < .001) but not for patient reports of these items. Higher concordance with EuroQoL EQ-5D questionnaire and global question scores was observed for patient-reported symptoms than for clinician-reported symptoms.
Longitudinally collected clinician CTCAE assessments better predict unfavorable clinical events, whereas patient reports better reflect daily health status. These perspectives are complementary, each providing clinically meaningful information. Inclusion of both types of data in treatment trial results and drug labels appears to be warranted.
在癌症治疗试验中，不良症状数据的标准来源是临床医生使用美国国立癌症研究所的不良事件通用术语标准（CTCAE）中的条目进行报告。患者自我报告已被提议作为额外的数据来源，但这种转变的影响尚不清楚。
接受化疗的肺癌患者及其临床医生在连续的门诊就诊时纵向独立报告六种CTCAE症状和卡氏功能状态。为了比较患者报告与临床医生报告如何与标志性临床事件相关，使用了时间依赖性Cox回归模型来测量达到特定CTCAE分级严重程度阈值与死亡风险和急诊就诊之间的关联。为了测量CTCAE报告与日常健康状况指标的一致性，计算了每种症状与欧洲生活质量五维度量表（EuroQoL EQ-5D）问卷和总体问题得分的肯德尔等级相关系数。统计检验为双侧检验。
共纳入163例患者，平均随访12个月（范围=1 - 28个月），平均就诊11次，67例（41%）死亡。临床医生在95%的就诊时提交了CTCAE报告，患者在80%的就诊时提交了报告。患者通常比临床医生更早且更频繁地报告症状。临床医生报告的疲劳（P <.001）、恶心（P =.01）、便秘（P =.038）和卡氏功能状态（P <.001）与死亡和急诊入院有统计学显著关联，但患者报告这些项目时未观察到这种关联。与临床医生报告的症状相比，患者报告的症状与欧洲生活质量五维度量表问卷和总体问题得分的一致性更高。
纵向收集的临床医生CTCAE评估能更好地预测不良临床事件，而患者报告能更好地反映日常健康状况。这些观点是互补的，各自提供了具有临床意义的信息。在治疗试验结果和药品标签中纳入这两种类型的数据似乎是有必要的。
