晚期转移性膀胱癌患者中患者与临床医生报告的症状一致性
Patient Versus Clinician Reported Symptoms Agreement in Advanced Metastatic Bladder Cancer Patients.
作者信息
Annakib Soufyan, Di Méglio Emma, Dibert-Bekoy Yona, Chevallier Thierry, Roubaud Guilhem, Fournel Pierre, Guillot Aline, Borchiellini Delphine, Pouessel Damien, Boughalem Elouen, Delva Remy, Barthelemy Philippe, Oudard Stéphane, Thibault Constance, Tosi Diego, Houédé Nadine, Fiteni Frédéric
机构信息
Medical Oncology Department, CHU Nîmes, Univ Montpellier, Nîmes, France.
Department of Biostatistics, Epidemiology, Public Health and Innovation in Methodology (BESPIM), CHU Nîmes, Univ Montpellier, Nîmes, France.
出版信息
Cancer Med. 2025 Apr;14(8):e70896. doi: 10.1002/cam4.70896.
BACKGROUND
Immune checkpoint inhibitors (ICIs) improved survival in patients with locally advanced or metastatic urothelial carcinoma (la/mUC). Patient-reported symptoms in this context were poorly studied. The study aimed to compare symptom severity between patients and clinicians.
METHODOLOGY
The secondary analysis of the AMI clinical trial comparing changes in the gut microbiota in patients with la/mUC treated with pembrolizumab was conducted in nine French centers. Secondary endpoints were expected in this prospective study. Patient-Reported Outcome-Common Terminology Criteria for Adverse Events (PRO-CTCAE) and CTCAE were assessed respectively by patients and clinicians before pembrolizumab initiation, and at each treatment visit until treatment cycle 12. Agreement in severity between clinicians and patients for grade ≥ 3 symptoms was calculated with Cohen's kappa coefficient. The toxicity index was generated for CTCAE and PRO-CTCAE to assess discordance in a longitudinal manner. The Wilcoxon test was used to compare clinicians' and patients' toxicity index and symptom severity frequencies.
RESULTS
Thirty-nine patients were included (M/F sex ratio: 2.5) from December 2020 to March 2022. PRO-CTCAE baseline completion rate was 77.5%. Cohen's kappa coefficient ranged from -0.017 (95% confidence interval (CI), [-0.039, 0.005]) for numbness/tingling to 0.161 (95% CI, [0.045, 0.276]) for fatigue. The patient self-rated symptom toxicity index was > 2 for all symptoms compared to ≤ 0.62 (fatigue) when assessed by clinicians in longitudinal reporting of symptom frequency and severity with a p value < 0.001. The three most commonly reported symptoms by patients and clinicians, respectively, were: Fatigue 53.3% versus 23.4%, generalized pain 42.4% versus 16.5%, and insomnia 41.1% versus 9.5%. Symptom frequency reports between clinicians and patients were statistically different (p < 0.009).
CONCLUSIONS
Symptom severity assessment showed discordance between patients and physicians. Clinicians reported fewer symptoms and graded them less severely than patients. PROs should be used to accurately reflect patient experience.
TRIAL REGISTRATION
ClinicalTrials.gov identifier: NCT04566029.
背景
免疫检查点抑制剂（ICIs）提高了局部晚期或转移性尿路上皮癌（la/mUC）患者的生存率。在这种情况下，患者报告的症状研究较少。本研究旨在比较患者和临床医生之间症状的严重程度。
方法
在法国的九个中心对一项比较帕博利珠单抗治疗la/mUC患者肠道微生物群变化的AMI临床试验进行了二次分析。在这项前瞻性研究中设定了次要终点。在开始使用帕博利珠单抗之前以及直到治疗周期12的每次治疗访视时，分别由患者和临床医生评估患者报告的不良事件通用术语标准（PRO-CTCAE）和CTCAE。使用Cohen's kappa系数计算临床医生和患者对于≥3级症状严重程度的一致性。生成CTCAE和PRO-CTCAE的毒性指数以纵向评估不一致性。使用Wilcoxon检验比较临床医生和患者的毒性指数以及症状严重程度频率。
结果
2020年12月至2022年3月纳入了39名患者（男/女性别比：2.5）。PRO-CTCAE基线完成率为77.5%。Cohen's kappa系数范围从麻木/刺痛的-0.017（95%置信区间（CI），[-0.039, 0.005]）到疲劳的0.161（95%CI，[0.045, 0.276]）。在症状频率和严重程度的纵向报告中，与临床医生评估时≤0.62（疲劳）相比，患者自我评定的所有症状的症状毒性指数均>2，p值<0.001。患者和临床医生分别最常报告的三种症状是：疲劳53.3%对23.4%，全身疼痛42.4%对16.5%，失眠41.1%对9.5%。临床医生和患者之间的症状频率报告存在统计学差异（p<0.009）。
结论
症状严重程度评估显示患者和医生之间存在不一致。临床医生报告的症状较少，且对其严重程度的分级低于患者。应使用患者报告结局来准确反映患者的体验。
试验注册
ClinicalTrials.gov标识符：NCT04566029。
Zotero 插件