Marschner Norbert, Staehler Michael, Müller Lothar, Nusch Arnd, Harde Johanna, Koska Michaela, Jänicke Martina, Goebell Peter J
Outpatient Centre for Interdisciplinary Oncology and Haematology, Freiburg, Germany.
Ludwig Maximilian University of Munich, University Hospital Campus Grosshadern, Urological Clinic and Outpatients Clinic, Munich, Germany.
Clin Genitourin Cancer. 2017 Apr;15(2):e209-e215. doi: 10.1016/j.clgc.2016.08.022. Epub 2016 Sep 8.
Because "real-life" patients often do not meet the strict eligibility criteria of clinical trials, we assessed the trial eligibility of patients with advanced or metastatic renal cell carcinoma (mRCC) in routine practice and compared the survival of "trial-ineligible" and potentially "trial-eligible" patients.
The present prospective, multicenter German cohort study is recruiting patients from 110 oncology/urology outpatient centers and hospitals at initiation of systemic first-line treatment. The demographic, clinical, treatment, and survival data were collected. We defined patients as "trial-ineligible" when ≥ 1 exclusion criterion (Karnofsky performance status < 80%, hemoglobin less than the lower limit of normal, non-clear cell carcinoma histology) was documented. Otherwise, the patients were considered "trial-eligible".
Of 732 patients included, 57% were classified as "trial-ineligible". Overall, the median first-line progression-free survival (PFS) was 7.9 months (95% confidence interval [CI], 6.9-8.9 months). The median first-line PFS of "trial-eligible" and "trial-ineligible" patients was 11.0 months (95% CI, 9.6-13.1 months) and 5.3 months (95% CI, 4.6-6.5 months), respectively. The median OS of the "trial-eligible" and "trial-ineligible" patients was 26.0 months (95% CI, 22.1-29.7 months) and 12.6 months (95% CI, 10.6-15.8 months), respectively.
Our data suggest that patients in routine practice differ from patients treated in clinical trials and that almost 60% of mRCC patients in German routine practice would be ineligible for participation in clinical trials. While their first-line PFS and OS were shorter than those of "trial-eligible" patients, the PFS and OS of "trial-eligible" patients were comparable with the results from clinical trials. Physicians should be aware of these differences when discussing treatment options and outcome expectations with patients.
由于“现实生活中的”患者往往不符合临床试验严格的入选标准，我们评估了晚期或转移性肾细胞癌（mRCC）患者在常规临床实践中的试验入选资格，并比较了“不符合试验标准”和潜在“符合试验标准”患者的生存率。
这项前瞻性、多中心德国队列研究在系统性一线治疗开始时，从110个肿瘤学/泌尿外科学门诊中心和医院招募患者。收集了人口统计学、临床、治疗和生存数据。当记录到≥1项排除标准（卡诺夫斯基功能状态<80%、血红蛋白低于正常下限、非透明细胞癌组织学）时，我们将患者定义为“不符合试验标准”。否则，患者被视为“符合试验标准”。
在纳入的732例患者中，57%被归类为“不符合试验标准”。总体而言，一线无进展生存期（PFS）的中位数为7.9个月（95%置信区间[CI]，6.9 - 8.9个月）。“符合试验标准”和“不符合试验标准”患者的一线PFS中位数分别为11.0个月（95%CI，9.6 - 13.1个月）和5.3个月（95%CI，4.6 - 6.5个月）。“符合试验标准”和“不符合试验标准”患者的总生存期（OS）中位数分别为26.0个月（95%CI，22.1 - 29.7个月）和12.6个月（95%CI，10.6 - 15.8个月）。
我们的数据表明，常规临床实践中的患者与临床试验中的患者不同，德国常规临床实践中近60%的mRCC患者不符合参与临床试验的条件。虽然他们的一线PFS和OS短于“符合试验标准”的患者，但“符合试验标准”患者的PFS和OS与临床试验结果相当。医生在与患者讨论治疗方案和预后期望时应意识到这些差异。
