Abstract
Background
Clinical data from patients with non–clear cell renal cell carcinoma (nccRCC) receiving
targeted therapy are limited, and many clinical trials have excluded these patients
from study entry. We sought to investigate the outcomes of patients with nccRCC treated
in clinical trials in the modern era compared with the outcomes of patients with clear
cell RCC (ccRCC).
Patients and Methods
We conducted a retrospective study of patients with metastatic RCC who had received
targeted therapy in Pfizer-sponsored phase II and III clinical trials from 2003 to
2013. Associations between the histologic type and treatment outcome (overall survival
[OS] and progression-free survival [PFS]) were assessed using the log-rank test on
univariate analysis or the Wald χ2 test from Cox regression on multivariable analysis, adjusted for baseline characteristics,
including age, sex, Eastern Cooperative Oncology Group performance status, body mass
index, International Metastatic RCC Database Consortium risk factors, previous nephrectomy,
previous therapy, metastatic sites, angiotensin system inhibitor use, and statin use.
Results
We identified 4527 patients with metastatic RCC: 4235 with ccRCC and 337 with nccRCC.
Overall, the median OS was shorter for those with nccRCC than for those with ccRCC
(15.7 vs. 20.2 months; hazard ratio [HR], 1.41; 95% confidence interval 1.22-1.63;
P < .001). When stratified by the International Metastatic RCC Database Consortium
risk group, the median OS was inferior for the intermediate- and poor-risk patients
with nccRCC than for those with ccRCC. However, no differences were found in the favorable
risk group for nccRCC versus ccRCC. The patients with nccRCC who had received vascular
endothelial growth factor-targeted therapy had shorter PFS compared with that of ccRCC
patients (median, 6.1 vs. 8.5 months; HR, 1.49; P < .001) but similar PFS when treated with mammalian target of rapamycin inhibitors
(median, 4.3 vs. 4.4 months; HR, 0.92; P = .63).
Conclusion
Our findings have confirmed that patients with nccRCC are underrepresented in clinical
trials and highlight the need for further prospective studies exploring current and
novel agents for this patient population.
Keywords
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Article info
Publication history
Published online: March 21, 2017
Accepted:
March 10,
2017
Received in revised form:
March 8,
2017
Received:
January 3,
2017
Footnotes
G.d.V. and R.R.M. contributed equally to this work.
Identification
Copyright
© 2017 Elsevier Inc. All rights reserved.
