Introduction: The efficacy of systemic therapy with immune checkpoint inhibitors (ICIs) for metastatic renal cell carcinoma (mRCC) has been demonstrated based on results of clinical trials; however, in trial-ineligible patients who do not meet the eligibility criteria for trials, its efficacy remains unclear.
Methods: We retrospectively evaluated 93 mRCC patients treated with ICIs including nivolumab plus ipilimumab, pembrolizumab plus axitinib, or avelumab plus axitinib as first-line therapy. The criteria for ineligibility for clinical trials was having at least one of the following: Karnofsky performance status (KPS) <70%, hemoglobin level <9.0 g/dL, eGFR <40 mL/min/1.73 m2, platelet count <100000 /µL, neutrophil count <1500 /µL, nonclear-cell histology, and brain metastasis. The patients were divided into two groups based on the ineligibility criteria, and progression-free survival (PFS), overall survival (OS), and objective response rate (ORR) were compared between the groups.
Results: Of the 93 patients, 48 (52%) were classified into the trial-ineligible group. The most frequent reason for being ineligible was a low eGFR (n=20, 45%), followed by nonclear cell histology (n=17, 36%) and low KPS (n=12, 25%). There was no significant difference in the PFS (median PFS: 24.0 vs. 11.0 months, p=0.416), OS (1-year OS: 87.0% vs.85.3%, p=0.634), or ORR (52% vs. 42%, p=0.308) between the trial-eligible and trial-ineligible groups (Figure 1A and 1B). In patients treated with nivolumab plus ipilimumab (n=67), the PFS (median PFS: 8.3 vs. 11.0 months, p=0.965), OS (1-year OS: 91.5% vs.83.1%, p=0.283), and ORR (50% vs. 45%, p=0.691) did not differ between the two groups (Figure 2A and 2B).
Conclusions: In a real-world setting, a substantial number of patients did not meet the eligibility criteria for clinical trials. Even in such patients, ICI-based treatment exhibited feasible anti-tumor activity, compared to that in trial-eligible patients.
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