Pembrolizumab for treating advanced urothelial carcinoma in patients with impaired performance status: Analysis of a Japanese nationwide cohort

Katsuhiro Ito, Takashi Kobayashi, Takahiro Kojima, Kensuke Hikami, Takeshi Yamada, Kosuke Ogawa, Kenji Nakamura, Naoto Sassa, Akira Yokomizo, Takashige Abe, Kazunari Tsuchihashi, Shuichi Tatarano, Junichi Inokuchi, Ryotaro Tomida, Maki Fujiwara, Atsushi Takahashi, Kazumasa Matsumoto, Kosuke Shimizu, Hiromasa Araki, Ryoma KurahashiYu Osaki, Yu Tashiro, Masayuki Uegaki, Osamu Ogawa, Hiroshi Kitamura, Hiroyuki Nishiyama

Research output: Contribution to journalArticlepeer-review

16 Citations (Scopus)


Background: The benefits of pembrolizumab in patients with advanced urothelial carcinoma (UC) and impaired performance status (PS) remain unknown. This study assessed the safety and efficacy of pembrolizumab in patients with platinum-refractory UC and Eastern Cooperative Oncology Group PS ≥2 to identify which subgroups may benefit from this drug. Methods: This retrospective nationwide cohort study collected clinicopathological information for 755 patients from 59 institutions. The overall response rate (ORR) and overall survival (OS) were compared among the patients with PS 0–1, 2, and 3–4. Multivariate analysis was conducted to identify factors predicting OS in patients with PS ≥2. Results: The numbers of patients with PS 0–1, 2, and 3–4 were 602, 98, and 55, respectively; the ORRs in these groups were 29.5, 15.3, and 9.1%, respectively, and the median OS times were 14.3, 3.1, and 2.4 months, respectively. In multivariate Cox regression analysis, a neutrophil–lymphocyte ratio (NLR) ≥3.5 (hazard ratio [HR] = 1.897) and liver metastasis (HR = 2.072) were associated with OS in the PS ≥2 subgroup. The median OS of patients with PS ≥2 without either risk factor was 6.8 months, compared with 3.1 months for patients with one risk factor and 2.3 months for patients with both risk factors. Conclusions: PS ≥2 portended worse ORR and OS than PS ≤1 despite a comparable safety profile. Among the patients with impaired PS, patients with NLR <3.5 and no liver metastasis may most greatly benefit from pembrolizumab therapy.

Original languageEnglish
Pages (from-to)3188-3196
Number of pages9
JournalCancer Medicine
Issue number10
Publication statusPublished - May 2021

All Science Journal Classification (ASJC) codes

  • Oncology
  • Radiology Nuclear Medicine and imaging
  • Cancer Research


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