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Background <p>Cisplatin-based neoadjuvant chemotherapy (NAC) improves survival in muscle-invasive bladder cancer (MIBC) as long as disease progression does not occur during treatment. However, predictors of NAC sensitivity remain elusive in clinical practice. This study evaluated the efficacy of NAC followed by radical cystectomy (NAC-RC) in cStage II–IIIA MIBC and identified the risk factors associated with residual extravesical disease.</p> Methods <p>Clinical data from 1474 patients who underwent radical cystectomy for cStage II–IIIA urothelial carcinoma were collected from 36 institutions of the Japanese Urological Oncology Group. Overall survival (OS) and non-urinary tract recurrence-free survival (NUT-RFS) were compared between the NAC-RC and upfront RC groups using the Kaplan–Meier method adjusted by inverse probability of treatment weighting. Logistic regression was used to identify independent risk factors for RED.</p> Results <p>Pathological complete response (pT0N0) was achieved in 33.1 and 20.2% of cStage II and IIIA patients in the NAC-RC group, respectively, compared with 16.3 and 4.5% in the RC group. NAC significantly improved the OS and NUT-RFS in the IPTW-adjusted cohort. BCG-unresponsiveness, low serum albumin levels, and a high neutrophil-to-lymphocyte ratio were independent predictors of RED in the NAC-RC cohort. Squamous differentiation was associated with worse prognosis but a favorable response to NAC in some tumors.</p> Conclusions <p>Cisplatin-based NAC improves outcomes in patients with cStage II–IIIA MIBC, including some tumors with squamous differentiation; however, its benefits may be limited in BCG-unresponsive cases. Given the biological heterogeneity of urothelial cancer, individualized treatment planning that integrates biological features and treatment history is needed for patients with MIBC.</p>

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Efficacy of cisplatin-based neoadjuvant chemotherapy and risk factors for residual extravesical disease in muscle-invasive bladder cancer: insights from a nationwide cohort

  • 期刊论文

  • 发表日期：

    2025年7月21日

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  • Ryoichi Saito,
  • Rikiya Taoka,
  • Jun Miki,
  • Wataru Fukuokaya,
  • Yoshiyuki Matsui,
  • Shingo Hatakeyama,
  • Takashi Kawahara,
  • Ayumu Matsuda,
  • Taketo Kawai,
  • Tomokazu Sazuka,
  • Minoru Kato,
  • Takeshi Sano,
  • Fumihiko Urabe,
  • Soki Kashima,
  • Hirohito Naito,
  • Yoji Murakami,
  • Makito Miyake,
  • Kei Daizumoto,
  • Yuto Matsushita,
  • Takuji Hayashi,
  • Junichi Inokuchi,
  • Yusuke Sugino,
  • Kenichiro Shiga,
  • Noriya Yamaguchi,
  • Shingo Yamamoto,
  • Keiji Yasue,
  • Takashige Abe,
  • Shotaro Nakanishi,
  • Katsuyoshi Hashine,
  • Masato Fujii,
  • Kiyoaki Nishihara,
  • Hiroaki Matsumoto,
  • Shuichi Tatarano,
  • Koichiro Wada,
  • Sho Sekito,
  • Ryo Maruyama,
  • Naotaka Nishiyama,
  • Hiroyuki Nishiyama,
  • Hiroshi Kitamura,
  • Hidefumi Kinoshita

摘要

Background

Cisplatin-based neoadjuvant chemotherapy (NAC) improves survival in muscle-invasive bladder cancer (MIBC) as long as disease progression does not occur during treatment. However, predictors of NAC sensitivity remain elusive in clinical practice. This study evaluated the efficacy of NAC followed by radical cystectomy (NAC-RC) in cStage II–IIIA MIBC and identified the risk factors associated with residual extravesical disease.

Methods

Clinical data from 1474 patients who underwent radical cystectomy for cStage II–IIIA urothelial carcinoma were collected from 36 institutions of the Japanese Urological Oncology Group. Overall survival (OS) and non-urinary tract recurrence-free survival (NUT-RFS) were compared between the NAC-RC and upfront RC groups using the Kaplan–Meier method adjusted by inverse probability of treatment weighting. Logistic regression was used to identify independent risk factors for RED.

Results

Pathological complete response (pT0N0) was achieved in 33.1 and 20.2% of cStage II and IIIA patients in the NAC-RC group, respectively, compared with 16.3 and 4.5% in the RC group. NAC significantly improved the OS and NUT-RFS in the IPTW-adjusted cohort. BCG-unresponsiveness, low serum albumin levels, and a high neutrophil-to-lymphocyte ratio were independent predictors of RED in the NAC-RC cohort. Squamous differentiation was associated with worse prognosis but a favorable response to NAC in some tumors.

Conclusions

Cisplatin-based NAC improves outcomes in patients with cStage II–IIIA MIBC, including some tumors with squamous differentiation; however, its benefits may be limited in BCG-unresponsive cases. Given the biological heterogeneity of urothelial cancer, individualized treatment planning that integrates biological features and treatment history is needed for patients with MIBC.