Propensity Score Analysis of Regorafenib Versus Trifluridine/Tipiracil in Patients with Metastatic Colorectal Cancer Refractory to Standard Chemotherapy (REGOTAS)

A Japanese Society for Cancer of the Colon and Rectum Multicenter Observational Study

Toshikazu Moriwaki, Shota Fukuoka, Hiroya Taniguchi, Atsuo Takashima, Yusuke Kumekawa, Takeshi Kajiwara, Kentaro Yamazaki, Taito Esaki, Chinatsu Makiyama, Tadamichi Denda, Hironaga Satake, Takeshi Suto, Naotoshi Sugimoto, Masanobu Enomoto, Toshiaki Ishikawa, Tomomi Kashiwada, Masahiko Sugiyama, Yoshito Komatsu, Hiroyuki Okuyama, Eishi Baba & 6 others Daisuke Sakai, Tomoki Watanabe, Takao Tamura, Kimihiro Yamashita, Masahiko Gosho, Yasuhiro Shimada

Research output: Contribution to journalArticle

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Abstract

Background: This study compared the efficacy of regorafenib and trifluridine/tipiracil (TFTD) in patients with metastatic colorectal cancer (mCRC) who are refractory to standard chemotherapy, because despite their clinical approval, it still remains unclear which of these two drugs should be used as initial treatment. Materials and Methods: The clinical data of patients with mCRC who were treated with regorafenib or TFTD and those of drug-naive patients, between June 2014 and September 2015, were retrospectively collected from 24 institutions in Japan. Overall survival (OS) was evaluated using the Cox's proportional hazard models based on propensity score adjustment for baseline characteristics. Results: A total of 550 patients (223 patients in the regorafenib group and 327 patients in the TFTD group) met all criteria. The median OS was 7.9 months (95% confidence interval [CI], 6.8–9.2) in the regorafenib group and 7.4 months (95% CI, 6.6–8.3) in the TFTD group. The propensity score adjusted analysis showed that OS was similar between the two groups (adjusted hazard ratio [HR], 0.96; 95% CI, 0.78–1.18). In the subgroup analysis, a significant interaction with age was observed. Regorafenib showed favorable survival in patients aged <65 years (HR, 1.29; 95% CI, 0.98–1.69), whereas TFTD was favored in patients aged ≥65 years (HR, 0.78; 95% CI, 0.59–1.03). Conclusion: No significant difference in OS between regorafenib and TFTD was observed in patients with mCRC. Although the choice of the drug by age might affect survival, a clearly predictive biomarker to distinguish the two drugs should be identified in further studies. Implications for Practice: Previous studies of patients with metastatic colorectal cancer refractory to standard chemotherapy had demonstrated that both regorafenib and trifluridine/tipiracil could result in increased overall survival compared with placebo, but there are no head-to-head trials. This large, multicenter, observational study retrospectively compared the efficacy of regorafenib and trifluridine/tipiracil in 550 patients with metastatic colorectal cancer refractory to standard chemotherapy who had access to both drugs. Although no difference in overall survival was found between the two drugs in adjusted analysis using propensity score, regorafenib showed favorable survival in patients aged <65 years, whereas trifluridine/tipiracil was favored in patients aged ≥65 years in the subgroup analysis.

Original languageEnglish
Pages (from-to)7-15
Number of pages9
JournalOncologist
Volume23
Issue number1
DOIs
Publication statusPublished - Jan 1 2018

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Trifluridine
Propensity Score
Rectal Neoplasms
Colonic Neoplasms
Multicenter Studies
Observational Studies
Colorectal Neoplasms
Drug Therapy
Survival
Confidence Intervals
Pharmaceutical Preparations
regorafenib

All Science Journal Classification (ASJC) codes

  • Oncology
  • Cancer Research

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Propensity Score Analysis of Regorafenib Versus Trifluridine/Tipiracil in Patients with Metastatic Colorectal Cancer Refractory to Standard Chemotherapy (REGOTAS) : A Japanese Society for Cancer of the Colon and Rectum Multicenter Observational Study. / Moriwaki, Toshikazu; Fukuoka, Shota; Taniguchi, Hiroya; Takashima, Atsuo; Kumekawa, Yusuke; Kajiwara, Takeshi; Yamazaki, Kentaro; Esaki, Taito; Makiyama, Chinatsu; Denda, Tadamichi; Satake, Hironaga; Suto, Takeshi; Sugimoto, Naotoshi; Enomoto, Masanobu; Ishikawa, Toshiaki; Kashiwada, Tomomi; Sugiyama, Masahiko; Komatsu, Yoshito; Okuyama, Hiroyuki; Baba, Eishi; Sakai, Daisuke; Watanabe, Tomoki; Tamura, Takao; Yamashita, Kimihiro; Gosho, Masahiko; Shimada, Yasuhiro.

In: Oncologist, Vol. 23, No. 1, 01.01.2018, p. 7-15.

Research output: Contribution to journalArticle

Moriwaki, T, Fukuoka, S, Taniguchi, H, Takashima, A, Kumekawa, Y, Kajiwara, T, Yamazaki, K, Esaki, T, Makiyama, C, Denda, T, Satake, H, Suto, T, Sugimoto, N, Enomoto, M, Ishikawa, T, Kashiwada, T, Sugiyama, M, Komatsu, Y, Okuyama, H, Baba, E, Sakai, D, Watanabe, T, Tamura, T, Yamashita, K, Gosho, M & Shimada, Y 2018, 'Propensity Score Analysis of Regorafenib Versus Trifluridine/Tipiracil in Patients with Metastatic Colorectal Cancer Refractory to Standard Chemotherapy (REGOTAS): A Japanese Society for Cancer of the Colon and Rectum Multicenter Observational Study', Oncologist, vol. 23, no. 1, pp. 7-15. https://doi.org/10.1634/theoncologist.2017-0275
Moriwaki, Toshikazu ; Fukuoka, Shota ; Taniguchi, Hiroya ; Takashima, Atsuo ; Kumekawa, Yusuke ; Kajiwara, Takeshi ; Yamazaki, Kentaro ; Esaki, Taito ; Makiyama, Chinatsu ; Denda, Tadamichi ; Satake, Hironaga ; Suto, Takeshi ; Sugimoto, Naotoshi ; Enomoto, Masanobu ; Ishikawa, Toshiaki ; Kashiwada, Tomomi ; Sugiyama, Masahiko ; Komatsu, Yoshito ; Okuyama, Hiroyuki ; Baba, Eishi ; Sakai, Daisuke ; Watanabe, Tomoki ; Tamura, Takao ; Yamashita, Kimihiro ; Gosho, Masahiko ; Shimada, Yasuhiro. / Propensity Score Analysis of Regorafenib Versus Trifluridine/Tipiracil in Patients with Metastatic Colorectal Cancer Refractory to Standard Chemotherapy (REGOTAS) : A Japanese Society for Cancer of the Colon and Rectum Multicenter Observational Study. In: Oncologist. 2018 ; Vol. 23, No. 1. pp. 7-15.
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abstract = "Background: This study compared the efficacy of regorafenib and trifluridine/tipiracil (TFTD) in patients with metastatic colorectal cancer (mCRC) who are refractory to standard chemotherapy, because despite their clinical approval, it still remains unclear which of these two drugs should be used as initial treatment. Materials and Methods: The clinical data of patients with mCRC who were treated with regorafenib or TFTD and those of drug-naive patients, between June 2014 and September 2015, were retrospectively collected from 24 institutions in Japan. Overall survival (OS) was evaluated using the Cox's proportional hazard models based on propensity score adjustment for baseline characteristics. Results: A total of 550 patients (223 patients in the regorafenib group and 327 patients in the TFTD group) met all criteria. The median OS was 7.9 months (95{\%} confidence interval [CI], 6.8–9.2) in the regorafenib group and 7.4 months (95{\%} CI, 6.6–8.3) in the TFTD group. The propensity score adjusted analysis showed that OS was similar between the two groups (adjusted hazard ratio [HR], 0.96; 95{\%} CI, 0.78–1.18). In the subgroup analysis, a significant interaction with age was observed. Regorafenib showed favorable survival in patients aged <65 years (HR, 1.29; 95{\%} CI, 0.98–1.69), whereas TFTD was favored in patients aged ≥65 years (HR, 0.78; 95{\%} CI, 0.59–1.03). Conclusion: No significant difference in OS between regorafenib and TFTD was observed in patients with mCRC. Although the choice of the drug by age might affect survival, a clearly predictive biomarker to distinguish the two drugs should be identified in further studies. Implications for Practice: Previous studies of patients with metastatic colorectal cancer refractory to standard chemotherapy had demonstrated that both regorafenib and trifluridine/tipiracil could result in increased overall survival compared with placebo, but there are no head-to-head trials. This large, multicenter, observational study retrospectively compared the efficacy of regorafenib and trifluridine/tipiracil in 550 patients with metastatic colorectal cancer refractory to standard chemotherapy who had access to both drugs. Although no difference in overall survival was found between the two drugs in adjusted analysis using propensity score, regorafenib showed favorable survival in patients aged <65 years, whereas trifluridine/tipiracil was favored in patients aged ≥65 years in the subgroup analysis.",
author = "Toshikazu Moriwaki and Shota Fukuoka and Hiroya Taniguchi and Atsuo Takashima and Yusuke Kumekawa and Takeshi Kajiwara and Kentaro Yamazaki and Taito Esaki and Chinatsu Makiyama and Tadamichi Denda and Hironaga Satake and Takeshi Suto and Naotoshi Sugimoto and Masanobu Enomoto and Toshiaki Ishikawa and Tomomi Kashiwada and Masahiko Sugiyama and Yoshito Komatsu and Hiroyuki Okuyama and Eishi Baba and Daisuke Sakai and Tomoki Watanabe and Takao Tamura and Kimihiro Yamashita and Masahiko Gosho and Yasuhiro Shimada",
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TY - JOUR

T1 - Propensity Score Analysis of Regorafenib Versus Trifluridine/Tipiracil in Patients with Metastatic Colorectal Cancer Refractory to Standard Chemotherapy (REGOTAS)

T2 - A Japanese Society for Cancer of the Colon and Rectum Multicenter Observational Study

AU - Moriwaki, Toshikazu

AU - Fukuoka, Shota

AU - Taniguchi, Hiroya

AU - Takashima, Atsuo

AU - Kumekawa, Yusuke

AU - Kajiwara, Takeshi

AU - Yamazaki, Kentaro

AU - Esaki, Taito

AU - Makiyama, Chinatsu

AU - Denda, Tadamichi

AU - Satake, Hironaga

AU - Suto, Takeshi

AU - Sugimoto, Naotoshi

AU - Enomoto, Masanobu

AU - Ishikawa, Toshiaki

AU - Kashiwada, Tomomi

AU - Sugiyama, Masahiko

AU - Komatsu, Yoshito

AU - Okuyama, Hiroyuki

AU - Baba, Eishi

AU - Sakai, Daisuke

AU - Watanabe, Tomoki

AU - Tamura, Takao

AU - Yamashita, Kimihiro

AU - Gosho, Masahiko

AU - Shimada, Yasuhiro

PY - 2018/1/1

Y1 - 2018/1/1

N2 - Background: This study compared the efficacy of regorafenib and trifluridine/tipiracil (TFTD) in patients with metastatic colorectal cancer (mCRC) who are refractory to standard chemotherapy, because despite their clinical approval, it still remains unclear which of these two drugs should be used as initial treatment. Materials and Methods: The clinical data of patients with mCRC who were treated with regorafenib or TFTD and those of drug-naive patients, between June 2014 and September 2015, were retrospectively collected from 24 institutions in Japan. Overall survival (OS) was evaluated using the Cox's proportional hazard models based on propensity score adjustment for baseline characteristics. Results: A total of 550 patients (223 patients in the regorafenib group and 327 patients in the TFTD group) met all criteria. The median OS was 7.9 months (95% confidence interval [CI], 6.8–9.2) in the regorafenib group and 7.4 months (95% CI, 6.6–8.3) in the TFTD group. The propensity score adjusted analysis showed that OS was similar between the two groups (adjusted hazard ratio [HR], 0.96; 95% CI, 0.78–1.18). In the subgroup analysis, a significant interaction with age was observed. Regorafenib showed favorable survival in patients aged <65 years (HR, 1.29; 95% CI, 0.98–1.69), whereas TFTD was favored in patients aged ≥65 years (HR, 0.78; 95% CI, 0.59–1.03). Conclusion: No significant difference in OS between regorafenib and TFTD was observed in patients with mCRC. Although the choice of the drug by age might affect survival, a clearly predictive biomarker to distinguish the two drugs should be identified in further studies. Implications for Practice: Previous studies of patients with metastatic colorectal cancer refractory to standard chemotherapy had demonstrated that both regorafenib and trifluridine/tipiracil could result in increased overall survival compared with placebo, but there are no head-to-head trials. This large, multicenter, observational study retrospectively compared the efficacy of regorafenib and trifluridine/tipiracil in 550 patients with metastatic colorectal cancer refractory to standard chemotherapy who had access to both drugs. Although no difference in overall survival was found between the two drugs in adjusted analysis using propensity score, regorafenib showed favorable survival in patients aged <65 years, whereas trifluridine/tipiracil was favored in patients aged ≥65 years in the subgroup analysis.

AB - Background: This study compared the efficacy of regorafenib and trifluridine/tipiracil (TFTD) in patients with metastatic colorectal cancer (mCRC) who are refractory to standard chemotherapy, because despite their clinical approval, it still remains unclear which of these two drugs should be used as initial treatment. Materials and Methods: The clinical data of patients with mCRC who were treated with regorafenib or TFTD and those of drug-naive patients, between June 2014 and September 2015, were retrospectively collected from 24 institutions in Japan. Overall survival (OS) was evaluated using the Cox's proportional hazard models based on propensity score adjustment for baseline characteristics. Results: A total of 550 patients (223 patients in the regorafenib group and 327 patients in the TFTD group) met all criteria. The median OS was 7.9 months (95% confidence interval [CI], 6.8–9.2) in the regorafenib group and 7.4 months (95% CI, 6.6–8.3) in the TFTD group. The propensity score adjusted analysis showed that OS was similar between the two groups (adjusted hazard ratio [HR], 0.96; 95% CI, 0.78–1.18). In the subgroup analysis, a significant interaction with age was observed. Regorafenib showed favorable survival in patients aged <65 years (HR, 1.29; 95% CI, 0.98–1.69), whereas TFTD was favored in patients aged ≥65 years (HR, 0.78; 95% CI, 0.59–1.03). Conclusion: No significant difference in OS between regorafenib and TFTD was observed in patients with mCRC. Although the choice of the drug by age might affect survival, a clearly predictive biomarker to distinguish the two drugs should be identified in further studies. Implications for Practice: Previous studies of patients with metastatic colorectal cancer refractory to standard chemotherapy had demonstrated that both regorafenib and trifluridine/tipiracil could result in increased overall survival compared with placebo, but there are no head-to-head trials. This large, multicenter, observational study retrospectively compared the efficacy of regorafenib and trifluridine/tipiracil in 550 patients with metastatic colorectal cancer refractory to standard chemotherapy who had access to both drugs. Although no difference in overall survival was found between the two drugs in adjusted analysis using propensity score, regorafenib showed favorable survival in patients aged <65 years, whereas trifluridine/tipiracil was favored in patients aged ≥65 years in the subgroup analysis.

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