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Efficacy and safety of regorafenib in Japanese patients with advanced gastrointestinal stromal tumors

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Abstract

Background

Regorafenib is an oral multi-kinase inhibitor that has been established as third-line treatment for patients after the failure of imatinib and sunitinib. However, since clinical data of regorafenib in the Japanese population are still lacking, the management of regorafenib is mainly based on the clinical experience of each oncologist. The aim of this study was to evaluate the efficacy and safety of regorafenib in a Japanese population.

Methods

Thirty-three patients treated with regorafenib for metastatic and recurrent gastrointestinal stromal tumors were retrospectively enrolled. This study investigated the anti-tumor effect, including overall survival, progression-free survival, and safety, which was evaluated based on the incidence of adverse events.

Results

The median overall survival of patients treated with regorafenib was 23.8 months and the 1-year overall survival rate was 80.0%, the median progression-free survival was 7.1 months and the 1-year progression-free survival rate was 40.2%. The responses to regorafenib were partial response in 3 cases (9.1%), stable disease in 17 (51.5%), progressive disease in 10 (30.3%), and non-evaluable in 3 (9.1%). The disease control rate was 54.0%. Treatment-related adverse events were reported in all patients, with the most common being hand-foot syndrome (72.7%), followed by liver damage (36.4%) and diarrhea (27.3%), and six patients (20.0%) were discontinued due to adverse events.

Conclusion

This is the first report of Japanese patients with gastrointestinal stromal tumors treated with regorafenib. Regorafenib showed efficacy and a manageable safety profile in Japanese patients with advanced gastrointestinal stromal tumors, which was comparable with previous studies.

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Data availability

The data that support the findings of this study are available on request from the corresponding author, Tsuyoshi Takahashi. The data are not publicly available because they contain information that can compromise the privacy of research participants.

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Acknowledgements

We would like to thank Editage (www.editage.com) for English language editing.

Author information

Authors and Affiliations

  1. Department of Gastroenterological Surgery, Osaka University Graduate School of Medicine, 2-2, Yamadaoka, Suita-City, Osaka, 565-0871, Japan

    Ryugo Teranishi, Tsuyoshi Takahashi, Yukinori Kurokawa, Takuro Saito, Kazuyoshi Yamamoto, Kotaro Yamashita, Koji Tanaka, Tomoki Makino, Kiyokazu Nakajima, Hidetoshi Eguchi & Yuichiro Doki

  2. Department of Surgery, Japan Community Health Care Organization Osaka Hospital, 4-2-78, Fukushima-ku, Osaka City, Osaka, 553-0003, Japan

    Toshirou Nishida

  3. Department of Surgical Pathology, Hyogo College of Medicine, Hyogo, Japan

    Seiichi Hirota

  4. Department of Surgery, Osaka General Medical Center, 3-1-56, Bandai-Higashi, Sumiyoshi-ku, Osaka City, Osaka, 558-8558, Japan

    Masaaki Motoori

  5. Department of Surgery, Osaka International Cancer Institute, 3-1-69, Otemae, Chuo-ku, Osaka City, Osaka, 541-8567, Japan

    Takeshi Omori

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  1. Ryugo Teranishi
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Contributions

RT wrote the initial draft of the manuscript. TT contributed to the analysis and interpretation of the data and assisted in the preparation of the manuscript. All other authors contributed to data collection and interpretation, and critically reviewed the manuscript. All authors approved the final version of the manuscript and agreed to be accountable for all aspects of the work in ensuring that questions related to the accuracy or integrity of any part of the work are appropriately investigated and resolved.

Corresponding author

Correspondence to Tsuyoshi Takahashi.

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There are no conflicts of interest to declare.

Ethics approval and consent to participate

The Human Ethics Review Committee of Osaka University Graduate School of Medicine approved the protocol for this retrospective study, and each participant provided signed consent. All procedures were performed in accordance with the Declaration of Helsinki.

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Consent for publication was obtained from all individuals whose data appears in the paper.

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Teranishi, R., Takahashi, T., Nishida, T. et al. Efficacy and safety of regorafenib in Japanese patients with advanced gastrointestinal stromal tumors. Int J Clin Oncol 27, 1164–1172 (2022). https://doi.org/10.1007/s10147-022-02159-z

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