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Investigational New Drugs

, Volume 37, Issue 6, pp 1239–1246 | Cite as

A multicenter, prospective phase II trial of gemcitabine plus axitinib in patients with renal cell carcinoma with a predominant sarcomatoid component

  • Inkeun Park
  • Hyo Jin Lee
  • Woo Kyoon Bae
  • Shinkyo Yoon
  • Jae Lyun LeeEmail author
PHASE II STUDIES

Summary

Introduction We conducted a multicenter, phase 2 trial using gemcitabine plus axitinib (GX) in patients with recurrent or metastatic sarcomatoid renal cell carcinoma (SRCC) to evaluate its efficacy and safety. Methods Patients with advanced RCC and a sarcomatoid component of ≥25% on resected kidney or exclusive sarcomatoid carcinoma on needle biopsy were included. Patients received gemcitabine 1000 mg/m2 intravenously on days 1 and 8 of a 3-week cycle and axitinib 5 mg twice daily. Primary endpoint was objective response rate (ORR) according to the response evaluation criteria in solid tumors version 1.1, and secondary end points were progression-free (PFS) and overall (OS) survivals and adverse events. Results Twenty-five patients were enrolled. Median age was 61 (range: 33–80), and 84% were men. The Eastern Cooperative Oncology Group performance status was one in 23 patients (92%). Clear cell carcinoma was the most common histology of the carcinoma component (60%). ORR was 56%, and 28% patients achieved stable disease with a control rate of 84%. With a median follow-up duration of 24.8 months, the median PFS was 4.2 months (95% CI, 2.3–6.1) and median OS was 8.4 months (95% CI 3.3–13.4 months). The most common grade 3 or higher adverse events were neutropenia (36%), hypertension (12%), and anorexia (12%). Most adverse events were manageable, and no unexpected toxicities were found. Conclusion GX showed promising efficacy in patients with SRCC. GX could be considered as a treatment option for patients with SRCC and should be confirmed in larger clinical trials.

Keywords

Renal cell carcinoma Sarcomatoid Gemcitabine Axitinib Chemotherapy 

Notes

Acknowledgements

We thank Pfizer Korea and the Chong Kun Dang Pharmaceutical Corporation for their kind donation of axitinib and gemcitabine, respectively. This study was presented in part during the Genitourinary Cancers Symposium, 14-16 February 2019, in San Francisco, CA.

Compliance and ethical standards

Conflict of interest

Inkeun Park received honoraria for consulting or advisory roles from Pfizer Korea, Eisai Korea, Bristol-Myers Squibb pharma Korea, Astellas Pharma Korea, Janssen Korea, and iPSEN Korea. Jae Lyun Lee received honoraria for consulting or advisory roles from Pfizer Korea, Eisai Korea, Sanofi-Aventis Korea, Bristol-Myers Squibb Korea, Astellas Pharma, and Amgen Korea, and his institution received research funds from Pfizer, grants from Janssen, grants from Novartis, grants from Bristol-Myers Squibb, grants from Reche/Genentech, grants from AstraZeneca/MedImmune, grants from MSD.

Ethical approval

All procedures performed in studies involving human participants were in accordance with the ethical standards of the institutional and/or national research committee and with the 1964 Helsinki declaration and its later amendments or comparable ethical standards.

Informed consent

This study was approved by the institutional review board of each participating centers and Informed consent was obtained from all individual participants included in the study.

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Authors and Affiliations

  • Inkeun Park
    • 1
  • Hyo Jin Lee
    • 2
  • Woo Kyoon Bae
    • 3
  • Shinkyo Yoon
    • 4
  • Jae Lyun Lee
    • 4
    Email author
  1. 1.Division of Medical Oncology, Department of Internal Medicine, Gil Medical CenterGachon University College of MedicineIncheonRepublic of Korea
  2. 2.Department of Internal MedicineChungnam National University School of MedicineDaejeonRepublic of Korea
  3. 3.Department of Hemato-OncologyChonnam National University Hwasun HospitalHwasunRepublic of Korea
  4. 4.Department of Oncology, Asan Medical CenterUniversity of Ulsan College of MedicineSeoulRepublic of Korea

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