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Use of Gefitinib in EGFR-Amplified Refractory Solid Tumors: An Open-Label, Single-Arm, Single-Center Prospective Pilot Study

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Abstract

Background

Treatment options for patients with chemotherapy-refractory solid tumors are limited.

Objective

We conducted an open-label, single-arm, single-center phase II trial to evaluate the efficacy and safety of gefitinib in patients with chemotherapy-refractory solid tumors and EGFR amplification or sensitivity to an EGFR inhibitor identified through a drug-screening platform with patient-derived tumor cells (PDCs).

Patients and methods

EGFR amplification was detected by targeted sequencing. Sensitivity to an EGFR inhibitor was established in chemical screening using PDCs. Gefitinib (250 mg daily) was administered continuously in 28-day cycles until the occurrence of disease progression, unacceptable toxicity, or death due to any cause. The primary endpoint was the objective response rate (ORR).

Results

In total, 15 patients were assigned to the present study. The most common tumor type was glioblastoma multiforme (n = 9, 60%), followed by gastric cancer (n = 3, 20%), anal squamous cancer, rectal cancer, and sarcoma (each n = 1, 6.7%). Among 13 evaluable patients, one patient had a partial response and five had stable disease, with an ORR of 7.7% and a disease control rate of 46.1%. The median progression-free survival was 2.1 months (95% confidence interval [CI] 0.77–3.43). The most common adverse events were diarrhea (26.7%) and skin rash (26.7%).

Conclusion

Gefitinib demonstrated modest anti-tumor activity and a manageable safety profile in chemotherapy-refractory solid tumors with EGFR amplification or sensitivity to an EGFR inhibitor identified through a drug-screening platform with PDCs.

ClinicalTrials.gov identifier

NCT02447419.

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Authors and Affiliations

  1. Division of Hematology-Oncology, Department of Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, 81 Irwon-ro, Gangnam-gu, Seoul, 06351, South Korea

    Seonggyu Byeon, Jung Yong Hong, Jeeyun Lee, Se Hoon Park, Joon Oh Park, Young Suk Park, Ho Yeong Lim, Won Ki Kang & Seung Tae Kim

  2. Department of Neurosurgery, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, South Korea

    Do-Hyun Nam

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  1. Seonggyu Byeon
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Correspondence to Seung Tae Kim.

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Conflict of Interest

Seonggyu Byeon, Jung Yong Hong, Jeeyun Lee, Do-Hyun Nam, Se Hoon Park, Joon Oh Park, Young Suk Park, Ho Yeong Lim, Won Ki Kang, and Seung Tae Kim declare that they have no conflicts of interest that might be relevant to the contents of this article.

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Byeon, S., Hong, J.Y., Lee, J. et al. Use of Gefitinib in EGFR-Amplified Refractory Solid Tumors: An Open-Label, Single-Arm, Single-Center Prospective Pilot Study. Targ Oncol 15, 185–192 (2020). https://doi.org/10.1007/s11523-020-00706-0

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  • DOI: https://doi.org/10.1007/s11523-020-00706-0

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