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Bevacizumab Plus Erlotinib Combination Therapy for Advanced Hereditary Leiomyomatosis and Renal Cell Carcinoma-Associated Renal Cell Carcinoma: A Multicenter Retrospective Analysis in Korean Patients

Cancer Research and Treatment 2019년 51권 4호 p.1549 ~ 1556
 ( Choi Yeon-Joo ) - Seoul National University Hospital Department of Internal Medicine

 ( Keam Bhum-Suk ) - Seoul National University Hospital Department of Internal Medicine
 ( Kim Mi-So ) - Seoul National University Hospital Department of Internal Medicine
 ( Yoon Shin-Kyo ) - University of Ulsan College of Medicine Asan Medical Center Department of Oncology
 ( Kim Dal-Yong ) - Dongguk University Ilsan Hospital Division of Hematology and Medical Oncology
 ( Choi Jong-Gwon ) - Konyang University Hospital Department of Internal Medicine
 ( Seo Ja-Young ) - Gachon University Gil Medical Center Department of Laboratory Medicine
 ( Park In-Keun ) - Gachon University Gil Medical Center Department of Internal Medicine
 ( Lee Jae-Lyun ) - University of Ulsan College of Medicine Asan Medical Center Department of Oncology

Abstract


Purpose: Hereditary leiomyomatosis and renal cell carcinoma (HLRCC) is a rare genetic syndrome resulting from germline mutations in fumarate hydratase. The combination of bevacizumab plus erlotinib showed promising interim results for HLRCC-associated RCC. Based on these results, we analyzed the outcome of bevacizumab plus erlotinib in Korean patients with HLRCC-associated RCC.

Materials and Methods: We retrospectively reviewed the efficacy and safety of bevacizumab plus erlotinib in patients with HLRCC-associated RCC who were confirmed to have germline mutations in fumarate hydratase. The primary endpoint was the objective response rate (ORR), while the secondary endpoints were progression-free survival (PFS) and overall survival (OS).

Result: We identified 10 patients with advanced HLRCC-associated RCC who received bevacizumab plus erlotinib. Median age at diagnosis was 41 years, and five of the patients had received the combination as first- or second-line treatments. The ORR was 50% and the median PFS and OS were 13.3 and 14.1 months, respectively. Most adverse events were predictable and manageable by conventional measures, except for one instance where a patient died of gastrointestinal bleeding.

Conclusion: This is the first real-world outcome of the treatment of advanced HLRCC-associated RCC. Bevacizumab plus erlotinib therapy showed promising activity with moderate toxicity. We should be increasingly aware of HLRCC-associated RCC and bevacizumab plus erlotinib should be a first-line treatment for this condition, unless other promising data are published.

키워드

Hereditary leiomyomatosis and renal cell carcinoma; Bevacizumab; Erlotinib; Renal cell carcinoma; Fumarate hydratase; Non-clear cell
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