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

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.

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).

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.

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.

Cancer research and treatment : official journal of Korean Cancer Association. 2019 Mar 25 [Epub ahead of print]

Yeonjoo Choi, Bhumsuk Keam, Miso Kim, Shinkyo Yoon, Dalyong Kim, Jong Gwon Choi, Ja Young Seo, Inkeun Park, Jae Lyun Lee

Division of Medical Oncology, Department of Internal Medicine, Seoul National University Hospital, Seoul, Korea., Department of Oncology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea., Division of Hematology and Medical Oncology, Dongguk University Ilsan Hospital, Ilsan, Korea., Department of Internal Medicine, Konyang University Hospital, Daejeon, Korea., Department of Laboratory Medicine, Gachon University Gil Medical Center, Incheon, Korea., Division of Medical Oncology, Department of Internal Medicine, Gachon University Gil Medical Center, Incheon, Korea.

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