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Vandetanib for the Management of Advanced Medullary Thyroid Cancer: A Real-World Multicenter Experience

Endocrinology and Metabolism 2020년 35권 3호 p.587 ~ 594
김미진, 윤지희, 안종화, 전민지, 김희경, 임동준, 강호철, 김인주, 송영기, 김태영, 김보현,
소속 상세정보
김미진 ( Kim Mi-Jin ) - Pusan National University Hospital Department of Internal Medicine
윤지희 ( Yoon Jee-Hee ) - Chonnam National University Hwasun Hospital Department of Internal Medicine
안종화 ( Ahn Jong-Hwa ) - University of Ulsan College of Medicine Asan Medical Center Department of Internal Medicine
전민지 ( Jeon Min-Ji ) - University of Ulsan College of Medicine Asan Medical Center Department of Internal Medicine
김희경 ( Kim Hee-Kyung ) - Chonnam National University Hwasun Hospital Department of Internal Medicine
임동준 ( Lim Dong-Jun ) - Catholic University College of Medicine Seoul St. Mary’s Hospital Department of Internal Medicine
강호철 ( Kang Ho-Cheol ) - Chonnam National University Hwasun Hospital Department of Internal Medicine
김인주 ( Kim In-Joo ) - Pusan National University Hospital Department of Internal Medicine
송영기 ( Shong Young-Kee ) - University of Ulsan College of Medicine Asan Medical Center Department of Internal Medicine
김태영 ( Kim Tae-Yong ) - University of Ulsan College of Medicine Asan Medical Center Department of Internal Medicine
김보현 ( Kim Bo-Hyun ) - Pusan National University Hospital Department of Internal Medicine

Abstract


Background: Vandetanib is the most widely used tyrosine kinase inhibitor for the treatment of patients with advanced medullary thyroid cancer (MTC). However, only limited data regarding its use outside clinical trials are available. We aimed to evaluate the efficacy and safety of vandetanib in patients with advanced MTC in routine clinical practice.

Methods: In this multicenter retrospective study, 12 patients with locally advanced or metastatic MTC treated with vandetanib at four tertiary hospitals were included. The primary outcome was the objective response rate (ORR) based on the Response Evaluation Criteria in Solid Tumors. The progression-free survival (PFS), overall survival (OS), and toxicities were also evaluated.

Results: Eleven patients (92%) had distant metastasis and 10 (83%) had disease progression at enrollment. Partial response was observed in five patients (ORR, 42%) and stable disease lasting ≥24 weeks was reported in an additional five patients (83%). During the median 31.7 months of follow-up, disease progression was seen in five patients (42%); of these, two died due to disease progression. The median PFS was 25.9 months, while the median OS was not reached. All patients experienced adverse events (AEs) which were generally consistent with the known safety profile of vandetanib. Vandetanib was discontinued in two patients due to skin toxicity.

Conclusion: Consistent with the phase III trial, this study confirmed the efficacy of vandetanib for advanced MTC in terms of both ORR and PFS in the real-world setting. Vandetanib was well tolerated in the majority of patients, and there were no fatal AEs.

키워드

Thyroid neoplasms; Protein kinase inhibitors; Progression-free survival; Toxicity

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