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Multicenter Study
. 2020 Sep;35(3):587-594.
doi: 10.3803/EnM.2020.687. Epub 2020 Sep 22.

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

Mijin Kim  1 , Jee Hee Yoon  2 , Jonghwa Ahn  3 , Min Ji Jeon  3 , Hee Kyung Kim  2 , Dong Jun Lim  4 , Ho-Cheol Kang  2 , In Joo Kim  1 , Young Kee Shong  3 , Tae Yong Kim  3 , Bo Hyun Kim  1
Affiliations
Multicenter Study

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

Mijin Kim et al. Endocrinol Metab (Seoul). 2020 Sep.

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.

Keywords: Progression-free survival; Protein kinase inhibitors; Thyroid neoplasms; Toxicity.

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Conflict of interest statement

CONFLICTS OF INTEREST

No potential conflict of interest relevant to this article was reported.

Figures

Fig. 1
Fig. 1
Best percent change in the total sum of target lesion diameters. PR, partial response; SD, stable disease.
Fig. 2
Fig. 2
Kaplan-Meier plots for (A) progression-free survival (PFS) and (B) overall survival (OS).
See this image and copyright information in PMC

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