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Vandetanib for the Management of Advanced Medullary Thyroid Cancer: A Real-World Multicenter Experience
Mijin Kim1, Jee Hee Yoon2, Jonghwa Ahn3
1Department of Internal Medicine, Biomedical Research Institute, Pusan National University Hospital, Busan, Korea.
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.
Insights
Vandetanib effectively treats advanced medullary thyroid cancer (MTC) in real-world practice, showing good objective response rates and progression-free survival. The drug was generally well-tolerated, with no fatal adverse events observed.
Area of Science:
- Oncology
- Pharmacology
Background:
- Limited real-world data exists for vandetanib in advanced medullary thyroid cancer (MTC).
- Vandetanib is a key tyrosine kinase inhibitor for MTC treatment.
Purpose of the Study:
- To evaluate the efficacy and safety of vandetanib in advanced MTC patients in routine clinical practice.
- To assess objective response rate (ORR), progression-free survival (PFS), and overall survival (OS).
Main Methods:
- Multicenter retrospective study of 12 advanced MTC patients treated with vandetanib.
- Assessed ORR, PFS, OS, and toxicities according to established criteria.
Main Results:
- Objective response rate (ORR) was 42% (partial response in 5 patients).
- An additional 5 patients (83% overall) had stable disease for ≥24 weeks.
- Median PFS was 25.9 months; no fatal adverse events occurred.
Conclusions:
- Vandetanib demonstrates efficacy in advanced MTC in real-world settings, consistent with clinical trials.
- The drug is generally well-tolerated, with manageable adverse events.
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