PMID- 32981301 OWN - NLM STAT- MEDLINE DCOM- 20210730 LR - 20220325 IS - 2093-5978 (Electronic) IS - 2093-596X (Print) IS - 2093-596X (Linking) VI - 35 IP - 3 DP - 2020 Sep TI - Vandetanib for the Management of Advanced Medullary Thyroid Cancer: A Real-World Multicenter Experience. PG - 587-594 LID - 10.3803/EnM.2020.687 [doi] AB - 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. FAU - Kim, Mijin AU - Kim M AD - Department of Internal Medicine, Biomedical Research Institute, Pusan National University Hospital, Busan, Korea. FAU - Yoon, Jee Hee AU - Yoon JH AD - Department of Internal Medicine, Chonnam National University Hwasun Hospital, Hwasun, Korea. FAU - Ahn, Jonghwa AU - Ahn J AD - Department of Internal Medicine, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea. FAU - Jeon, Min Ji AU - Jeon MJ AD - Department of Internal Medicine, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea. FAU - Kim, Hee Kyung AU - Kim HK AD - Department of Internal Medicine, Chonnam National University Hwasun Hospital, Hwasun, Korea. FAU - Lim, Dong Jun AU - Lim DJ AD - Department of Internal Medicine, Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea. FAU - Kang, Ho-Cheol AU - Kang HC AD - Department of Internal Medicine, Chonnam National University Hwasun Hospital, Hwasun, Korea. FAU - Kim, In Joo AU - Kim IJ AD - Department of Internal Medicine, Biomedical Research Institute, Pusan National University Hospital, Busan, Korea. FAU - Shong, Young Kee AU - Shong YK AD - Department of Internal Medicine, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea. FAU - Kim, Tae Yong AU - Kim TY AD - Department of Internal Medicine, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea. FAU - Kim, Bo Hyun AU - Kim BH AD - Department of Internal Medicine, Biomedical Research Institute, Pusan National University Hospital, Busan, Korea. LA - eng PT - Journal Article PT - Multicenter Study DEP - 20200922 PL - Korea (South) TA - Endocrinol Metab (Seoul) JT - Endocrinology and metabolism (Seoul, Korea) JID - 101554139 RN - 0 (Piperidines) RN - 0 (Protein Kinase Inhibitors) RN - 0 (Quinazolines) RN - YO460OQ37K (vandetanib) RN - Thyroid cancer, medullary SB - IM MH - Carcinoma, Neuroendocrine/*drug therapy/mortality/pathology MH - Disease-Free Survival MH - Female MH - Humans MH - Male MH - Middle Aged MH - Piperidines/adverse effects/*therapeutic use MH - Protein Kinase Inhibitors/adverse effects/*therapeutic use MH - Quinazolines/adverse effects/*therapeutic use MH - Republic of Korea MH - Retrospective Studies MH - Thyroid Neoplasms/*drug therapy/mortality/pathology PMC - PMC7520595 OTO - NOTNLM OT - Progression-free survival OT - Protein kinase inhibitors OT - Thyroid neoplasms OT - Toxicity COIS- CONFLICTS OF INTEREST No potential conflict of interest relevant to this article was reported. EDAT- 2020/09/29 06:00 MHDA- 2021/07/31 06:00 PMCR- 2020/09/01 CRDT- 2020/09/28 03:42 PHST- 2020/04/21 00:00 [received] PHST- 2020/07/16 00:00 [accepted] PHST- 2020/09/28 03:42 [entrez] PHST- 2020/09/29 06:00 [pubmed] PHST- 2021/07/31 06:00 [medline] PHST- 2020/09/01 00:00 [pmc-release] AID - EnM.2020.687 [pii] AID - enm-2020-687 [pii] AID - 10.3803/EnM.2020.687 [doi] PST - ppublish SO - Endocrinol Metab (Seoul). 2020 Sep;35(3):587-594. doi: 10.3803/EnM.2020.687. Epub 2020 Sep 22.