Background: (rearranged during transfection) variants are the most prevalent oncogenic events in medullary thyroid cancer (MTC). In advanced disease, multi-tyrosine kinase inhibitors (MKIs) cabozantinib and vandetanib are the approved standard treatment irrespective of status. The actual outcome of patients with -positive MTC treated with MKIs is ill described.

Methods: We here retrospectively determined the oncogene variant status with a targeted DNA Custom Panel in a prospectively collected cohort of 48 patients with advanced MTC treated with vandetanib and/or cabozantinib at four German referral centers. Progression-free survival (PFS) and overall survival (OS) probabilities were estimated using the Kaplan-Meier method.

Results: In total, 44/48 (92%) patients had germline or somatic variants. The M918T variant was found in 29/44 (66%) cases. In total, 2/32 (6%) patients with a somatic variant had further somatic variants, while in 1/32 (3%) patient with a germline variant, additional variants were found. Only 1/48 (2%) patient had a pathogenic variant, and no variants were found in 3 cases. In first-line treatment, the median OS was 53 (95% CI (95% confidence interval), 32-NR (not reached); = 36), and the median PFS was 21 months (12-39; = 33) in -positive MTC patients. In second-line treatment, the median OS was 18 (13-79; = 22), and the median PFS was 3.5 months (2-14; = 22) in -positive cases.

Conclusions: variants were highly prevalent in patients with advanced MTC. The treatment results in -positive cases were similar to those reported in unselected cohorts.

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http://www.ncbi.nlm.nih.gov/pmc/articles/PMC9324961PMC
http://dx.doi.org/10.3390/cancers14143405DOI Listing

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