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Endocrine Abstracts (2024) 101 OP12-03 | DOI: 10.1530/endoabs.101.OP-12-03

1Hospices Civils de Lyon, Hospices Civils de Lyon, Université Lyon 1, Endocrinology, Bron, France; 2Gustave Roussy and Endocan Tuthyref, [email protected], Villejuif, France; 3Hôpital Claude Huriez, Lille, France; 4Pitié Salpêtrière Hospital, Hôpitaux Universitaires Pitié Salpêtrière, Thyroid and Endocrine Tumor Unit, Paris, France; 5Centre Paul Strauss-Icans, Strasbourg, France; 6Centre Léon Bérard, Lyon, France; 7Institut du Thorax, Saint-Herblain, France; 8Centre Antoine Cassagne, Nice, France; 9Chu Larrey, Service D’ Endocrinologie, Department of Endocrinology and Metabolic Diseases, Chu Larrey, Toulouse Cedex 9, France; 10Institut Bergonié, Bordeaux, France; 11Centre François Baclesse, Nuclear Medicine, Caen, France; 12Centre Jean Perrin, Nuclear Medicine, Clermont-Ferrand, France; 13Saint Louis, Hôpital Saint Louis, Paris, France; 14Centre Universitaire du Cancer-Oncopole, Nuclear Medicine, Toulouse, France; 15Hôpital de la Cavale Blanche, Endocrinology, Brest, France; 16Chu D’angers, Service D’endocrinologie, Angers, France; 17Hospices Civils de Lyon, Lyon, France; 18Gustave Roussy, Villejuif, France; 19Gustave Roussy, Université Paris-Sud, Villejuif, France; 20Federation D’endocrinologie, Hopital Louis Pradel, Bron Cedex, France


Introduction: Vandetanib was the first tyrosine kinase inhibitor approved for the first line systemic treatment of progressive metastatic or locally advanced medullary thyroid carcinomas (MTC). Previous studies reported median duration of treatment (DOT) around 20 months with a subset of patients presenting long-term responses. Little is known on the impact of RET mutation status on the response duration.

Objectives: To evaluate the proportion and compare characteristics of vandetanib long term treated (LgTT) patients, vs short term treated patient (ShTT) in the MTC cohort of the French ENDOCAN-TUTHYREF network.

Method: Multicenter retrospective study on MTC patients with first line vandetanib treatment identified in the ENDOCAN-TUTHYREF database. Patients with less than 3 months treatment were considered non-evaluable and excluded from the analysis (n = 28). LgTT were defined as patients receiving Vandetanib more than 24 months Their characteristics were compared to that of patients treated less than 24 months (ShTT) using non parametric tests. Factors associated with overall survival (OS) were evaluated using log-rank test.

Results: From126 patients identified in the database, diagnosed between 1971 and 2022, who received vandetanib as first line systemic treatment (median follow-up 38 months (IQR 18-81)), 60% (71/118 evaluable) had RET mutation, 67%(85/126) were progressive at treatment initiation and 42% (53/126) were LgTT. Their median DOT was 49 months compared to 11 months in the ShTT group. Adverse events (AE) led to treatment discontinuation in 18/126 patients while 125 AE in 61 patients led to a dose decrease. Reasons for discontinuation were not different between the two groups. An objective response was observed in 38% of LgTT and 14% of ShTT (P-value=0.013). Lower ECOG (P-value=0.04), longer delay between diagnosis and treatment (P-value=0.02) were associated with LgTT; metachronous distant metastasis (P-value=0.05) and younger age at treatment (P-value=0.06) tended to be associated with LgTT, while RET mutation status (P-value=0.4, NA=8), sex, progressive disease at initiation, vandetanib dose and localization of metastasis were not associated with LgTT. OS since treatment initiation was longer in LgTT than in ShTT (90.7 (71.5-176.4) vs 26 (18.7-42.7) months in ShTT, P-value=4.10-7) while OS from treatment discontinuation was comparable between the 2 groups (17.7 (10.4-38.6) vs 12.9 months (8.85 -27), P-value=0.7).

Conclusion: Among MTC patients treated with first line vandetanib, long-term disease control was observed in 42% regardless RET mutation status, and associated with metachronous metastatic disease and longer delay between diagnosis and treatment.

Volume 101

46th Annual Meeting of the European Thyroid Association (ETA) 2024

European Thyroid Association 

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