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Optimal timing of early thyroidectomy in young patients with MEN2A and its impact on disease outcome

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Autor(es):
Marmitt, Laura ; Valente, Flavia Oliveira Facuri ; Camacho, Cleber Pinto ; da Silveira Duval, Marta Amaro ; Lindsey, Susan Chow ; Zanella, Andre Borsatto ; Vaz, Carla Ferreira ; de Barros Maciel, Rui Monteiro ; Maia, Ana Luiza
Número total de Autores: 9
Tipo de documento: Artigo Científico
Fonte: ENDOCRINE; v. N/A, p. 10-pg., 2025-09-30.
Resumo

Background RET genetic screening identifies individuals at risk of hereditary medullary thyroid carcinoma. The optimal timing for thyroidectomy in gene carriers is debated. Methods Outcomes of MEN2A patients diagnosed at <= 20 years were evaluated. Results Of 104 patients, four were index cases, and 100 (96.2%) diagnosed by genetic screening. The mean age at diagnosis was 11.2 +/- 5.6 years, and 65.4% were female. The most common pathogenic variant occurred at codon 634 (70.2%). Excluding index cases, 83 patients underwent thyroidectomy, 15 were under active surveillance, and two awaited surgery. The mean age at surgery was 13.2 +/- 5.0 years, and the median preoperative basal serum calcitonin (preCTN) level was 28.0 pg/mL (9.9-60.0). Tumor size ranged 0.1-3.5 cm. After a mean period of 10.9 years, 74 (91.4%) patients achieved an excellent response, while 7 (8.6%) had persistent disease (4 with biochemical and 3 with structural disease). Variables associated with persistent disease included age at diagnosis, age at surgery, preCTN levels, and lymph node or distant metastasis. ROC analysis assessed preCTN level as a predictor of lymph node metastasis and persistent disease; optimal preCTN cutoff values were 28.5 pg/mL (100% sensitivity and 53% specificity) and 71.9 pg/mL (100% sensitivity and 88% specificity). Conclusions Most young MEN2A patients who underwent thyroidectomy later than recommended by guidelines were disease-free after long-term follow-up, suggesting the possibility of delaying surgery in selected RET carriers with low serum CTN levels. (AU)

Processo FAPESP: 21/02752-6 - Neoplasia Endócrina múltipla tipo 2 e Carcinoma Medular da Tiroide: novas questões da biologia do desenvolvimento, genética, imunologia, epidemiologia, mecanismo de doença e manejo clínico
Beneficiário:Rui Monteiro de Barros Maciel
Modalidade de apoio: Auxílio à Pesquisa - Temático