Distant Metastases From Childhood Differentiated Thyroid Carcinoma: Clinical Course and Mutational Landscape

Author:

Nies Marloes12ORCID,Vassilopoulou-Sellin Rena2,Bassett Roland L3ORCID,Yedururi Sireesha4ORCID,Zafereo Mark E5ORCID,Cabanillas Maria E2ORCID,Sherman Steven I2ORCID,Links Thera P1ORCID,Waguespack Steven G26ORCID

Affiliation:

1. Department of Endocrinology, Internal Medicine, University of Groningen, University Medical Center Groningen, GZ Groningen, the Netherlands

2. Department of Endocrine Neoplasia and Hormonal Disorders, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA

3. Department of Biostatistics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA

4. Department of Abdominal Imaging, University of Texas MD Anderson Cancer Center, Houston, Texas, USA

5. Department of Head and Neck Surgery, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA

6. Department of Pediatrics–Patient Care, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA

Abstract

Abstract Context Distant metastases (DM) from childhood differentiated thyroid carcinoma (DTC) are uncommon and published studies are limited. Objective This work aimed to describe the outcomes of patients with DM from childhood DTC and to evaluate the molecular landscape of these tumors. Methods A retrospective study was conducted at a tertiary cancer center including patients with pediatric DTC (diagnosed at age ≤ 18 years from 1946 to 2019) and DM. Results We identified 148 patients; 144 (97%) had papillary thyroid carcinoma (PTC) and 104 (70%) were female. Median age at DTC diagnosis was 13.4 years (interquartile range [IQR], 9.9-15.9 years). Evaluable individuals received a median of 2 (IQR, 1-3) radioactive iodine (RAI) treatments at a median cumulative administered activity of 238.0 mCi (IQR, 147.5-351.0 mCi). The oncogenic driver was determined in 64 of 69 PTC samples: RET fusion (38/64; 59%), NTRK1/3 fusions (18/64; 28%), and the BRAF V600E mutation (8/64; 13%). At last evaluation, 93% had persistent disease. The median overall and disease-specific survival after DTC diagnosis were 50.7 and 52.8 years, respectively. Eight (5%) PTC patients died of disease after a median of 30.7 years (IQR, 20.6-37.6 years). Conclusion Childhood DTC with DM persists in most patients despite multiple courses of RAI, but disease-specific death is uncommon, typically occurring decades after diagnosis. Fusion genes are highly prevalent in PTC, and all identified molecular alterations have appropriate targeted therapies. Future studies should focus on expanding genotype-phenotype correlations, determining how to integrate molecularly targeted therapy into treatment paradigms, and relying less on repeated courses of RAI to achieve cure in patients with DM from childhood DTC.

Funder

Junior Scientific Master Class Groningen

Prince Bernhard Culture

University of Texas MD Anderson Cancer Center

National Institutes of Health

National Cancer Institute Cancer Center

Publisher

The Endocrine Society

Subject

Biochemistry (medical),Clinical Biochemistry,Endocrinology,Biochemistry,Endocrinology, Diabetes and Metabolism

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