Sparling David P, Fabian Kendra, Harik Lara, Jobanputra Vaidehi, Anyane-Yeboa Kwame, Oberfield Sharon E, Fennoy Ilene
J Pediatr Endocrinol Metab. 2016 May 1;29(5):627-31. doi: 10.1515/jpem-2015-0253.
Thyroid dyshormonogenesis continues to be a significant cause of congenital hypothyroidism. Over time, forms of thyroid dyshormonogenesis can result in goiter, which can lead to difficult management decisions as the pathologic changes can both mimic or lead to thyroid cancer.
Herein we describe the cases of two brothers diagnosed with congenital hypothyroidism, with initial findings consistent with thyroid dyshormonogenesis. One brother eventually developed multinodular goiter with complex pathology on biopsy, resulting in thyroidectomy.
Whole exome sequencing revealed the brothers carry a novel frameshift mutation in thyroperoxidase; the mutation, while not previously described, was likely both deleterious and pathogenic. Conlcusions: These cases highlight the complex pathology that can occur within thyroid dyshormonogenesis, with similar appearance to possible thyroid cancer, leading to complex management decisions. They also highlight the role that a genetic diagnosis can play in interpreting the impact of dyshormonogenesis on nodular thyroid development, and the need for long-term follow-up in these patients.
甲状腺激素合成障碍仍然是先天性甲状腺功能减退症的一个重要病因。随着时间的推移,甲状腺激素合成障碍的形式可导致甲状腺肿大,由于病理变化既可能模仿甲状腺癌,也可能导致甲状腺癌,这会导致治疗决策困难。
在此,我们描述了两兄弟被诊断为先天性甲状腺功能减退症的病例,其初始检查结果与甲状腺激素合成障碍一致。其中一个兄弟最终发展为多结节性甲状腺肿,活检显示病理复杂,最终接受了甲状腺切除术。
全外显子组测序显示,这两兄弟在甲状腺过氧化物酶中携带一种新的移码突变;该突变虽然此前未被描述,但可能具有有害性和致病性。
这些病例凸显了甲状腺激素合成障碍可能出现的复杂病理情况,其外观与可能的甲状腺癌相似,导致了复杂的治疗决策。它们还凸显了基因诊断在解释激素合成障碍对结节性甲状腺发育的影响方面所起的作用,以及对这些患者进行长期随访的必要性。