Işık Emregül, Beck Peccoz Paolo, Campi Irene, Özön Alev, Alikaşifoğlu Ayfer, Gönç Nazlı, Kandemir Nurgün
Division of Pediatric Endocrinology, Department of Pediatrics, Hacettepe University Faculty of Medicine, Ankara, Turkey.
Turk J Pediatr. 2013 May-Jun;55(3):322-7.
Thyroid hormone resistance (THR) is a dominantly inherited syndrome characterized by reduced sensitivity to thyroid hormones. It is usually caused by mutations in the thyroid hormone receptor beta (THRB) gene. In the present report, we describe the clinical and laboratory characteristics and genetic analysis of patients with a novel THRB gene mutation. The index patient had been misdiagnosed as hyperthyroidism and treated with antithyroid drugs since eight days of age. Thyroid hormone results showed that thyrotropin (thyroid-stimulating hormone, TSH) was never suppressed despite elevated thyroid hormone levels, and there was no symptom suggesting hyperthyroidism. A heterozygous mutation at codon 350 located in exon 9 of the THRB gene was detected in all the affected members of the family. It is important to consider thyroid hormone levels in association with TSH levels to prevent inappropriate treatment and the potential complications, such as clinical hypothyroidism or an increase in goiter size.
甲状腺激素抵抗(THR)是一种常染色体显性遗传综合征,其特征为对甲状腺激素的敏感性降低。它通常由甲状腺激素受体β(THRB)基因突变引起。在本报告中,我们描述了具有新型THRB基因突变患者的临床和实验室特征以及基因分析。索引患者自8日龄起被误诊为甲状腺功能亢进症并接受抗甲状腺药物治疗。甲状腺激素检测结果显示,尽管甲状腺激素水平升高,但促甲状腺激素(甲状腺刺激激素,TSH)从未被抑制,且没有提示甲状腺功能亢进症的症状。在该家族所有受影响成员中均检测到位于THRB基因第9外显子的第350密码子处的杂合突变。为防止不适当的治疗以及潜在并发症,如临床甲状腺功能减退症或甲状腺肿大小增加,结合TSH水平考虑甲状腺激素水平很重要。