一名被误诊为分泌促甲状腺激素垂体腺瘤的患者因 THRB 基因突变而对甲状腺激素 Beta 产生抗药性。

Wenjun Liao, Nipawan Waisayanand, Kanda Fanhchaksai, W Edward Visser, Marcel E Meima, Karn Wejaphikul
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引用次数: 0

摘要

T3和T4浓度升高的同时TSH不受抑制,这在产生TSH的肿瘤和甲状腺激素β抵抗(RTHβ)中均可发现,这给诊断带来了挑战。我们在此展示了一名 54 岁女性的病例,她出现心悸、甲状腺肿大、游离 T4 升高,但 TSH 浓度未受抑制(TSH 2.2 mIU/L [正常范围,NR 0.27-4.2 mIU/L],FT4 59.08 pmol/L [NR 12.0-22.0 pmol/L])。由于磁共振成像显示垂体微腺瘤(4 毫米),她被诊断为分泌促甲状腺激素的垂体腺瘤,并接受了经蝶手术。病理报告显示没有肿瘤细胞。随后的基因检测发现,THRB 基因中存在一个致病变体,导致 T3 受体同工酶 beta 1(TRβ1)中出现 His435Arg 氨基酸置换,提示为 RTHβ。体外和体内研究发现,His435Arg突变的TRβ1(TRβ1-H435R)完全取消了T3诱导的转录激活、核受体核心抑制因子1释放、类固醇受体辅助激活因子1招募和T3诱导的甲状腺激素靶基因表达,证实了该变体的致病性。在一名RTHβ患者身上发现垂体微腺瘤,导致误诊为TSH生成肿瘤,并进行了不必要的手术。基因检测被证明是准确诊断的关键,建议在类似的临床情况下尽早考虑。
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Resistance to Thyroid Hormone Beta Due to THRB Mutation in a Patient Misdiagnosed With TSH-Secreting Pituitary Adenoma.

Elevated concentrations of T3 and T4 concomitant with nonsuppressed TSH are found in both TSH-producing tumors and resistance to thyroid hormone beta (RTHβ), posing a diagnostic challenge. We demonstrate here a 54-year-old female who presented with palpitations, goiter, and elevated free T4 with nonsuppressed TSH concentrations (TSH 2.2 mIU/L [normal range, NR 0.27-4.2 mIU/L] and FT4 59.08 pmol/L [NR 12.0-22.0 pmol/L]). Because magnetic resonance imaging revealed a pituitary microadenoma (4 mm), she was diagnosed with TSH-secreting pituitary adenoma and underwent transsphenoidal surgery. Pathological reports showed no tumor cells. Subsequent genetic testing revealed a pathogenic variant in the THRB gene resulting in a His435Arg amino acid substitution in the T3 receptor isoform beta 1 (TRβ1), suggestive of RTHβ. In vitro and ex vivo studies revealed that the His435Arg mutated TRβ1 (TRβ1-H435R) completely abolishes the T3-induced transcriptional activation, nuclear receptor corepressor 1 release, steroid receptor coactivator 1 recruitment, and T3-induced thyroid hormone target gene expression, confirming the pathogenicity of this variant. The identification of a pituitary microadenoma in a patient with RTHβ led to a misdiagnosis of a TSH-producing tumor and unnecessary surgery. Genetic testing proved pivotal for an accurate diagnosis, suggesting earlier consideration in similar clinical scenarios.

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