Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Endocrinology Unit, Department of Clinical Sciences and Community Health, University of Milan, Milan, Italy.
Best Pract Res Clin Endocrinol Metab. 2018 Apr;32(2):141-154. doi: 10.1016/j.beem.2018.01.002. Epub 2018 Jan 31.
Metabolic disorders deriving from the non-responsiveness of target organs to hormones, which manifest clinically similar to the deficiency of a given hormone itself, derive from molecular alterations affecting specific hormone receptors. Pseudohypoparathyroidism (PHP) and related disorders exemplify an unusual form of hormone resistance as the underlying molecular defect is a partial deficiency of the α subunit of the stimulatory G protein (Gsα), a key regulator of cAMP signaling pathway, or, as more recently described, of downstream effector proteins of the same pathway, such as PKA regulatory subunit 1A (R1A) and phosphodyestarase type 4D (PDE4D). In this group of diseases, resistance to hormones such as PTH, TSH, gonadotropins and GHRH may be variably present, so that the clinical and molecular overlap among these different but related disorders represents a challenge for endocrinologists as to differential diagnosis and genetic counseling. This review will describe the presenting features of multiple resistance in PHP and related disorders, focusing on both our current understanding and future challenges.
由靶器官对激素无反应引起的代谢紊乱,其临床表现类似于特定激素本身的缺乏,源于影响特定激素受体的分子改变。假性甲状旁腺功能减退症 (PHP) 和相关疾病是一种不寻常的激素抵抗形式,其潜在的分子缺陷是刺激 G 蛋白 (Gsα) 的α亚单位的部分缺乏,这是 cAMP 信号通路的关键调节剂,或者,如最近所描述的,是同一通路的下游效应蛋白,如蛋白激酶 A 调节亚基 1A (R1A) 和磷酸二酯酶 4D (PDE4D)。在这群疾病中,对甲状旁腺激素 (PTH)、促甲状腺激素 (TSH)、促性腺激素和生长激素释放激素等激素的抵抗可能存在差异,因此这些不同但相关疾病的临床表现和分子重叠对内分泌学家在鉴别诊断和遗传咨询方面构成了挑战。这篇综述将描述 PHP 和相关疾病中多重抵抗的表现特征,重点介绍我们目前的理解和未来的挑战。