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1a型假性甲状旁腺功能减退症中的生长激素缺乏:多激素抵抗的另一种表现。

Growth hormone deficiency in pseudohypoparathyroidism type 1a: another manifestation of multihormone resistance.

作者信息

Germain-Lee Emily L, Groman Joshua, Crane Janet L, Jan de Beur Suzanne M, Levine Michael A

机构信息

Department of Pediatrics, Division of Endocrinology and the Ilyssa Center for Molecular Endocrinology, The Johns Hopkins University School of Medicine, Baltimore, Maryland 21287, USA.

出版信息

J Clin Endocrinol Metab. 2003 Sep;88(9):4059-69. doi: 10.1210/jc.2003-030028.

Abstract

Albright hereditary osteodystrophy (AHO) is a genetic disorder caused by heterozygous inactivating mutations in GNAS1, the gene encoding the alpha-chain of G(s), and is associated with short stature, obesity, brachydactyly, and sc ossifications. AHO patients with GNAS1 mutations on maternally inherited alleles also manifest resistance to multiple hormones (e.g. PTH, TSH, LH, FSH), a variant termed pseudohypoparathyroidism (PHP) type 1a, due to paternal imprinting of G alpha(s) transcripts in specific tissues. Recent evidence has shown that G alpha(s) transcripts are also imprinted in the pituitary somatotrophs that secrete GH. Because this imprinting could influence GHRH-dependent stimulation of somatotrophs, we hypothesized that maternally inherited GNAS1 mutations would impair GH secretion. We studied GH status in 13 subjects with PHP type 1a. GH responses to arginine/L-dopa and arginine/GHRH were deficient in nine subjects, all of whom were obese and had low serum concentrations of IGF-I. By contrast, none of the four GH-sufficient subjects were obese, and all had normal IGF-I levels. Our data indicate that GH deficiency is common (69%) in PHP type 1a and may contribute to the obesity and short stature typical of AHO. We propose that GH status be evaluated in all patients with PHP type 1a.

摘要

奥尔布赖特遗传性骨营养不良(AHO)是一种遗传性疾病,由编码G(s)α链的基因GNAS1杂合失活突变引起,与身材矮小、肥胖、短指畸形和皮下骨化有关。母系遗传等位基因上携带GNAS1突变的AHO患者还表现出对多种激素(如甲状旁腺激素、促甲状腺激素、促黄体生成素、促卵泡生成素)的抵抗,这种变异被称为1a型假性甲状旁腺功能减退(PHP),这是由于特定组织中Gα(s)转录本的父系印记所致。最近的证据表明,Gα(s)转录本在分泌生长激素(GH)的垂体生长激素细胞中也存在印记。由于这种印记可能影响生长激素释放激素(GHRH)对生长激素细胞的刺激作用,我们推测母系遗传的GNAS1突变会损害GH分泌。我们研究了13例1a型PHP患者的GH状态。9例患者对精氨酸/左旋多巴和精氨酸/GHRH的GH反应不足,所有这些患者均肥胖且血清胰岛素样生长因子-I(IGF-I)浓度较低。相比之下,4例GH充足的患者均不肥胖,且所有患者的IGF-I水平均正常。我们的数据表明,GH缺乏在1a型PHP中很常见(69%),可能是导致AHO典型的肥胖和身材矮小的原因。我们建议对所有1a型PHP患者进行GH状态评估。

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