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两名泰萨二氏病患者的中枢性性早熟表现。

Presentation of central precocious puberty in two patients with Tay-Sachs disease.

机构信息

Division of Pediatric Endocrinology, Dokuz Eylul University School of Medicine, Izmir, Turkey.

Division of Pediatric Metabolism and Nutrition, Dokuz Eylul University School of Medicine, Izmir, Turkey.

出版信息

Hormones (Athens). 2018 Sep;17(3):415-418. doi: 10.1007/s42000-018-0043-3. Epub 2018 Jun 25.

Abstract

Tay-Sachs disease is an autosomal recessive type of lysosomal storage disorder. The disease is very rare in Turkey, with an incidence of 0.54/100,000. The clinical manifestations of Tay-Sachs disease include progressive developmental delay, seizures, deafness, blindness, spasticity, and dystonia, which are caused by the accumulation of gangliosides in the central nervous system. To date, only one case indicating the association between Tay-Sachs disease and central precocious puberty has been reported. Although the mechanism of this association is not clear, it is thought to be due to ganglioside accumulation in the central nervous system or the inhibition of the hypothalamic inhibiting pathway. Herein, we report two patients with genetically proven Tay-Sachs disease who developed central precocious puberty during follow-up. Pubertal development in patients affected by Tay-Sachs disease should be carefully assessed.

摘要

泰萨二氏病是一种常染色体隐性溶酶体贮积症。该病在土耳其非常罕见,发病率为 0.54/100000。泰萨二氏病的临床表现包括进行性发育迟缓、癫痫、耳聋、失明、痉挛和肌张力障碍,这些是由于神经节苷脂在中枢神经系统中的积累引起的。迄今为止,仅报道了一例表明泰萨二氏病与中枢性性早熟之间存在关联的病例。尽管这种关联的机制尚不清楚,但据认为是由于中枢神经系统中神经节苷脂的积累或下丘脑抑制途径的抑制。在此,我们报告了两名经基因证实患有泰萨二氏病的患者在随访期间发生中枢性性早熟。患有泰萨二氏病的患者的青春期发育应仔细评估。

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