Nieschlag Eberhard, Werler Steffi, Wistuba Joachim, Zitzmann Michael
Centre of Reproductive Medicine and Andrology, University of Münster, D-48129 Münster, Germany; Center of Excellence in Genomic Medicine Research, King Abdulaziz University, Jeddah, Saudi Arabia.
Centre of Reproductive Medicine and Andrology, University of Münster, D-48129 Münster, Germany.
Ann Endocrinol (Paris). 2014 May;75(2):88-97. doi: 10.1016/j.ando.2014.03.007. Epub 2014 Apr 30.
The Klinefelter syndrome (KS), with an incidence of 1 to 2 per 1000 male neonates, is one of the most frequent congenital chromosome disorders. The 47,XXY karyotype causes infertility, testosterone deficiency and a spectrum of further symptoms and comorbidities. In recent years, significant progress has been made in the elucidation of the pathophysiology and the treatment of the KS. It became clear that, to a large extent, the clinical picture is determined by gene dosage effects of the supernumerary X-chromosome. The origin of the extra X-chromosome from either the father or the mother influences behavioural features of patients with KS. The CAGn polymorphism of the androgen receptor, located on the X-chromosome, has a distinct impact on the KS phenotype. KS predisposes to the metabolic syndrome and its cardiovascular sequelae, contributing to the increased mortality of patients with KS. Neuroimaging studies have correlated anomalies in brain structures with psychosocial problems. The unexpected possibility to produce pregnancies and live birth with either ejaculated sperm--about 8% of KS men have a few sperm in semen--or with sperm extracted from individual tubules obtained by testicular biopsy can be considered a breakthrough. Testosterone substitution requires further optimisation in terms of when to initiate therapy and which preparations and dosages to use. Recently developed animal models help to further elucidation the genetic and pathophysiological basis and may lead to new therapeutic approaches to KS.
克兰费尔特综合征(KS)的发病率为每1000例男性新生儿中有1至2例,是最常见的先天性染色体疾病之一。47,XXY核型会导致不育、睾酮缺乏以及一系列其他症状和合并症。近年来,在阐明KS的病理生理学和治疗方面取得了重大进展。很明显,很大程度上临床症状是由额外X染色体的基因剂量效应决定的。额外的X染色体来自父亲或母亲会影响KS患者的行为特征。位于X染色体上的雄激素受体的CAGn多态性对KS表型有明显影响。KS易患代谢综合征及其心血管后遗症,导致KS患者死亡率增加。神经影像学研究已将脑结构异常与心理社会问题联系起来。利用射出的精子(约8%的KS男性精液中有少量精子)或通过睾丸活检从单个小管中提取的精子实现怀孕和活产这种意想不到的可能性可被视为一项突破。睾酮替代疗法在何时开始治疗以及使用何种制剂和剂量方面需要进一步优化。最近开发的动物模型有助于进一步阐明遗传和病理生理基础,并可能带来针对KS的新治疗方法。