Department of Clinical Genetics, Maastricht UMC, Maastricht, The Netherlands.
Eur J Hum Genet. 2010 Sep;18(9):993-8. doi: 10.1038/ejhg.2010.67. Epub 2010 May 12.
The Prader-Willi syndrome (PWS) is a genetic disorder caused by the absent expression of the paternal copy of maternally imprinted genes in chromosome region 15q11-13. The frequencies of different subtypes in PWS are usually given in literature as 70% deletion, 25-30% maternal uniparental disomy (mUPD) and 3-5% others (imprinting centre (IC) defects and translocations). Little is known about factors that influence the frequency of genetic subtypes in PWS. The study sample comprised 102 adults with clinically and genetically confirmed PWS, contacted through the Dutch Prader-Willi Parent Association and through physicians specialized in treating persons with intellectual disabilities. Genetic testing showed 55 persons (54%) with a paternal deletion, 44 persons (43%) with an mUPD and 3 persons (3%) with a defect of the IC. The observed distribution in our study differed from that in literature (70% deletion, 30% mUPD), which was statistically significant (z-score: P<0.05). This was mainly caused by a higher proportion of mUPD in the advanced age groups. Differences in maternal age and BMI of persons with PWS could not explain the differences in distribution across the age groups. Our study population had a much broader age range, compared with other studies, because of a predominance of elderly people (40+ years) with PWS. In other studies, these elderly persons might have been undiagnosed and/or underreported because of a lack of genetic diagnosis. The results underline both the need for correct genetic diagnosis in all persons with PWS and adjustment of the guidelines for preventive management in adulthood.
普拉德-威利综合征(PWS)是一种由染色体 15q11-13 区域母源印迹基因父源拷贝缺失引起的遗传疾病。文献中通常给出 PWS 的不同亚型频率为 70%缺失、25-30%母源单亲二体(mUPD)和 3-5%其他(印迹中心(IC)缺陷和易位)。关于影响 PWS 遗传亚型频率的因素知之甚少。研究样本包括通过荷兰普拉德-威利父母协会和专门治疗智力障碍患者的医生联系的 102 名经临床和基因确诊的 PWS 成人。基因检测显示 55 名(54%)患者存在父源缺失,44 名(43%)患者存在 mUPD,3 名(3%)患者存在 IC 缺陷。我们的研究观察到的分布与文献中的分布不同(70%缺失,30% mUPD),这具有统计学意义(z 分数:P<0.05)。这主要是由于高龄组 mUPD 比例较高所致。PWS 患者的母亲年龄和 BMI 的差异不能解释年龄组之间的分布差异。与其他研究相比,我们的研究人群年龄范围更广,因为患有 PWS 的老年人(40 岁以上)居多。在其他研究中,由于缺乏基因诊断,这些老年人可能未被诊断和/或未被报告。这些结果强调了对所有 PWS 患者进行正确基因诊断的必要性,以及对成年期预防管理指南进行调整的必要性。