Snyder Emma A, San Roman Adrianna K, Piña-Aguilar Raul E, Steeves Marcie A, McNamara Erin A, Souter Irene, Hayes Frances J, Levitsky Lynne L, Lin Angela E
MassGeneral Hospital for Children, Medical Genetics, Department of Pediatrics, Boston, MA, USA.
Whitehead Institute for Biomedical Research, Cambridge, MA, USA.
Eur J Med Genet. 2021 Mar;64(3):104140. doi: 10.1016/j.ejmg.2021.104140. Epub 2021 Jan 29.
Despite numerous clinical series, consistent karyotype-phenotype correlations for Turner syndrome have not been established, although a lower level of 45,X is generally thought to be associated with a milder phenotype. This limits personalized counseling for women with 45,X/46,XX mosaicism. To better understand the phenotypic spectrum associated with various levels of 45,X/46,XX mosaicism, we compared patients evaluated in the Massachusetts General Hospital Turner Syndrome Clinic to determine if cardiac, renal, and thyroid abnormalities correlated with the percentage of 45,X cells present in a peripheral blood karyotype. of the 118 patients included in the study, 78 (66%) patients had non-mosaic 45,X and 40 (34%) patients had varying levels of 45,X/46,XX mosaicism. Patients with ≤70% 45,X compared with those with >70% 45,X had a significantly lower frequency of cardiac and renal anomalies. The presence of hypothyroidism was somewhat lower for the ≤70% 45,X group, but was not statistically significant. Supplemental tissue testing on another tissue type, typically buccal mucosa, was often useful in counseling patients with 45,X mosaicism. Given the modest sample size of patients with varying levels of mosaicism and the variability of Turner syndrome abnormalities, we hope this preliminary study will inspire a multicenter collaboration, which may lead to modification of clinical guidelines. Because several patients with ≤70% 45,X were ascertained from perinatal care referrals, we still advise women with 45,X mosaicism pursuing pregnancy to receive standard Turner syndrome cardiac surveillance. There is an opportunity to personalize counseling and surveillance for patients based on percentage of 45,X cells on chromosome analysis.
尽管有大量临床系列研究,但特纳综合征的核型与表型之间尚未建立一致的相关性,尽管一般认为45,X比例较低与较温和的表型相关。这限制了对45,X/46,XX嵌合体女性的个性化咨询。为了更好地了解与不同水平的45,X/46,XX嵌合体相关的表型谱,我们比较了在马萨诸塞州总医院特纳综合征诊所接受评估的患者,以确定心脏、肾脏和甲状腺异常是否与外周血核型中45,X细胞的百分比相关。在纳入研究的118例患者中,78例(66%)为非嵌合型45,X,40例(34%)为不同水平的45,X/46,XX嵌合体。与45,X>70%的患者相比,45,X≤70%的患者心脏和肾脏异常的发生率显著较低。45,X≤70%组甲状腺功能减退的发生率略低,但无统计学意义。对另一种组织类型(通常为颊黏膜)进行补充组织检测,在为45,X嵌合体患者提供咨询时通常很有用。鉴于嵌合水平不同的患者样本量较小以及特纳综合征异常的变异性,我们希望这项初步研究能激发多中心合作,这可能会导致临床指南的修订。由于部分45,X≤70%的患者是通过围产期护理转诊确诊的,我们仍然建议患有45,X嵌合体且正在备孕的女性接受标准的特纳综合征心脏监测。有机会根据染色体分析中45,X细胞的百分比为患者提供个性化咨询和监测。