Suppr超能文献

GH 成功治疗了一名新发 46,XX,add(X)(p36),t(X;Y)(p36.3;p11.2)、生长障碍和 SHOX 单倍体不足的女性。

GH successful treatment in a female with a de novo 46,XX,add(X)(p36),t(X;Y)(p36.3;p11.2), growth impairment and SHOX-haploinsufficiency.

机构信息

Department of Health Promotion Sciences Maternal and Infantile Care, Internal Medicine and Medical Specialities "G. D'Alessandro", University of Palermo, Palermo, Italy.

出版信息

Ital J Pediatr. 2019 Aug 14;45(1):100. doi: 10.1186/s13052-019-0694-y.

Abstract

Children with chromosome translocations, concerning X chromosome, have a genetic pattern different from Turner syndrome; however, when a translocation involves the of part of X chromosome including short stature homeobox-containing Sex-determining Region Y gene, growth may be severely compromised.We describe the clinical case of a 2.2-year-old-female, arrived at our paediatric unit for a decrease of height velocity. The karyotype was 46,XX,add(X)(p36.3). Array comparative genomic hybridization showed a fragment of Y chromosome, extended from 8.803.981 (Yp11.2) to 28.767.604 (Yq11.23). The final karyotype was 46,XX,add(X)(p36),t(X;Y)(p36.3;p11.2). Fluorescence in situ Hybridization analysis using Sex-determining Region Y probe revealed no signal on the derivative Y chromosome.At the admittance, height was 84.5 cm (- 1.24 SDS); SPAN was 79 cm; sitting height: 72.4 cm; weight was 17.5 kg. Bone age was 1.2 years. Multiplex Ligation Probe Amplification showed a heterozygous deletion of the Short Stature Homeobox-containing gene and of the pseudoautosomal region-1. This result correlated with Leri-Weill Syndrome. She started Growth Hormone treatment, with a good response.The case described shows a rare translocation, involving the X chromosome and including SHOX gene and the pseudoautosomal region-1. At our knowledge, this is the first case of a patient with a karyotype 46,XX,add(X)(p36),t(X;Y)(p36.3;p11.2) and Short Stature Homeobox-containing gene haploinsufficiency, successfully treated with Growth Hormone.

摘要

患有 X 染色体易位的儿童具有不同于特纳综合征的遗传模式;然而,当易位涉及 X 染色体的部分包括短臂同源盒基因和性决定区 Y 基因时,生长可能会受到严重影响。我们描述了一名 2.2 岁女性的临床病例,她因身高生长速度下降而来到我们的儿科病房。该患者的核型为 46,XX,add(X)(p36.3)。比较基因组杂交阵列显示,一条来自 Y 染色体的片段,从 8.803.981(Yp11.2)延伸至 28.767.604(Yq11.23)。最终的核型为 46,XX,add(X)(p36),t(X;Y)(p36.3;p11.2)。使用性决定区 Y 探针的荧光原位杂交分析显示,衍生 Y 染色体上没有信号。入院时,身高为 84.5cm(-1.24SDS);SPAN 为 79cm;坐高为 72.4cm;体重为 17.5kg。骨龄为 1.2 岁。多重连接探针扩增显示,短臂同源盒基因和假常染色体区域 1 存在杂合性缺失。这一结果与 Leri-Weill 综合征相关。她开始接受生长激素治疗,反应良好。所描述的病例显示了一种罕见的易位,涉及 X 染色体,包括 SHOX 基因和假常染色体区域 1。据我们所知,这是首例 X 染色体 46,XX,add(X)(p36),t(X;Y)(p36.3;p11.2)和短臂同源盒基因单倍不足的患者,成功接受了生长激素治疗。

相似文献

3
Prevalence of SHOX haploinsufficiency among short statured children.
Pediatr Res. 2017 Feb;81(2):335-341. doi: 10.1038/pr.2016.233. Epub 2016 Nov 4.
4
Translocation (Y;22) resulting in the loss of SHOX and isolated short stature.
Am J Med Genet A. 2004 Mar 1;125A(2):186-90. doi: 10.1002/ajmg.a.20346.
5
SHOX gene deletion screening by FISH in children with short stature and Madelung deformity and their characteristics.
J Pediatr Endocrinol Metab. 2018 Nov 27;31(11):1273-1278. doi: 10.1515/jpem-2018-0038.
8
9
Acute lymphoblastic leukemia in a child with Leri-Weill syndrome and complete SHOX gene deletion: A Case Report.
Biomed Pap Med Fac Univ Palacky Olomouc Czech Repub. 2018 Mar;162(1):65-70. doi: 10.5507/bp.2018.002. Epub 2018 Feb 21.
10
Trisomy of the short stature homeobox-containing gene (SHOX), resulting from a duplication-deletion of the X chromosome.
Clin Endocrinol (Oxf). 2002 May;56(5):671-5. doi: 10.1046/j.1365-2265.2002.01504.x.

引用本文的文献

1
Efficacy and Safety of Growth Hormone (GH) Therapy in Patients with SHOX Gene Variants.
Children (Basel). 2025 Mar 4;12(3):325. doi: 10.3390/children12030325.

本文引用的文献

2
Sex Assignment in Conditions Affecting Sex Development.
J Clin Res Pediatr Endocrinol. 2017 Dec 30;9(Suppl 2):106-112. doi: 10.4274/jcrpe.2017.S009. Epub 2017 Dec 27.
4
Impaired GH secretion in patients with SHOX deficiency and efficacy of recombinant human GH therapy.
Horm Res Paediatr. 2012;78(5-6):279-87. doi: 10.1159/000345354. Epub 2012 Nov 28.
5
Short stature due to SHOX deficiency: genotype, phenotype, and therapy.
Horm Res Paediatr. 2011 Feb;75(2):81-9. doi: 10.1159/000324105. Epub 2011 Feb 4.
6
Genotypes and phenotypes in children with short stature: clinical indicators of SHOX haploinsufficiency.
J Med Genet. 2007 May;44(5):306-13. doi: 10.1136/jmg.2006.046581. Epub 2006 Dec 20.
7
Italian cross-sectional growth charts for height, weight and BMI (2 to 20 yr).
J Endocrinol Invest. 2006 Jul-Aug;29(7):581-93. doi: 10.1007/BF03344156.

文献AI研究员

20分钟写一篇综述,助力文献阅读效率提升50倍。

立即体验

用中文搜PubMed

大模型驱动的PubMed中文搜索引擎

马上搜索

文档翻译

学术文献翻译模型,支持多种主流文档格式。

立即体验