非裔美国/波多黎各儿童的2型罗斯蒙-汤姆森综合征在不同人群中表现出诊断挑战。

IF 1.7 4区 生物学 Q3 GENETICS & HEREDITY
Eden Teferedegn, Kosuke Izumi, Rebecca Ahrens-Nicklas, Elizabeth Bhoj, Alyssa Rippert
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引用次数: 0

摘要

罗森德-汤姆逊综合征(RTS)是一种罕见的遗传病,其特征是皮损、毛发稀疏、身材矮小、骨骼异常、白内障和恶性肿瘤风险增加。主要症状通常是典型的皮疹,伴有脸颊和面部的红斑,并蔓延到四肢的伸肌表面。经过数月至数年,这种皮疹逐渐发展为千皮病(网状色素沉着、毛细血管扩张和点状萎缩)。识别这种特征性皮疹可能有助于RTS患者的早期诊断和治疗;然而,对于来自不同人群的患者,尤其是肤色较深的患者,这可能更具挑战性。在此,我们报告了一个4岁11个月大的非裔美国/波多黎各男性,其表现为进食困难,皮肤鳞片,毛发稀疏,身材矮小,远视和发育迟缓。最初,他的四肢上的千皮病被认为是牛皮癣样皮炎。外显子组测序显示,在RECQL4基因c.1568_1573delGCCCCTinsCCCCC (p.Ser523fs)和c.2412_2420delGGCCGGGCG (p.Ala805_Arg807del)中发现复合杂合致病变异,证实诊断为RTS 2型。本报告强调了RTS在不同人群中误诊或延迟诊断的风险,并强调了综合分子评估在诊断和管理罕见疾病中的重要性。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Rothmund-Thomson Syndrome Type 2 in an African American/Puerto Rican Child Demonstrates Diagnostic Challenges in Diverse Population.

Rothumnd-Thomson syndrome (RTS) is a rare genetic condition characterized by poikiloderma, sparse hair, short stature, skeletal abnormalities, cataracts, and increased risk for malignancies. The presenting symptom is often a classic rash with erythema on the cheeks and face with spread to extensor surfaces of extremities. Gradually over months to years, this rash develops into poikiloderma (reticulated hyper- and hypopigmentation, telangiectasias, and areas of punctate atrophy). Identification of this characteristic rash may facilitate early diagnosis and management in patients with RTS; however, this may be more challenging in patients from diverse populations, especially with darker skin. Herein, we report an African-American/Puerto Rican 4-year, 11-month-old male who presented with feeding difficulties, scaly patches of skin, sparse hair, short stature, hypertelorism, and developmental delay. Initially, poikiloderma on his extremities was considered to be psoriasiform dermatitis. Exome sequencing revealed compound heterozygous pathogenic variants in the RECQL4 gene, c.1568_1573delGCCCCTinsCCCCC (p.Ser523fs) and c.2412_2420delGGCCGGGCG (p.Ala805_Arg807del) confirming a diagnosis of RTS type 2. This report underscores the risk of misdiagnosis or delayed diagnoses of RTS in diverse populations and highlights the importance of comprehensive molecular evaluation in diagnosing and managing rare conditions.

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来源期刊
CiteScore
3.50
自引率
5.00%
发文量
432
审稿时长
2-4 weeks
期刊介绍: The American Journal of Medical Genetics - Part A (AJMG) gives you continuous coverage of all biological and medical aspects of genetic disorders and birth defects, as well as in-depth documentation of phenotype analysis within the current context of genotype/phenotype correlations. In addition to Part A , AJMG also publishes two other parts: Part B: Neuropsychiatric Genetics , covering experimental and clinical investigations of the genetic mechanisms underlying neurologic and psychiatric disorders. Part C: Seminars in Medical Genetics , guest-edited collections of thematic reviews of topical interest to the readership of AJMG .
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