col9a1相关疾病伴胸突,无骨骺发育不良:1例报告及文献复习。

IF 1.9 3区 医学 Q2 ORTHOPEDICS
Bukola A Olarewaju, Erin R Alexander, Monica M Crowe, Kristina Dandurand, David Melville, Fadi Shamoun, Mayowa A Osundiji
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引用次数: 0

摘要

COL9A1编码IX型胶原异源三聚体的α -1链,是透明软骨中胶原原纤维的重要组成部分。有初步的证据表明COL9A1突变可能与常染色体显性多发性骨骺发育不良(MED)有关,MED是一种影响长骨骨骺的疾病。在临床科学文献中,常染色体显性col9a1相关疾病(MIM 614135)中仅有2例报道(均来自同一家族),目前对其表型知之甚少。在这里,我们报告了与先前报道的病例相比,与新型COL9A1突变相关的临床和影像学发现。我们研究了一名22岁的男性,他有慢性腿痛和膝关节松弛史,有胸突病史,需要胸托,身高(身高= 186.9 cm),双侧有压源性脚丘疹。随后进行了基因小组测试和骨骼放射检查。基因面板检测显示COL9A1中存在一杂合致病变异,标记为c.188del (p.Phe63Serfs*3),预计会导致功能丧失。病人的长骨在x线片上都显得纤细,没有明显的骨骺发育不良。我们的研究结果表明,除了骨骺发育不良外,col9a1相关疾病的表型谱可能还包括其他骨骼异常(如胸状突和细长的长骨)。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
COL9A1-related disorder with pectus carinatum, without epiphyseal dysplasia: case report and review of literature.

COL9A1 encodes the alpha-1 chain of type IX collagen heterotrimer, which is a vital component of collagen fibrils in hyaline cartilage. There are preliminary lines of evidence suggesting that COL9A1 mutations may be associated with autosomal dominant multiple epiphyseal dysplasia (MED), a disorder affecting the epiphysis of long bones. With only 2 reported cases (both from the same family) of MED in autosomal dominant COL9A1-related disorders (MIM 614135) in the clinical scientific literature hitherto, the phenotype is poorly understood at present. Here, we report the clinical and imaging findings associated with a novel COL9A1 mutation in comparison to the previously reported cases. We studied a 22-year-old male, who presented with a chronic history of leg pain and laxity of the knee joint, in the context of a history of pectus carinatum requiring a chest brace, and tall stature (height = 186.9 cm) with bilateral piezogenic pedal papules. Gene panel testing and a radiographic skeletal survey were subsequently performed. Gene panel test showed a heterozygous pathogenic variant in the COL9A1 that is denoted as c.188del (p.Phe63Serfs*3) and is predicted to result in a loss-of-function. The patient's long bones all appeared slender on a radiograph, without apparent epiphyseal dysplasia. Our findings suggest that the phenotypic spectrum of COL9A1-related disorders may potentially include other skeletal anomalies (such as pectus carinatum and slender appearance of long bones) aside from epiphyseal dysplasia.

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来源期刊
Skeletal Radiology
Skeletal Radiology 医学-核医学
CiteScore
4.40
自引率
9.50%
发文量
253
审稿时长
3-8 weeks
期刊介绍: Skeletal Radiology provides a forum for the dissemination of current knowledge and information dealing with disorders of the musculoskeletal system including the spine. While emphasizing the radiological aspects of the many varied skeletal abnormalities, the journal also adopts an interdisciplinary approach, reflecting the membership of the International Skeletal Society. Thus, the anatomical, pathological, physiological, clinical, metabolic and epidemiological aspects of the many entities affecting the skeleton receive appropriate consideration. This is the Journal of the International Skeletal Society and the Official Journal of the Society of Skeletal Radiology and the Australasian Musculoskelelal Imaging Group.
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