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扩展布鲁克综合征的表型:严重肢体畸形、关节挛缩、先天性心脏病和肺出血。

Expanding the phenotype of Bruck syndrome: Severe limb deformity, arthrogryposis, congenital cardiac disease and pulmonary hemorrhage.

作者信息

Sandy Jessica L, Perez Darazel, Goh Shuxiang, Forsey Jonathan, Rajagopalan Sulekha, Trivedi Amit, Munns Craig F

机构信息

Department of Endocrinology, The Children's Hospital at Westmead, Westmead, New South Wales, Australia.

School of Women's and Children, University of New South Wales, Sydney, New South Wales, Australia.

出版信息

Am J Med Genet A. 2023 Jan;191(1):265-270. doi: 10.1002/ajmg.a.63007. Epub 2022 Oct 25.

Abstract

Bruck syndrome is a rare collagen disorder with autosomal recessive inheritance caused by pathogenic variants in either FKBP10 or PLOD2 genes. It is characterized by bone fragility and fractures similar in severity and variability to osteogenesis-imperfecta as well as congenital joint contractures. This article describes an infant with a homozygous (partial) gene deletion of PLOD2 that includes the start codon and would be expected to lead to nonfunctional protein product. The infant had a severe phenotype of Bruck syndrome and is the only reported case of Bruck syndrome with congenital cardiac disease (triscuspid valve dysplasia with severe regurgitation, mitral valve prolapses with moderate regurgitation, and pulmonary hypertension) and pulmonary hemorrhage. We hypothesize that the additional feature of congenital cardiac disease in this case was due to the underlying defect in type I collagen, and that the pulmonary hemorrhage was multifactorial, with underlying vessel fragility, rib fractures, and high pulmonary pressures likely to be major contributing factors. Management was largely supportive with the use of bisphosphonates to assist in pain management. Care was complicated by comorbid cardiopulmonary compromise, limited evidence-base guiding care, and difficulties in discussing end-of-life care.

摘要

布鲁克综合征是一种罕见的胶原蛋白紊乱疾病,呈常染色体隐性遗传,由FKBP10或PLOD2基因的致病变异引起。其特征为骨脆性和骨折,严重程度和变异性与成骨不全相似,以及先天性关节挛缩。本文描述了一名患有PLOD2纯合(部分)基因缺失的婴儿,该缺失包括起始密码子,预计会导致无功能的蛋白质产物。该婴儿具有严重的布鲁克综合征表型,是唯一报告的患有先天性心脏病(三尖瓣发育异常伴严重反流、二尖瓣脱垂伴中度反流和肺动脉高压)和肺出血的布鲁克综合征病例。我们推测,该病例中先天性心脏病这一额外特征是由于I型胶原蛋白的潜在缺陷所致,而肺出血是多因素的,潜在的血管脆性、肋骨骨折和高肺压可能是主要促成因素。治疗主要是支持性的,使用双膦酸盐来辅助疼痛管理。合并的心肺功能不全、指导治疗的证据有限以及讨论临终关怀的困难使护理变得复杂。

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