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多中心性骨质溶解、结节病和关节病的一种新型基因突变:病例报告及文献复习

A novel gene mutation for multicentric osteolysis nodulosis and arthropathy: Case report and review of literature.

作者信息

Shakiba Marjan, Alaei Fariba

机构信息

Pediatric Endocrinology and Metabolism Department, Mofid Children's Hospital, Faculty of Medicine, Shahid Beheshti University of Medical Sciences, Tehran, Iran.

Pediatric Cardiology Department, Mofid Children's Hospital, Faculty of Medicine, Shahid Beheshti University of Medical Sciences, Tehran, Iran.

出版信息

Heliyon. 2023 Mar 24;9(4):e14865. doi: 10.1016/j.heliyon.2023.e14865. eCollection 2023 Apr.

Abstract

Frank-Ter Haar syndrome (FTHS), Winchester syndrome (WS), Torg syndrome (TS) and Multicentric Osteolysis Nodulosis and Arthropathy (MONA) are progressive skeletal dysplasia consisting of acro-osteolysis. Mutation in Matrix Metalloproteinase 2 (MMP2), Matrix Metalloproteinase 14 (MMP14) and SH3PXD2B are known genetic defects in these disorders. We hereby report a 5 years and 9 months old girl suffering from progressive limb deformity. She is the first child of a relative couple, who was referred to metabolic disorders' clinic due to poor growth and bone pain. On physical examination, minor facial dysmorphism, hypertrichosis, severe hand deformity with limitation in range of motion in carpal, metacarpal and phalangeal joints, hallux valgus deformity of feet, soft tissue hypertrophy and nodule formation in palmoplantar areas were detected. Her past history indicated a cardiac defect resulting in open heart surgery at 8 months of age. Genetic study revealed a new homozygote nonsense mutation in MMP2 gene explaining her clinical manifestations. We recommend careful evaluation and follow-up of patients with congenital heart disease, as it may be the first presentation of a genetic multisystem disorder. Early differentiation of the disease from other skeletal dysplasia and rheumatologic disorders could prevent unnecessary management.

摘要

弗兰克 - 特哈尔综合征(FTHS)、温彻斯特综合征(WS)、托格综合征(TS)和多中心骨质溶解结节病与关节病(MONA)是由肢端骨质溶解组成的进行性骨骼发育异常。基质金属蛋白酶2(MMP2)、基质金属蛋白酶14(MMP14)和SH3PXD2B的突变是这些疾病已知的遗传缺陷。我们在此报告一名5岁9个月大的患有进行性肢体畸形的女孩。她是一对近亲夫妇的第一个孩子,因生长发育不良和骨痛被转诊至代谢紊乱门诊。体格检查发现轻微面部畸形、多毛症、严重手部畸形,腕关节、掌指关节和指关节活动范围受限,足部拇外翻畸形,手掌足底软组织肥大和结节形成。她的既往史显示曾在8个月大时因心脏缺陷接受心脏直视手术。基因研究发现MMP2基因有一个新的纯合无义突变,解释了她的临床表现。我们建议对先天性心脏病患者进行仔细评估和随访,因为这可能是一种遗传性多系统疾病的首发表现。早期将该疾病与其他骨骼发育异常和风湿性疾病区分开来可以避免不必要的治疗。

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