Medical Genetics Department, Koç University School of Medicine (KUSoM), İstanbul, Turkey.
Department of Dermatology, Koç University School of Medicine (KUSoM), İstanbul, Turkey.
Am J Med Genet A. 2019 Jan;179(1):123-129. doi: 10.1002/ajmg.a.60686. Epub 2018 Dec 18.
Terminal osseous dysplasia with pigmentary defects (TODPD; MIM #300244) is an extremely rare, X-linked dominant, in utero male-lethal disease, characterized by skeletal dysplasia of the limbs, pigmentary defects of the skin, and recurrent digital fibromatosis of childhood. Delayed/abnormal ossification of bones of the hands and feet, joint contractures, and dysmorphic facial features may accompany. A single recurrent mutation (c.5217 G>A) of the FLNA gene which causes cryptic splicing was identified as the cause of the disease. We here present the first TODPD case from Turkey with full-blown phenotype who exhibit unique additional findings, hypopigmented patch on the lower extremity following Blaschko's lines and smooth muscle hamartoma of the scalp in review of all the previously reported TODPD cases.
伴骨发育不良和色素缺失的终末器官发育障碍(TODPD;MIM#300244)是一种极其罕见的 X 连锁显性、宫内致死性疾病,其特征为肢体骨骼发育不良、皮肤色素缺失以及儿童复发性指(趾)纤维瘤病。可伴有骨骼延迟/异常骨化、关节挛缩和面部畸形。FLNA 基因突变(c.5217G>A)导致剪接隐匿,被鉴定为该病的病因。我们在此介绍了首例来自土耳其的 TODPD 病例,该病例具有完整的表型,表现出独特的附加发现,即在 Blaschko 线以下的下肢出现色素减退斑和头皮平滑肌错构瘤,对所有之前报道的 TODPD 病例进行了回顾。