Falahati Vahid, Fallahi Mahsa, Shahriarpour Mona, Ghasemi Ali, Ghaffari Kazem
Department of Pediatric Hematology and Oncology, Faculty of Medicine, Arak University of Medical Sciences, Arak, Iran.
Department of Dermatology, Faculty of Medicine, Arak University of Medical Sciences, Arak, Iran.
Adv Biomed Res. 2024 Feb 26;13:20. doi: 10.4103/abr.abr_232_23. eCollection 2024.
Klippel Trenaunay Syndrome (KTS) is an uncommon inherited syndrome identified by venous varicosities and capillary abnormalities. von Willebrand Disease is the most common inherited hemorrhage disturbance in humans, leading to insufficiency in von Willebrand Factor, which is a complex multimeric protein with two functions: it forms a bridge between the platelets and injured vascular areas and it attaches factor VIII and stabilizes it. We present a 13-year-old son with a typical clinical manifestation of KTS, including "port-wine stains" as capillary malformation, venous malformation, and hypertrophy of the left lower extremity, who also suffers from von Willebrand Disease type 3. He has been suffering from these two rare conditions since birth. The occurrence of KTS with von Willebrand Factor deficiency in a patient has so far not been reported, which may propose a mutation in the putative common regulatory gene that caused this uncommon phenotype.
克-特综合征(KTS)是一种罕见的遗传性综合征,其特征为静脉静脉曲张和毛细血管异常。血管性血友病是人类最常见的遗传性出血障碍,会导致血管性血友病因子不足,该因子是一种具有两种功能的复杂多聚体蛋白:它在血小板和受损血管区域之间形成桥梁,并附着凝血因子VIII并使其稳定。我们报告一名13岁男性,具有典型的KTS临床表现,包括作为毛细血管畸形的“葡萄酒色斑”、静脉畸形和左下肢肥大,他还患有3型血管性血友病。他自出生以来就患有这两种罕见疾病。迄今为止,尚未报道过患者同时患有KTS和血管性血友病因子缺乏症的情况,这可能提示在假定的共同调控基因中发生了突变,导致了这种罕见的表型。