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发现一种罕见关联:遗传性血栓性血小板减少性紫癜与巨大胎盘血池

Discovering a Rare Connection: Hereditary Thrombotic Thrombocytopenic Purpura and Large Placental Lakes.

作者信息

Dokuzboy Refika S, Çetin Dilek, Tuğcu Ali U, Oğuz Şerife S

机构信息

Division of Neonatology, Department of Pediatrics, Ministry of Health, Ankara Bilkent City Hospital, Ankara, Türkiye.

出版信息

J Pediatr Hematol Oncol. 2025 Jul 1;47(5):246-249. doi: 10.1097/MPH.0000000000003053. Epub 2025 May 24.

DOI:10.1097/MPH.0000000000003053
PMID:40424567
Abstract

Hereditary thrombotic thrombocytopenic purpura (hTTP) is a rare genetic disorder caused by mutations in the ADAMTS13 (a disintegrin-like and metalloprotease with thrombospondin type 1 motif, member 13) gene, leading to deficient or absent ADAMTS13 activity. Without ADAMTS13, ultralarge von Willebrand factor (ULVWF) molecules are not properly cleaved, resulting in the formation of platelet-rich thrombi, platelet consumption, organ ischemia, and microangiopathic hemolytic anemia. We report a female newborn who presented with respiratory distress, jaundice, anemia, and thrombocytopenia. Prenatal ultrasonography revealed a large placental lake. She was diagnosed with hTTP and successfully treated with fresh frozen plasma (FFP) transfusion. Genetic analysis revealed a pathogenic homozygous mutation in the ADAMTS13 gene. To our knowledge, this report is the first to document large placental lakes in a newborn with hTTP, suggesting a potential link between fetal ADAMTS13 deficiency and abnormal placentation.

摘要

遗传性血栓性血小板减少性紫癜(hTTP)是一种罕见的遗传性疾病,由ADAMTS13(含Ⅰ型血小板反应蛋白基序的解聚素样金属蛋白酶13)基因突变引起,导致ADAMTS13活性缺乏或缺失。缺乏ADAMTS13时,超大血管性血友病因子(ULVWF)分子无法正常裂解,导致富含血小板的血栓形成、血小板消耗、器官缺血和微血管病性溶血性贫血。我们报告了一名患有呼吸窘迫、黄疸、贫血和血小板减少症的女新生儿。产前超声检查发现一个大的胎盘血池。她被诊断为hTTP,并通过输注新鲜冰冻血浆(FFP)成功治疗。基因分析显示ADAMTS13基因存在致病性纯合突变。据我们所知,本报告首次记录了患有hTTP的新生儿出现大胎盘血池,提示胎儿ADAMTS13缺乏与胎盘形成异常之间可能存在联系。

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