Refika S Dokuzboy, Dilek Çetin, Ali U Tuğcu, Şerife S Oğuz
{"title":"发现罕见的联系:遗传性血栓性血小板减少性紫癜和大胎盘湖。","authors":"Refika S Dokuzboy, Dilek Çetin, Ali U Tuğcu, Şerife S Oğuz","doi":"10.1097/MPH.0000000000003053","DOIUrl":null,"url":null,"abstract":"<p><p>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.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"246-249"},"PeriodicalIF":0.8000,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Discovering a Rare Connection: Hereditary Thrombotic Thrombocytopenic Purpura and Large Placental Lakes.\",\"authors\":\"Refika S Dokuzboy, Dilek Çetin, Ali U Tuğcu, Şerife S Oğuz\",\"doi\":\"10.1097/MPH.0000000000003053\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>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.</p>\",\"PeriodicalId\":16693,\"journal\":{\"name\":\"Journal of Pediatric Hematology/Oncology\",\"volume\":\" \",\"pages\":\"246-249\"},\"PeriodicalIF\":0.8000,\"publicationDate\":\"2025-07-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Journal of Pediatric Hematology/Oncology\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1097/MPH.0000000000003053\",\"RegionNum\":4,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2025/5/24 0:00:00\",\"PubModel\":\"Epub\",\"JCR\":\"Q4\",\"JCRName\":\"HEMATOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of Pediatric Hematology/Oncology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1097/MPH.0000000000003053","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2025/5/24 0:00:00","PubModel":"Epub","JCR":"Q4","JCRName":"HEMATOLOGY","Score":null,"Total":0}
Discovering a Rare Connection: Hereditary Thrombotic Thrombocytopenic Purpura and Large Placental Lakes.
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.
期刊介绍:
Journal of Pediatric Hematology/Oncology (JPHO) reports on major advances in the diagnosis and treatment of cancer and blood diseases in children. The journal publishes original research, commentaries, historical insights, and clinical and laboratory observations.