The Progress in Research in Hereditary Thrombotic Thrombocytopenic Purpura in Children
10.12376/j.issn.2097-0501.2022.04.007
- VernacularTitle:儿童遗传性血栓性血小板减少性紫癜的研究进展
- Author:
Lingling FU
1
;
Runhui WU
1
Author Information
1. Department 2 of Hematology, National Center for Children's Health, Beijing Children's Hospital, Capital Medical University, Beijing 100045, China.
- Publication Type:Journal Article
- Keywords:
children;
hereditary thrombotic thrombocytopenic purpura;
ADAMTS13;
treatment
- From:
JOURNAL OF RARE DISEASES
2022;1(4):400-406
- CountryChina
- Language:Chinese
-
Abstract:
Hereditary thrombotic thrombocytopenic purpura (hTTP) in children is a rare but severe and fatal thrombotic microangiopathy. The etiology of the disease is the persistent severe deficiency of the enzyme ADAMTS13 gene mutation, resulting in microangiopathic hemolytic anemia, thrombocytopenia, neuropsychiatric symptoms, fever, and renal involvement. Different from adults, children with hTTP present earlier onset of the disease and are more likely to develop long-term complications in brain and kidney, so that the need for preventive replacement therapy is more urgent. This article reviews the research progress of hTTP in children.
