Wiskott-Aldrich syndrome related diseases / 中华实用儿科临床杂志

ABSTRACT

The Wiskott-Aldrich syndrome (WAS) related disorders,which include WAS,X-linked thrombocytopenia (XLT),and X-linked congenital neutropenia (XLN),are caused by WAS gene mutation and inherited in an X-linked recessive manner.The main clinical features of these disorders are eczema,thrombocytopenia and immunodeficiency.They can lead to severe complications,so early diagnosis and early treatment are necessary.In severe cases,allogeneic hematopoietic stem cell transplantation is required.