title: Successful bone marrow transplantation in a Chinese boy with Wiskott-Aldrich syndrome.
abstract: We describe the successful use of HLA-compatible sibling bone marrow transplantation (BMT) in a 17-month-old Chinese boy in whom Wiskott-Aldrich syndrome (WAS) was diagnosed on the basis of eczema, thrombocytopenia, recurrent otitis media and abnormal immunological tests. The conditioning chemotherapy included 2 days' oral busulfan, 40 mg/m2/6 hours, and 2 days' intravenous cyclophosphamide, 60 mg/kg/day (BU2CY2). Complete hematological chimerism was achieved 3 weeks after transplantation. Eight months after his BMT the eczema has resolved, platelet count is normal, and he no longer has frequent infections. BU2CY2 as a preconditioning regimen gave complete lymphohematopoietic engraftment in this WAS patient with no evidence of graft-versus-host disease. The excellent clinical response of this patient and the inevitable fatal outcome of WAS support the opinion that where a histocompatible donor is available, BMT at the earliest opportunity is the best option. We believe this is the first case of successful BMT in a Chinese patient with WAS.
authors.affiliation: Department of Pediatrics, College of Medicine, National Taiwan University Hospital, Taipei.
authors.forename: M T
authors.initials: MT
authors.lastname: Huang
authors.forename: C C
authors.initials: CC
authors.lastname: Chou
authors.forename: S T
authors.initials: ST
authors.lastname: Jou
authors.forename: K H
authors.initials: KH
authors.lastname: Lin
authors.forename: K H
authors.initials: KH
authors.lastname: Hsieh
mesh_terms: D044466:Asian People; D016026:Bone Marrow Transplantation; D002066:Busulfan / Q000627:therapeutic use; D003520:Cyclophosphamide / Q000627:therapeutic use; D006801:Humans; D007223:Infant; D008297:Male; D014923:Wiskott-Aldrich Syndrome / Q000628:therapy*
chemical_list: D003520:Cyclophosphamide; D002066:Busulfan