Huang M.-T.Chou C.-C.SHIANN-TANG JOUKAI-HSIN LINHsieh K.-H.2021-01-052021-01-0519960125-877Xhttps://www.scopus.com/inward/record.uri?eid=2-s2.0-0030418645&partnerID=40&md5=e64b28b34f4a47e3e246ceb2755581b2https://scholars.lib.ntu.edu.tw/handle/123456789/537786We 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.[SDGs]SDG3busulfan; cyclophosphamide; immunosuppressive agent; article; bone marrow transplantation; case report; child; chinese; eczema; human; intravenous drug administration; male; oral drug administration; otitis media; preschool child; thrombocytopenia; wiskott aldrich syndrome; Asian Continental Ancestry Group; Bone Marrow Transplantation; Busulfan; Cyclophosphamide; Humans; Infant; Male; Wiskott-Aldrich Syndromejournal article89808012-s2.0-0030418645