title: Successful allogeneic stem cell transplant for leukocyte adhesion deficiency using an adjusted busulfan-containing regimen.
abstract: LAD is a rare and fatal congenital disorder in which there is a defect of the leukocyte adhesion molecule (CD18) on neutrophils. Severe LAD results in frequent and potentially fatal infections. Although allogeneic HSCT is the only curative treatment for severe LAD, strategies for HSCT remain to be established since a high engraftment failure rate and severe persistent infection are still seen. We report a four-month-old boy with LAD who was successfully treated with allogeneic HSCT from an HLA-complete matched sibling following a conditioning regimen of pharmacokinetically adjusted individual busulfan doses.
authors.affiliation: Department of Pediatrics, Osaka University Graduate School of Medicine, Osaka, Japan.
authors.forename: Akira
authors.initials: A
authors.lastname: Yoshimoto
authors.forename: Yoshiko
authors.initials: Y
authors.lastname: Hashii
authors.forename: Hiroko
authors.initials: H
authors.lastname: Kashiwagi
authors.forename: Makoto
authors.initials: M
authors.lastname: Koizumi
authors.forename: Sadao
authors.initials: S
authors.lastname: Tokimasa
authors.forename: Hiroyuki
authors.initials: H
authors.lastname: Fujisaki
authors.forename: Hideaki
authors.initials: H
authors.lastname: Ohta
authors.forename: Naomi
authors.initials: N
authors.lastname: Hanai
authors.forename: Keiichi
authors.initials: K
authors.lastname: Ozono
authors.forename: Junichi
authors.initials: J
authors.lastname: Hara
mesh_terms: D002066:Busulfan / Q000627:therapeutic use*; D005500:Follow-Up Studies; D006084:Graft Rejection / Q000517:prevention & control; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D007223:Infant; D018370:Leukocyte-Adhesion Deficiency Syndrome / Q000601:surgery*; D008297:Male; D019172:Transplantation Conditioning / Q000379:methods*; D014184:Transplantation, Homologous
chemical_list: D007166:Immunosuppressive Agents; D002066:Busulfan