title: Case Reports of Severe Congenital Neutropenia Treated With Unrelated Cord Blood Transplantation With Reduced-intensity Conditioning.
abstract: Allogeneic stem cell transplantation is a curative treatment for severe congenital neutropenia (SCN). However, a standard conditioning regimen and donor source have not been established. We report 3 consecutive cases of SCN who were successfully treated by cord blood transplantation (CBT) with reduced-intensity conditioning consisting of fludarabine, melphalan, and low-dose total body irradiation. All cases achieved complete donor chimerism without severe infectious complications and have maintained normal neutrophil counts for between 3 and 9 years after CBT. These results suggest that CBT with reduced-intensity conditioning can be an alternative therapy for SCN when human leukocyte antigen-matched bone marrow donor is unavailable.
authors.affiliation: *Department of Pediatrics, Kyoto City Hospital †Department of Pediatrics, Graduate School of Medical Science, Kyoto Prefectural University of Medicine ‡Uji-Tokushukai Medical Center, Uji, Kyoto, Japan.
authors.forename: Shinya
authors.initials: S
authors.lastname: Osone
authors.forename: Toshihiko
authors.initials: T
authors.lastname: Imamura
authors.forename: Yoko
authors.initials: Y
authors.lastname: Fukushima-Nakase
authors.forename: Ayaka
authors.initials: A
authors.lastname: Kitamura-Masaki
authors.forename: Sotaro
authors.initials: S
authors.lastname: Kanai
authors.forename: Tomohiko
authors.initials: T
authors.lastname: Imai
authors.forename: Shinsaku
authors.initials: S
authors.lastname: Imashuku
authors.forename: Hiroshi
authors.initials: H
authors.lastname: Kuroda
mesh_terms: D000080984:Congenital Bone Marrow Failure Syndromes; D036101:Cord Blood Stem Cell Transplantation / Q000379:methods*; D005260:Female; D006801:Humans; D008297:Male; D009503:Neutropenia / Q000151:congenital* / Q000601:surgery; D019172:Transplantation Conditioning / Q000379:methods*