title: Outcome of unrelated umbilical cord blood transplantation in 88 patients with primary immunodeficiency in Japan.
abstract: We report the results of umbilical cord blood transplantation (UCBT) performed in 88 patients with primary immunodeficiency (PID) between 1998 and 2008 in Japan; severe combined immunodeficiency (SCID, n = 40), Wiskott-Aldrich syndrome (WAS, n = 23), chronic granulomatous disease (n = 7), severe congenital neutropaenia (SCN, n = 5) and other immunodeficiencies (n = 13). Five-year overall survival (5-year OS) for all patients was 69% [95% confidence interval (CI), 57-78%], and was 71% and 82% for SCID and WAS, respectively. The main cause of death before day 100 was infection (17/19), while that after day 100 was graft-versus-host disease (GVHD) (5/7). Using multivariate analyses, pre-transplant infection, no conditioning, ≥ 2 human leucocyte antigen (HLA) mismatches or diagnosis other than SCID, SCN or WAS were all associated with poor prognosis. Reduced-intensity conditioning was associated with decreased overall mortality compared with myeloablative therapy. The cumulative incidence of grade 2-4 acute GVHD at day 100 was 28% (95% CI, 19-38%), and that of chronic GVHD at day 180 was 13% (95% CI, 7-23%). We conclude that UCBT should be considered for PID patients without an HLA-matched sibling. The control of pre-transplant infection and selection of HLA-matched donors will lead to a better outcome.
authors.affiliation: Department of Paediatrics and Developmental Biology, Tokyo Medical and Dental University Graduate School of Medical and Dental Sciences, Tokyo, Japan. tmorio.ped@tmd.ac.jp
authors.forename: Tomohiro
authors.initials: T
authors.lastname: Morio
authors.forename: Yoshiko
authors.initials: Y
authors.lastname: Atsuta
authors.forename: Daisuke
authors.initials: D
authors.lastname: Tomizawa
authors.forename: Tokiko
authors.initials: T
authors.lastname: Nagamura-Inoue
authors.forename: Koji
authors.initials: K
authors.lastname: Kato
authors.forename: Tadashi
authors.initials: T
authors.lastname: Ariga
authors.forename: Keisei
authors.initials: K
authors.lastname: Kawa
authors.forename: Kazutoshi
authors.initials: K
authors.lastname: Koike
authors.forename: Hisamichi
authors.initials: H
authors.lastname: Tauchi
authors.forename: Michiko
authors.initials: M
authors.lastname: Kajiwara
authors.forename: Toshiro
authors.initials: T
authors.lastname: Hara
authors.forename: Shunichi
authors.initials: S
authors.lastname: Kato
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D036101:Cord Blood Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods*; D004812:Epidemiologic Methods; D006085:Graft Survival; D006086:Graft vs Host Disease / Q000209:etiology; D006648:Histocompatibility; D006801:Humans; D007153:Immunologic Deficiency Syndromes / Q000628:therapy*; D007223:Infant; D007231:Infant, Newborn; D009894:Opportunistic Infections / Q000209:etiology; D016511:Severe Combined Immunodeficiency / Q000628:therapy; D019172:Transplantation Conditioning / Q000379:methods; D016896:Treatment Outcome; D014923:Wiskott-Aldrich Syndrome / Q000628:therapy; D055815:Young Adult