Outcome of unrelated umbilical cord blood transplantation in 88 patients with primary immunodeficiency in Japan

Tomohiro Morio, Yoshiko Atsuta, Daisuke Tomizawa, Tokiko Nagamura-Inoue, Koji Kato, Tadashi Ariga, Keisei Kawa, Kazutoshi Koike, Hisamichi Tauchi, Michiko Kajiwara, Toshiro Hara, Shunichi Kato

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44 Citations (Scopus)


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.

Original languageEnglish
Pages (from-to)363-372
Number of pages10
JournalBritish Journal of Haematology
Issue number3
Publication statusPublished - Aug 2011
Externally publishedYes

All Science Journal Classification (ASJC) codes

  • Hematology


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