Unrelated donor marrow transplantation in children with severe aplastic anaemia using cyclophosphamide, anti-thymocyte globulin and total body irradiation

Seiji Kojima, Jun Inaba, Ayami Yoshimi, Yoshiyuki Takahashi, Nobuhiro Watanabe, Kazuko Kudo, Keizo Horibe, Naoko Maeda, Koji Kato, Takaharu Matsuyama

Research output: Contribution to journalArticle

39 Citations (Scopus)

Abstract

We report a favourable outcome in 15 patients with severe aplastic anaemia (SAA) who were <20 years of age and who underwent bone marrow transplantation (BMT) from a human leucocyte antigen (HLA)-matched unrelated donor. All patients were non-responders to intensive immunosuppressive therapy (IST) and were multiply transfused. The conditioning regimen consisted of cyclophosphamide (60 mg/kg/d, on d -4 and -3), anti-thymocyte globulin (2·5 mg/kg/d, on d -5 to -2) and total body irradiation (2·5 Gy×2/d, on d -2 and -1). Patients received cyclosporine and methotrexate for prophylaxis of graft-versus-host disease (GVHD), except for the last four who received tacrolimus instead of cyclosporine. Donor/recipient pairs were identical for HLA class I and II antigens by serological typing, but four pairs were found to have a mismatch at the HLA-A, -B or -DRB1 locus by high-resolution typing. All patients achieved rapid engraftment and are alive at 2-86 months after transplantation (median follow-up, 51 months). Moderate to severe acute GVHD occurred in 5 out of 15 patients (33%); only one patient developed extensive chronic GVHD. Considering our encouraging results, unrelated donor transplantation for SAA is recommended as a salvage therapy in non-responders to IST.

Original languageEnglish
Pages (from-to)706-711
Number of pages6
JournalBritish Journal of Haematology
Volume114
Issue number3
DOIs
Publication statusPublished - Oct 22 2001
Externally publishedYes

All Science Journal Classification (ASJC) codes

  • Hematology

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