title: Long-term outcome of acquired aplastic anaemia in children: comparison between immunosuppressive therapy and bone marrow transplantation.
abstract: A total of 100 children under the age of 17 years with acquired aplastic anaemia (AA) were initially treated with immunosuppressive therapy (IST) (n = 63) or bone marrow transplantation (BMT) (n = 37) from an HLA-matched family donor. The projected 10-year survival rates were 55 +/- 8% and 97 +/- 3% respectively (P = 0.004). Because the IST group included 11 non-responders who were salvaged by BMT from an HLA-matched unrelated donor, we compared failure-free survival (FFS) between the groups. The probability of FFS at 10 years was 97 +/- 3% for the BMT group, compared with 40 +/- 8% for the IST group (P = 0.0001). Seven patients evolved to myelodysplastic syndrome (MDS) with monosomy 7 and the estimated cumulative incidence of MDS 10 years after diagnosis was 20 +/- 7% in the IST group. We compared the outcome of children treated with IST during the two consecutive periods of 1983-91 (group A, n = 40) and 1991-8 (group B, n = 23) to assess the impact of combined therapy with antithymocyte globulin and cyclosporin. The probability of FFS at 7 years follow-up was the same in the two groups (50 +/- 8% vs. 40 +/- 15%, P = 0.40). We recommend BMT as first-line therapy in paediatric severe AA patients with an HLA-matched family donor. Alternative donor BMT is recommended as salvage therapy in patients who relapse or do not respond to initial IST.
authors.affiliation: Department of Developmental Paediatrics, Nagoya University School of Medicine, Japan. kojimas@med.nagoya-u.ac.jp
authors.forename: S
authors.initials: S
authors.lastname: Kojima
authors.forename: K
authors.initials: K
authors.lastname: Horibe
authors.forename: J
authors.initials: J
authors.lastname: Inaba
authors.forename: A
authors.initials: A
authors.lastname: Yoshimi
authors.forename: Y
authors.initials: Y
authors.lastname: Takahashi
authors.forename: K
authors.initials: K
authors.lastname: Kudo
authors.forename: K
authors.initials: K
authors.lastname: Kato
authors.forename: T
authors.initials: T
authors.lastname: Matsuyama
mesh_terms: D000293:Adolescent; D000741:Anemia, Aplastic / Q000401:mortality / Q000601:surgery / Q000628:therapy*; D000961:Antilymphocyte Serum / Q000627:therapeutic use*; D016026:Bone Marrow Transplantation; D002452:Cell Count; D002648:Child; D002675:Child, Preschool; D016572:Cyclosporine / Q000627:therapeutic use*; D018572:Disease-Free Survival; D005260:Female; D005500:Follow-Up Studies; D006086:Graft vs Host Disease / Q000150:complications; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D007223:Infant; D008297:Male; D012189:Retrospective Studies; D018709:Statistics, Nonparametric; D013601:T-Lymphocytes / Q000276:immunology*
chemical_list: D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D016572:Cyclosporine