Long-term outcome of acquired aplastic anaemia in children

Comparison between immunosuppressive therapy and bone marrow transplantation

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

Research output: Contribution to journalArticle

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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.

Original languageEnglish
Pages (from-to)321-328
Number of pages8
JournalBritish Journal of Haematology
Volume111
Issue number1
DOIs
Publication statusPublished - Nov 23 2000
Externally publishedYes

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Aplastic Anemia
Immunosuppressive Agents
Bone Marrow Transplantation
Group Psychotherapy
Myelodysplastic Syndromes
Tissue Donors
Survival
Therapeutics
Salvage Therapy
Unrelated Donors
Antilymphocyte Serum
Cyclosporine
Survival Rate
Pediatrics
Recurrence
Incidence

All Science Journal Classification (ASJC) codes

  • Hematology

Cite this

Long-term outcome of acquired aplastic anaemia in children : Comparison between immunosuppressive therapy and bone marrow transplantation. / Kojima, Seiji; Horibe, Keizo; Inaba, Jun; Yoshimi, Ayami; Takahashi, Yoshiyuki; Kudo, Kazuko; Kato, Koji; Matsuyama, Takaharu.

In: British Journal of Haematology, Vol. 111, No. 1, 23.11.2000, p. 321-328.

Research output: Contribution to journalArticle

Kojima, Seiji ; Horibe, Keizo ; Inaba, Jun ; Yoshimi, Ayami ; Takahashi, Yoshiyuki ; Kudo, Kazuko ; Kato, Koji ; Matsuyama, Takaharu. / Long-term outcome of acquired aplastic anaemia in children : Comparison between immunosuppressive therapy and bone marrow transplantation. In: British Journal of Haematology. 2000 ; Vol. 111, No. 1. pp. 321-328.
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