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Long-term outcome of acquired aplastic anaemia in children: comparison between immunosuppressive therapy and bone marrow transplantation.
Br J Haematol. 2000 Oct; 111(1):321-8.BJ

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+Show Affiliations

Department of Developmental Paediatrics, Nagoya University School of Medicine, Japan. kojimas@med.nagoya-u.ac.jpNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Comparative Study
Journal Article

Language

eng

PubMed ID

11091219

Citation

Kojima, S, et al. "Long-term Outcome of Acquired Aplastic Anaemia in Children: Comparison Between Immunosuppressive Therapy and Bone Marrow Transplantation." British Journal of Haematology, vol. 111, no. 1, 2000, pp. 321-8.
Kojima S, Horibe K, Inaba J, et al. Long-term outcome of acquired aplastic anaemia in children: comparison between immunosuppressive therapy and bone marrow transplantation. Br J Haematol. 2000;111(1):321-8.
Kojima, S., Horibe, K., Inaba, J., Yoshimi, A., Takahashi, Y., Kudo, K., Kato, K., & Matsuyama, T. (2000). Long-term outcome of acquired aplastic anaemia in children: comparison between immunosuppressive therapy and bone marrow transplantation. British Journal of Haematology, 111(1), 321-8.
Kojima S, et al. Long-term Outcome of Acquired Aplastic Anaemia in Children: Comparison Between Immunosuppressive Therapy and Bone Marrow Transplantation. Br J Haematol. 2000;111(1):321-8. PubMed PMID: 11091219.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Long-term outcome of acquired aplastic anaemia in children: comparison between immunosuppressive therapy and bone marrow transplantation. AU - Kojima,S, AU - Horibe,K, AU - Inaba,J, AU - Yoshimi,A, AU - Takahashi,Y, AU - Kudo,K, AU - Kato,K, AU - Matsuyama,T, PY - 2000/11/25/pubmed PY - 2001/2/28/medline PY - 2000/11/25/entrez SP - 321 EP - 8 JF - British journal of haematology JO - Br. J. Haematol. VL - 111 IS - 1 N2 - 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. SN - 0007-1048 UR - https://www.unboundmedicine.com/medline/citation/11091219/Long_term_outcome_of_acquired_aplastic_anaemia_in_children:_comparison_between_immunosuppressive_therapy_and_bone_marrow_transplantation_ L2 - https://onlinelibrary.wiley.com/resolve/openurl?genre=article&sid=nlm:pubmed&issn=0007-1048&date=2000&volume=111&issue=1&spage=321 DB - PRIME DP - Unbound Medicine ER -