Treatment with marrow transplantation or immunosuppression of childhood acquired severe aplastic anemia: A report from the EBMT SAA Working Party

A. Locasciulli, L. Van 't Veer, A. Bacigalupo, J. Hows, M. T. Van Lint, E. Gluckman, C. Nissen, S. McCann, J. Vossen, A. Schrezenmeier, W. Hinterberger, A. Marin

Research output: Contribution to journalArticlepeer-review

Abstract

A total of 304 children under the age of 15 years with acquired severe aplastic anemia (SAA) received immunosuppressive therapy (IS) (n = 133) or a matched bone marrow transplant (BMT) (n = 171). The projected 10-year survival is 48% and 63% respectively (p = 0.002). Results following BMT have improved considerably over the years from 49% in 1970-80, to 70% in 1981-83 (p = 0.002) and to 81% between 1984-88 (p = 0.08). Other favorable prognostic factors are the use of cyclosporin A (p = 0.004), no previous therapy (p = 0.006) and early BMT (p = 0.009). In multivariate analysis only the year of treatment proved significant (p = 0.02). In contrast, results of IS are greatly dependent on the severity of pre-treatment neutropenia with survival of 56% versus 37% for neutrophils more or less than 0.2 x 109/l (p = 0.003). Poor survival was associated in univariate analysis with female sex (43%), post-hepatitis SAA (37%), children not receiving androgens (38%) and patients younger than 5 years (35%), especially if associated with a low neutrophil count (11%). In multivariate analysis only the degree of neutropenia proved signifcant (p = 0.005). These results suggest that IS is a satisfactory alternative therapy for children with moderately SAA in the absence of an HLA-identical sibling, although BMT remains the treatment of choice. In children under 5 years with very SAA, results with IS are so poor that a search for an unrelated matched donor is justified as early as possible.

Original languageEnglish
Pages (from-to)211-217
Number of pages7
JournalBone Marrow Transplantation
Volume6
Issue number3
Publication statusPublished - 1990

ASJC Scopus subject areas

  • Hematology
  • Transplantation

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