Cyclosporin A response and dependence in children with acquired aplastic anaemia: A multicentre retrospective study with long-term observation follow-up

Paola Saracco, Paola Quarello, Anna Paola Iori, Marco Zecca, Daniela Longoni, Johanna Svahn, Stefania Varotto, Gian Carlo Del Vecchio, Carlo Dufour, Ugo Ramenghi, Andrea Bacigalupo, Anna Locasciulli

Research output: Contribution to journalArticlepeer-review

Abstract

Immunosuppressive therapy (IST) with antithymocyte globulin and cyclosporin A (CyA) is the standard treatment for children with acquired aplastic anaemia (AAA) lacking a matched donor. Survival rates of more than 80% at 5 years are achieved, but the response is drug-dependent in 15-25% of cases. This study, of 42 consecutive children with AAA treated with IST, assessed the incidence of CyA-dependence, CyA and granulocyte colony-stimulating factor (G-CSF) tapering schedules and the impact of drug accumulation on progression to myelodysplasia/acute myeloid leukaemia (MDS/AML). Overall survival was 83% at 10 years. CyA-dependence without a predictive marker was observed in 18% of responders. Probability of discontinuing CyA was 60.5% at 10 years; a slow CyA tapering schedule was performed in 84% of patients; the cumulative incidence of relapse was 16% at 10 years. Relapse risk was significantly associated with rapid CyA discontinuation: 60% compared to 7.6% in the slow tapering group (P = 0.001). Cumulative incidence of MDS/AML was 8% at 10 years, with a significant correlation with both G-CSF cumulative dose and second IST. This long-term follow-up of children with AAA shows that IST with a slow CyA tapering course is an effective treatment with a low-relapse rate in these cases.

Original languageEnglish
Pages (from-to)197-205
Number of pages9
JournalBritish Journal of Haematology
Volume140
Issue number2
DOIs
Publication statusPublished - Jan 2008

Keywords

  • Aplastic anaemia
  • Children
  • Therapy

ASJC Scopus subject areas

  • Hematology

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