Outcome of poor response paediatric AML using early SCT

Neval E. Wareham, Carsten Heilmann, Jonas Abrahamsson, Erik Forestier, Britt Gustafsson, Shau-Yin Ha, Jesper Heldrup, Kirsi Jahnukainen, Olafur G. Jonsson, Birgitte Lausen, Josefine Palle, Bernward Zeller, Henrik Hasle

Research output: Contribution to journalArticlepeer-review


Background Children with poor response acute myeloid leukaemia (AML) generally have a very poor outcome. Allogeneic stem cell transplantation (SCT) is often recommended for these children but the benefit is unclear. The aim of this study was to investigate survival for poor response AML patients treated with SCT. Material and Methods Treatment was given according to the NOPHO-AML 2004 protocol. All patients received AIET (Cytarabine, Idarubicin, Etoposide, Thioguanine) and AM (Cytarabine, Mitoxantrone) as induction. We included poor response defined as > 15% blasts on day 15 after AIET (n=17) or > 5% blasts after AM (n=14, refractory disease). Poor response patients received intensively timed induction and proceeded to SCT when a donor was available. Results Thirty-one of 267 evaluable patients (12%) had a poor response. SCT was performed in 25; using matched unrelated donors in 13, matched sibling donors in 6, cord blood donor in 4, and haploidentical donor in two. The median follow-up for the 31 poor responding patients was 2.6 years (range 0.4 - 8.1 years) and 3-year probability of survival 70% (95% CI 59-77%). Conclusions The poor responders in the NOPHO-AML 2004 protocol had a favourable prognosis treated with time-intensive induction followed by SCT.
Original languageEnglish
Pages (from-to)187-194
JournalEuropean Journal of Haematology
Issue number3
Publication statusPublished - 2013

Subject classification (UKÄ)

  • Hematology

Free keywords

  • acute myeloid leukaemia
  • early stem cell transplantation
  • survival
  • poor
  • response


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