Characteristics and outcome of primary resistant disease in paediatric acute myeloid leukaemia

Lene Karlsson (Coresponding Author), Daniel Cheuk, Barbara De Moerloose, Henrik Hasle, Kirsi Jahnukainen, Kristian Løvvik Juul-Dam, Gertjan Kaspers, Zanna Kovalova, Birgitte Lausen, Ulrika Norén Nyström, Josefine Palle, Cornelis Jan Pronk, Kadri Saks, Anne Tierens, Bernward Zeller, Jonas Abrahamsson

Research output: Contribution to journalArticlepeer-review

2 Citations (Scopus)
7 Downloads (Pure)


A significant proportion of events in paediatric acute myeloid leukaemia (AML) are caused by resistant disease (RD). We investigated clinical and biological characteristics in 66 patients with RD from 1013 children with AML registered and treated according to the NOPHO-AML 93, NOPHO-AML 2004, DB AML-01 and NOPHO-DBH AML 2012 protocols. Risk factors for RD were age10 years or older and a white-blood-cell count (WBC) of 100 × 109/L or more at diagnosis. The five-year overall survival (OS) was 38% (95% confidence interval [CI]: 28%–52%). Of the 63 children that received salvage therapy with chemotherapy, 59% (N = 37) achieved complete remission (CR) with OS 57% (95% CI: 42%–75%) compared to 12% (95% CI: 4%–35%) for children that did not achieve CR. Giving more than two salvage chemotherapy courses did not increase CR rates. OS for all 43 patients receiving allogeneic haematopoietic stem cell transplantation (HSCT) was 49% (95% CI: 36%–66%). Those achieving CR and proceeding to HSCT had an OS of 56% (95% CI: 41%–77%, N = 30). This study showed that almost 40% of children with primary resistant AML can be cured with salvage therapy followed by HSCT. Children that did not achieve CR after two salvage courses with chemotherapy did not benefit from additional chemotherapy.

Original languageEnglish
Pages (from-to)757-765
Number of pages9
JournalBritish Journal of Haematology
Issue number4
Publication statusPublished - May 2023
Externally publishedYes


  • acute myeloid leukaemia
  • paediatric
  • resistant disease
  • survival

Field of Science*

  • 3.2 Clinical medicine

Publication Type*

  • 1.1. Scientific article indexed in Web of Science and/or Scopus database


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