TY - JOUR
T1 - Characteristics and outcome of primary resistant disease in paediatric acute myeloid leukaemia
AU - Karlsson, Lene
AU - Cheuk, Daniel
AU - de Moerloose, Barbara
AU - Hasle, Henrik
AU - Jahnukainen, Kirsi
AU - Juul-Dam, Kristian L. vvik
AU - Kaspers, Gertjan
AU - Kovalova, Zanna
AU - Lausen, Birgitte
AU - Nyström, Ulrika Norén
AU - Palle, Josefine
AU - Pronk, Cornelis Jan
AU - Saks, Kadri
AU - Tierens, Anne
AU - Zeller, Bernward
AU - Abrahamsson, Jonas
N1 - Funding Information: The study was financed by grants from the Swedish Childrens´ Cancer foundation and the Swedish state under the agreement between the Swedish government and the county councils, the ALF‐agreement (ALFGBG‐966256). Publisher Copyright: © 2023 The Authors. British Journal of Haematology published by British Society for Haematology and John Wiley & Sons Ltd.
PY - 2023/5
Y1 - 2023/5
N2 - 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.
AB - 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.
KW - acute myeloid leukaemia
KW - paediatric
KW - resistant disease
KW - survival
UR - http://www.scopus.com/inward/record.url?scp=85147567145&partnerID=8YFLogxK
U2 - https://doi.org/10.1111/bjh.18685
DO - https://doi.org/10.1111/bjh.18685
M3 - Article
C2 - 36762836
SN - 0007-1048
VL - 201
SP - 757
EP - 765
JO - British journal of haematology
JF - British journal of haematology
IS - 4
ER -