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International cooperative study identifies treatment strategy in childhood ambiguous lineage leukemia

Lookup NU author(s): Professor Julie Irving, Professor Anthony MoormanORCiD



This is the authors' accepted manuscript of an article that has been published in its final definitive form by American Society of Hematology, 2018.

For re-use rights please refer to the publisher's terms and conditions.


© American Society of Hematology. All rights reserved.Despite attempts to improve the definitions of ambiguous lineage leukemia (ALAL) during the last 2 decades, general therapy recommendations are missing. Herein, we report a large cohort of children with ALAL and propose a treatment strategy. A retrospective multinational study (International Berlin-Frankfurt-Münster Study of Leukemias of Ambiguous Lineage [iBFM-AMBI2012]) of 233 cases of pediatric ALAL patients is presented. Survival statistics were used to compare the prognosis of subsets and types of treatment. Five-year event-free survival (EFS) of patients with acute lymphoblastic leukemia (ALL)-type primary therapy (80% 6 4%) was superior to that of children who received acute myeloid leukemia (AML)-type or combined-type treatment (36% 6 7.2% and 50% 6 12%, respectively). When ALL- or AML-specific gene fusions were excluded, 5-year EFS of CD191 leukemia was 83% 6 5.3% on ALL-type primary treatment compared with 0% 6 0% and 28% 6 14% on AML-type and combined-type primary treatment, respectively. Superiority , of ALL-type treatment was documented in single-population mixed phenotype ALAL (using World Health Organization . and/or European Group for Immunophenotyping of Leukemia definitions) and bilineal ALAL. Treatment with ALL-type protocols is recommended for the majority of pediatric patients with ALAL, including cases with CD191 ALAL. AML-type treatment is preferred in a minority of ALAL cases with CD192 and no other lymphoid features. No overall benefit of transplantation was documented, and it could be introduced in some patients with a poor response to treatment. As no clear indicator was found for a change in treatment type, this is to be considered only in cases with ‡5% blasts after remission induction. The results provide a basis for a prospective trial. (Blood. 2018;132(3):264-276)

Publication metadata

Author(s): Hrusak O, De Haas V, Stancikova J, Vakrmanova B, Janotova I, Mejstrikova E, Capek V, Trka J, Zaliova M, Luks A, Bleckmann K, Moricke A, Irving J, Konatkowska B, Alexander TB, Inaba H, Schmiegelow K, Stokley S, Zemanova Z, Moorman AV, Rossi JG, Felice MS, Dalla-Pozza L, Morales J, Dworzak M, Buldini B, Basso G, Campbell M, Cabrera ME, Marinov N, Elitzur S, Izraeli S, Luria D, Feuerstein T, Kolenova A, Svec P, Kreminska O, Rabin KR, Polychronopoulou S, Da Costa E, Marquart HV, Kattamis A, Ratei R, Reinhardt D, Choi JK, Schrappe M, Stary J

Publication type: Article

Publication status: Published

Journal: Blood

Year: 2018

Volume: 132

Issue: 3

Pages: 264-276

Print publication date: 19/07/2018

Online publication date: 19/07/2018

Acceptance date: 09/04/2018

Date deposited: 28/03/2019

ISSN (print): 0006-4971

ISSN (electronic): 1528-0020

Publisher: American Society of Hematology


DOI: 10.1182/blood-2017-12-821363


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