Characteristics and outcome of patients with core-binding factor acute myeloid leukemia and FLT3-ITD: results from an international collaborative study

The aim of this study was to evaluate the prognostic impact of FLT3-ITD in core-binding factor acute myeloid leukemia (CBFAML) in an international, multicenter survey of 97 patients of whom 52% had t(8;21)(q22;q22) and 48% had inv(16)(p13q22)/t(16;16)(p13;q22). The median age of the patients was 53...

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Main Authors: Kayser, Sabine (Author) , Kramer, Michael (Author) , Martínez-Cuadrón, David (Author) , Grenet, Justin (Author) , Metzeler, Klaus H. (Author) , Sustkova, Zuzana (Author) , Luskin, Marlise R. (Author) , Brunner, Andrew M. (Author) , Elliott, Michelle A. (Author) , Gil, Cristina (Author) , Marini, Sandra Casal (Author) , Ráčil, Zdeněk (Author) , Cetkovsky, Petr (Author) , Novak, Jan (Author) , Perl, Alexander E. (Author) , Platzbecker, Uwe (Author) , Stölzel, Friedrich (Author) , Ho, Anthony Dick (Author) , Thiede, Christian (Author) , Stone, Richard M. (Author) , Röllig, Christoph (Author) , Montesinos, Pau (Author) , Schlenk, Richard Friedrich (Author) , Levis, Mark J. (Author)
Format: Article (Journal)
Language:English
Published: April 2022
In: Haematologica
Year: 2022, Volume: 107, Issue: 4, Pages: 836-843
ISSN:1592-8721
DOI:10.3324/haematol.2021.278645
Online Access:Verlag, lizenzpflichtig, Volltext: https://doi.org/10.3324/haematol.2021.278645
Verlag, lizenzpflichtig, Volltext: https://haematologica.org/article/view/haematol.2021.278645
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Author Notes:Sabine Kayser, Michael Kramer, David Martínez-Cuadrón, Justin Grenet, Klaus H. Metzeler, Zuzana Sustkova, Marlise R. Luskin, Andrew M. Brunner, Michelle A. Elliott, Cristina Gil, Sandra Casal Marini, Zdeněk Ráčil, Petr Cetkovsky, Jan Novak, Alexander E. Perl, Uwe Platzbecker, Friedrich Stölzel, Anthony D. Ho, Christian Thiede, Richard M. Stone, Christoph Röllig, Pau Montesinos, Richard F. Schlenk and Mark J. Levis

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245 1 0 |a Characteristics and outcome of patients with core-binding factor acute myeloid leukemia and FLT3-ITD  |b results from an international collaborative study  |c Sabine Kayser, Michael Kramer, David Martínez-Cuadrón, Justin Grenet, Klaus H. Metzeler, Zuzana Sustkova, Marlise R. Luskin, Andrew M. Brunner, Michelle A. Elliott, Cristina Gil, Sandra Casal Marini, Zdeněk Ráčil, Petr Cetkovsky, Jan Novak, Alexander E. Perl, Uwe Platzbecker, Friedrich Stölzel, Anthony D. Ho, Christian Thiede, Richard M. Stone, Christoph Röllig, Pau Montesinos, Richard F. Schlenk and Mark J. Levis 
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520 |a The aim of this study was to evaluate the prognostic impact of FLT3-ITD in core-binding factor acute myeloid leukemia (CBFAML) in an international, multicenter survey of 97 patients of whom 52% had t(8;21)(q22;q22) and 48% had inv(16)(p13q22)/t(16;16)(p13;q22). The median age of the patients was 53 years (range, 19-81). Complete remission after anthracycline-based induction (n=86) and non-intensive therapy (n=11) was achieved in 97% and 36% of the patients, respectively. The median follow-up was 4.43 years (95% confidence interval [95% CI]: 3.35-7.39 years). The median survival after intensive and non-intensive treatment was not reached and 0.96 years, respectively. Among intensively treated patients, inv(16) with trisomy 22 (n=11) was associated with a favorable 4-year relapse-free survival rate of 80% (95% CI: 59-100%) as compared to 38% (95% CI: 27-54%; P=0.02) in all other patients with CBFAML/ FLT3-ITD (n=75). Overall, 24 patients underwent allogeneic hematopoietic cell transplantation (HCT), 12 in first complete remission and 12 after relapse. Allogeneic HCT in first complete remission was not beneficial (P=0.60); however, allogeneic HCT seemed to improve median survival in relapsed patients compared to that of patients treated with chemotherapy (not reached vs. 0.6 years, respectively; P=0.002). Excluding patients with inv(16) with trisomy 22, our data indicate that compathe outcome of CBF-AML patients with FLT3-ITD may be inferior to that of patients without FLT3-ITD (based on previously published data), suggesting that prognostically CBF-AML patients with FLT3-ITD should not be classified as favorable-risk. FLT3-inhibitors may improve the outcome of these patients. 
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