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Kayser, Sabine; Kramer, Michael; Martinez-Cuadron, David; Grenet, Justin; Metzeler, Klaus H.; Sustkova, Zuzana; Luskin, Marlise R.; Brunner, Andrew M.; Elliott, Michelle A.; Gil, Cristina; Marini, Sandra Casal; Racil, Zdenek; Cetkovsky, Petr; Novak, Jan; Perl, Alexander E.; Platzbecker, Uwe; Stoelzel, Friedrich; Ho, Anthony D.; Thiede, Christian; Stone, Richard M.; Roellig, Christoph; Montesinos, Pau; Schlenk, Richard F. und Levis, Mark J. (2022): Characteristics and outcome of patients with core-binding factor acute myeloid leukemia and FLT3-ITD: results from an international collaborative study. In: Haematologica, Bd. 107, Nr. 4: S. 836-843

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Abstract

The aim of this study was to evaluate the prognostic impact of FLT3-ITD in core-binding factor acute myeloid leukemia (CBF-AML) 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 CBF-AML/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 the 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 favorable-risk. FLT3-inhibitors may improve the outcome of these patients.

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