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.

Department of Clinical Haematology Centro Hospitalar e Universitário de Coimbra Coimbra

Department of Internal Medicine 5 Heidelberg University Hospital Heidelberg

Department of Internal Medicine and Haematology 3rd Faculty of Medicine Charles University and Faculty Hospital Kralovske Vinohrady Charles University Prague Czech Republic

Department of Internal Medicine Hematology and Oncology Masaryk University and University Hospital Brno Brno Czech Republic

Department of Internal Medicine Hematology and Oncology Masaryk University and University Hospital Brno Brno Czech Republic; Institute of Hematology and Blood Transfusion Prague Czech Republic

Department of Medical Oncology Dana Farber Cancer Institute Boston MA

Department of Medicine 1 University Hospital Carl Gustav Carus Dresden

Division of Hematology and Oncology Abramson Cancer Center Perelman School of Medicine at the University of Pennsylvania Philadelphia PA

Division of Hematology Department of Internal Medicine Mayo Clinic Rochester Minnesota

Hematology Department Hospital Universitari i Politècnic La Fe València Spain; CIBERONC Instituto Carlos 3 Madrid

Hospital General Alicante

Institute of Hematology and Blood Transfusion Prague Czech Republic

Massachusetts General Hospital Boston MA

Medical Clinic and Policlinic 1 Hematology and Cellular Therapy University Hospital Leipzig Leipzig

Medical Clinic and Policlinic 1 Hematology and Cellular Therapy University Hospital Leipzig Leipzig Germany; Laboratory for Leukemia Diagnostics Department of Medicine 3 University Hospital LMU Munich Munich

Medical Clinic and Policlinic 1 Hematology and Cellular Therapy University Hospital Leipzig Leipzig Germany; NCT Trial Center National Center of Tumor Diseases German Cancer Research Center Heidelberg Germany; Department of Internal Medicine 5 Heidelberg University Hospital Heidelberg

NCT Trial Center National Center of Tumor Diseases German Cancer Research Center Heidelberg University Hospital Heidelberg

Sidney Kimmel Comprehensive Cancer Center Johns Hopkins University Baltimore Maryland

Haematologica. 2022 Apr 01;107(4):783-784. doi: 10.3324/haematol.2021.279409. PubMed

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