Haematologica (Aug 2021)

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

  • 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,
  • Mark J. Levis

DOI
https://doi.org/10.3324/haematol.2021.278645
Journal volume & issue
Vol. 107, no. 4

Abstract

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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.