Differential impact of allelic ratio and insertion site in FLT3-ITD-positive AML with respect to allogeneic transplantation

Richard F. Schlenk, Sabine Kayser, Lars Bullinger, Guido Kobbe, Jochen Casper, Mark Ringhoffer, Gerhard Held, Peter Brossart, Michael Lübbert, Helmut R. Salih, Thomas Kindler, Heinz A. Horst, Gerald Wulf, David Nachbaur, Katharina Götze, Alexander Lamparter, Peter Paschka, Verena I. Gaidzik, Veronica Teleanu, Daniela SpäthAxel Benner, Jürgen Krauter, Arnold Ganser, Hartmut Döhner, Konstanze Döhner

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327 Scopus citations

Abstract

The objective was to evaluate the prognostic and predictive impact of allelic ratio and insertion site (IS) of internal tandem duplications (ITDs), as well as concurrent gene mutations, with regard to postremission therapy in 323 patients with FLT3-ITD-positive acute myeloid leukemia (AML). Increasing FLT3-ITD allelic ratio (P 5 .004) and IS in the tyrosine kinase domain 1 (TKD1, P 5 .06) were associated with low complete remission (CR) rates. After postremission therapy including intensive chemotherapy (n 5 121) or autologous hematopoietic stem cell transplantation (HSCT, n 5 17), an allelic ratio ≥ 0.51 was associated with an unfavorable relapse-free (RFS,P5.0008) and overall survival (OS, P5.004); after allogeneic HSCT (n593), outcome was significantly improved in patients with a high allelic ratio (RFS, P5.02; OS,P5.03), whereas no benefit was seen in patients with a low allelic ratio (RFS, P 5 .38; OS, P 5 .64). Multivariable analyses revealed a high allelic ratio as a predictive factor for the beneficial effect of allogeneic HSCT; ITD IS in TKD1 remained an unfavorable factor, whereas no prognostic impact of concurrent gene mutations was observed. The clinical trials described herein were previously published or are registered as follows: AMLHD93 and AMLHD98A, previously published; AML SG 07-04, ClinicalTrials.gov identifier #NCT00151242.

Original languageEnglish
Pages (from-to)3441-3449
Number of pages9
JournalBlood
Volume124
Issue number23
DOIs
StatePublished - 30 Sep 2014

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