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@ARTICLE{Dhner:179738,
      author       = {H. Döhner and D. Weber and J. Krzykalla$^*$ and W. Fiedler
                      and G. G. Wulf and H. R. Salih and M. Lübbert and M. Kühn
                      and T. Schroeder and H. Salwender and K. S. Götze and J.
                      Westermann and L. Fransecky and K. Mayer and B. Hertenstein
                      and M. Ringhoffer and H.-J. Tischler and S.
                      Machherndl-Spandl and A. Schrade and P. Paschka and V. I.
                      Gaidzik and F. Theis and F. R. Thol and M. Heuser and R. F.
                      Schlenk$^*$ and L. Bullinger and M. Saadati and A.
                      Benner$^*$ and R. A. Larson and R. M. Stone and K. Döhner
                      and A. Ganser},
      title        = {{M}idostaurin plus intensive chemotherapy for younger and
                      older {P}atients with {AML} and {FLT}3 internal tandem
                      duplications.},
      journal      = {Blood advances},
      volume       = {6},
      number       = {18},
      issn         = {2473-9529},
      address      = {Washington, DC},
      publisher    = {American Society of Hematology},
      reportid     = {DKFZ-2022-00865},
      pages        = {5345-5355},
      year         = {2022},
      note         = {2022 Sep 27;6(18):5345-5355},
      abstract     = {We conducted a single-arm phase-II trial (AMLSG 16-10) to
                      evaluate midostaurin with intensive chemotherapy followed by
                      allogeneic hematopoietic-cell transplantation (HCT) and a
                      one-year midostaurin maintenance therapy in adult patients
                      with acute myeloid leukemia (AML) and FLT3 internal tandem
                      duplication (ITD). Patients 18-70 years of age with newly
                      diagnosed FLT3-ITD-positive AML were eligible. Primary and
                      key secondary endpoints were event-free (EFS) and overall
                      survival (OS). Results were compared to a historical cohort
                      of 415 patients treated on 5 prior AMLSG trials; statistical
                      analysis was performed using a double-robust adjustment with
                      propensity score weighting and covariate adjustment. Results
                      were also compared to patients (18-59yrs) treated on the
                      placebo arm of the CALGB 10603/RATIFY trial. The trial
                      accrued 440 patients (18-60yrs, n=312; 61-70yrs, n=128). In
                      multivariate analysis, EFS was significantly in favor of
                      patients treated within the AMLSG 16-10 trial compared to
                      the AMLSG control (HR 0.55; P<0.001); both in younger (HR
                      0.59; P<0.001) and older patients (HR 0.42; P<0.001).
                      Multivariate analysis also showed a significant beneficial
                      effect on OS compared to the AMLSG control (HR 0.57;
                      P<0.001) as well as to the CALGB 10603/RATIFY trial (HR
                      0.71; p=0.005). The treatment effect of midostaurin remained
                      significant in sensitivity analysis including allogeneic HCT
                      as a time-dependent covariate. Addition of midostaurin to
                      chemotherapy was safe in younger and older patients. In
                      comparison to historical controls, the addition of
                      midostaurin to intensive therapy led to a significant
                      improvement in outcome in younger and older patients with
                      AML and FLT3-ITD.},
      cin          = {C060 / W010},
      ddc          = {610},
      cid          = {I:(DE-He78)C060-20160331 / I:(DE-He78)W010-20160331},
      pnm          = {313 - Krebsrisikofaktoren und Prävention (POF4-313)},
      pid          = {G:(DE-HGF)POF4-313},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:35486475},
      doi          = {10.1182/bloodadvances.2022007223},
      url          = {https://inrepo02.dkfz.de/record/179738},
}