Sorafenib or placebo in patients with newly diagnosed acute myeloid leukaemia: long-term follow-up of the randomized controlled SORAML trial
Christoph Röllig, Hubert Serve, Richard Noppeney, Maher Hanoun, Utz Krug, Claudia D. Baldus, Christian Brandts, Volker Kunzmann, Hermann Einsele, Alwin Krämer, Carsten Müller‐Tidow, Kerstin Schäfer‐Eckart, Andreas Neubauer, Andreas Burchert, Aristoteles Giagounidis, Stefan W. Krause, Andréas Mackensen, Walter E. Aulitzky, Regina Herbst, Mathias Hänel, Norbert Frickhofen, Johannes Kullmer, Ulrich Kaiser, Alexander Kiani, Hartmut Link, Thomas Geer, Albrecht Reichle, Christian Junghanß, Roland Repp, Achim Meinhardt, Heinz Dürk, Ina‐Maria Klut, Martin Bornhäuser, Markus Schaich, Stefani Parmentier, Martin Görner, Christian Thiede, Malte von Bonin, Uwe Platzbecker, Johannes Schetelig, Michael Krämer, Wolfgang E. Berdel, Gerhard Ehninger, for the Study Alliance Leukaemia (SAL)
Abstract
Early results of the randomized placebo-controlled SORAML trial showed that, in patients with newly diagnosed acute myeloid leukaemia (AML), sorafenib led to a significant improvement in event-free (EFS) and relapse-free survival (RFS). In order to describe second-line treatments and their implications on overall survival (OS), we performed a study after a median follow-up time of 78 months. Newly diagnosed fit AML patients aged ≤60 years received sorafenib (n = 134) or placebo (n = 133) in addition to standard chemotherapy and as maintenance treatment. The 5-year EFS was 41 versus 27% (HR 0.68; p = 0.011) and 5-year RFS was 53 versus 36% (HR 0.64; p = 0.035). Allogeneic stem cell transplantation (allo SCT) was performed in 88% of the relapsed patients. Four years after salvage allo SCT, the cumulative incidence of relapse was 54 versus 35%, and OS was 32 versus 50%. The 5-year OS from randomization in all study patients was 61 versus 53% (HR 0.82; p = 0.282). In conclusion, the addition of sorafenib to chemotherapy led to a significant prolongation of EFS and RFS. Although the OS benefit did not reach statistical significance, these results confirm the antileukaemic activity of sorafenib.