Sorafenib Maintenance After Allogeneic Hematopoietic Stem Cell Transplantation for Acute Myeloid Leukemia With FLT3-Internal Tandem Duplication Mutation (SORMAIN)
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Sorafenib Maintenance After Allogeneic Hematopoietic Stem Cell Transplantation for Acute Myeloid Leukemia With FLT3-Internal Tandem Duplication Mutation (SORMAIN). / Burchert, Andreas; Bug, Gesine; Fritz, Lea V; Finke, Jürgen; Stelljes, Matthias; Röllig, Christoph; Wollmer, Ellen; Wäsch, Ralph; Bornhäuser, Martin; Berg, Tobias; Lang, Fabian; Ehninger, Gerhard; Serve, Hubert; Zeiser, Robert; Wagner, Eva-Maria; Kröger, Nicolaus; Wolschke, Christine; Schleuning, Michael; Götze, Katharina S; Schmid, Christoph; Crysandt, Martina; Eßeling, Eva; Wolf, Dominik; Wang, Ying; Böhm, Alexandra; Thiede, Christian; Haferlach, Torsten; Michel, Christian; Bethge, Wolfgang; Wündisch, Thomas; Brandts, Christian; Harnisch, Susanne; Wittenberg, Michael; Hoeffkes, Heinz-Gert; Rospleszcz, Susanne; Burchardt, Alexander; Neubauer, Andreas; Brugger, Markus; Strauch, Konstantin; Schade-Brittinger, Carmen; Metzelder, Stephan K.
In: J CLIN ONCOL, Vol. 38, No. 26, 10.09.2020, p. 2993-3002.Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review
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TY - JOUR
T1 - Sorafenib Maintenance After Allogeneic Hematopoietic Stem Cell Transplantation for Acute Myeloid Leukemia With FLT3-Internal Tandem Duplication Mutation (SORMAIN)
AU - Burchert, Andreas
AU - Bug, Gesine
AU - Fritz, Lea V
AU - Finke, Jürgen
AU - Stelljes, Matthias
AU - Röllig, Christoph
AU - Wollmer, Ellen
AU - Wäsch, Ralph
AU - Bornhäuser, Martin
AU - Berg, Tobias
AU - Lang, Fabian
AU - Ehninger, Gerhard
AU - Serve, Hubert
AU - Zeiser, Robert
AU - Wagner, Eva-Maria
AU - Kröger, Nicolaus
AU - Wolschke, Christine
AU - Schleuning, Michael
AU - Götze, Katharina S
AU - Schmid, Christoph
AU - Crysandt, Martina
AU - Eßeling, Eva
AU - Wolf, Dominik
AU - Wang, Ying
AU - Böhm, Alexandra
AU - Thiede, Christian
AU - Haferlach, Torsten
AU - Michel, Christian
AU - Bethge, Wolfgang
AU - Wündisch, Thomas
AU - Brandts, Christian
AU - Harnisch, Susanne
AU - Wittenberg, Michael
AU - Hoeffkes, Heinz-Gert
AU - Rospleszcz, Susanne
AU - Burchardt, Alexander
AU - Neubauer, Andreas
AU - Brugger, Markus
AU - Strauch, Konstantin
AU - Schade-Brittinger, Carmen
AU - Metzelder, Stephan K
PY - 2020/9/10
Y1 - 2020/9/10
N2 - PURPOSE: Despite undergoing allogeneic hematopoietic stem cell transplantation (HCT), patients with acute myeloid leukemia (AML) with internal tandem duplication mutation in the FMS-like tyrosine kinase 3 gene (FLT3-ITD) have a poor prognosis, frequently relapse, and die as a result of AML. It is currently unknown whether a maintenance therapy using FLT3 inhibitors, such as the multitargeted tyrosine kinase inhibitor sorafenib, improves outcome after HCT.PATIENTS AND METHODS: In a randomized, placebo-controlled, double-blind phase II trial (SORMAIN; German Clinical Trials Register: DRKS00000591), 83 adult patients with FLT3-ITD-positive AML in complete hematologic remission after HCT were randomly assigned to receive for 24 months either the multitargeted and FLT3-kinase inhibitor sorafenib (n = 43) or placebo (n = 40 placebo). Relapse-free survival (RFS) was the primary endpoint of this trial. Relapse was defined as relapse or death, whatever occurred first.RESULTS: With a median follow-up of 41.8 months, the hazard ratio (HR) for relapse or death in the sorafenib group versus placebo group was 0.39 (95% CI, 0.18 to 0.85; log-rank P = .013). The 24-month RFS probability was 53.3% (95% CI, 0.36 to 0.68) with placebo versus 85.0% (95% CI, 0.70 to 0.93) with sorafenib (HR, 0.256; 95% CI, 0.10 to 0.65; log-rank P = .002). Exploratory data show that patients with undetectable minimal residual disease (MRD) before HCT and those with detectable MRD after HCT derive the strongest benefit from sorafenib.CONCLUSION: Sorafenib maintenance therapy reduces the risk of relapse and death after HCT for FLT3-ITD-positive AML.
AB - PURPOSE: Despite undergoing allogeneic hematopoietic stem cell transplantation (HCT), patients with acute myeloid leukemia (AML) with internal tandem duplication mutation in the FMS-like tyrosine kinase 3 gene (FLT3-ITD) have a poor prognosis, frequently relapse, and die as a result of AML. It is currently unknown whether a maintenance therapy using FLT3 inhibitors, such as the multitargeted tyrosine kinase inhibitor sorafenib, improves outcome after HCT.PATIENTS AND METHODS: In a randomized, placebo-controlled, double-blind phase II trial (SORMAIN; German Clinical Trials Register: DRKS00000591), 83 adult patients with FLT3-ITD-positive AML in complete hematologic remission after HCT were randomly assigned to receive for 24 months either the multitargeted and FLT3-kinase inhibitor sorafenib (n = 43) or placebo (n = 40 placebo). Relapse-free survival (RFS) was the primary endpoint of this trial. Relapse was defined as relapse or death, whatever occurred first.RESULTS: With a median follow-up of 41.8 months, the hazard ratio (HR) for relapse or death in the sorafenib group versus placebo group was 0.39 (95% CI, 0.18 to 0.85; log-rank P = .013). The 24-month RFS probability was 53.3% (95% CI, 0.36 to 0.68) with placebo versus 85.0% (95% CI, 0.70 to 0.93) with sorafenib (HR, 0.256; 95% CI, 0.10 to 0.65; log-rank P = .002). Exploratory data show that patients with undetectable minimal residual disease (MRD) before HCT and those with detectable MRD after HCT derive the strongest benefit from sorafenib.CONCLUSION: Sorafenib maintenance therapy reduces the risk of relapse and death after HCT for FLT3-ITD-positive AML.
U2 - 10.1200/JCO.19.03345
DO - 10.1200/JCO.19.03345
M3 - SCORING: Journal article
C2 - 32673171
VL - 38
SP - 2993
EP - 3002
JO - J CLIN ONCOL
JF - J CLIN ONCOL
SN - 0732-183X
IS - 26
ER -
