Highlights
- •Sorafenib induces long-term survival in Fms-like tyrosine kinase 3 internal tandem duplication (FLT3-ITD)+ acute myeloid leukaemia (AML) relapsing after allogeneic stem cell transplantation (allo-SCT).
- •Sorafenib cures a minority of FLT3-ITD+ AML relapsing after allo-SCT.
- •Achievement of molecular negativity is strongly associated with cure.
Abstract
Background
Fms-like tyrosine kinase 3 internal tandem duplication (FLT3-ITD)–positive acute myeloid
leukaemia (AML) relapsing after allogeneic stem cell transplantation (allo-SCT) has
a dismal prognosis with limited therapeutic options. FLT3-ITD kinase inhibition is
a reasonable but palliative experimental treatment alternative in this situation.
Information on long-term outcome is not available.
Methods
We performed a long-term follow-up analysis of a previously reported cohort of 29
FLT3-ITD–positive AML patients, which were treated in relapse after allo-SCT with
sorafenib monotherapy.
Findings
With a median follow-up of 7.5 years, 6 of 29 patients (21%) are still alive. Excluding
one patient who received a second allo-SCT, five patients (17%) achieved sustained
complete remissions with sorafenib. Four of these patients are in treatment-free remission
for a median of 4.4 years.
Interpretation
Sorafenib may enable cure of a proportion of very poor risk FLT3-ITD–positive AML
relapsing after allo-SCT.
Keywords
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Article info
Publication history
Published online: October 18, 2017
Accepted:
September 8,
2017
Received in revised form:
September 3,
2017
Received:
June 25,
2017
Footnotes
☆Presented in part at the annual meeting of the Deutsche Gesellschaft für Hämatologie und Medizinische Onkologie, Leipzig 2016.
Identification
Copyright
© 2017 Elsevier Ltd. All rights reserved.
