S K Metzelder1, T Schroeder2, M Lübbert3, M Ditschkowski4, K Götze5, S Scholl6, R G Meyer7, P Dreger8, N Basara9, M F Fey10, H R Salih11, A Finck3, T Pabst10, A Giagounidis12, G Kobbe2, E Wollmer1, J Finke3, A Neubauer1, A Burchert13. 1. Philipps Universität Marburg, Universitätsklinikum Gießen und Marburg, Standort Marburg, Klinik für Hämatologie, Onkologie und Immunologie, Marburg, Germany. 2. University of Duesseldorf, Medical Faculty, Department of Hematology, Oncology and Clinical Immunology, Duesseldorf, Germany. 3. University of Freiburg, Department of Hematology/Oncology, Freiburg, Germany. 4. Department of Bone Marrow Transplantation, West German Cancer Center, University Hospital Essen, Germany. 5. Department of Internal Medicine III, Technische Universität München, Munich, Germany. 6. Abteilung Hämatologie/Onkologie, Universitätsklinikum Jena, Jena, Germany. 7. St.-Johannes-Hospital Dortmund, Klinik für Innere Medizin II, Dortmund, Germany. 8. Department of Medicine, University of Heidelberg, Heidelberg, Germany. 9. Malteser Krankenhaus St. Franziskus-Hospital, Medizinische Klinik I, Flensburg, Germany. 10. Department of Medical Oncology, Inselspital and University of Bern, Bern, Switzerland. 11. Department of Hematology/Oncology, Eberhard Karls-University, Tuebingen, Germany. 12. Marien Hospital Düsseldorf, Klinik für Onkologie, Hämatologie und Palliativmedizin, Düsseldorf, Germany. 13. Philipps Universität Marburg, Universitätsklinikum Gießen und Marburg, Standort Marburg, Klinik für Hämatologie, Onkologie und Immunologie, Marburg, Germany. Electronic address: burchert@staff.uni-marburg.de.
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.
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 AMLpatients, 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.
Authors: Keith W Pratz; Michelle A Rudek; B Douglas Smith; Judith Karp; Ivana Gojo; Amy Dezern; Richard J Jones; Jackie Greer; Christopher Gocke; Maria R Baer; Vu H Duong; Gary Rosner; Marianna Zahurak; John J Wright; Ashkan Emadi; Mark Levis Journal: Biol Blood Marrow Transplant Date: 2019-09-21 Impact factor: 5.742
Authors: Richard A Larson; Sumithra J Mandrekar; Lucas J Huebner; Ben L Sanford; Kristina Laumann; Susan Geyer; Clara D Bloomfield; Christian Thiede; Thomas W Prior; Konstanze Döhner; Guido Marcucci; Maria Teresa Voso; Rebecca B Klisovic; Ilene Galinsky; Andrew H Wei; Jorge Sierra; Miguel A Sanz; Joseph M Brandwein; Theo de Witte; Dietger Niederwieser; Frederick R Appelbaum; Bruno C Medeiros; Martin S Tallman; Jürgen Krauter; Richard F Schlenk; Arnold Ganser; Hubert Serve; Gerhard Ehninger; Sergio Amadori; Insa Gathmann; Hartmut Döhner; Richard M Stone Journal: Leukemia Date: 2021-03-02 Impact factor: 11.528