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Front. Oncol.
Sec. Hematologic Malignancies
Volume 14 - 2024 | doi: 10.3389/fonc.2024.1391743
This article is part of the Research Topic Allogenic Hematopoietic Cell Transplant in Hematological Malignancies: Controversies and Perspective View all 9 articles

Sorafenib maintenance in FLT3-ITD mutated AML after allogeneic HCT: a real world, single center experience

Provisionally accepted
  • 1 San Raffaele Scientific Institute (IRCCS), Milan, Lombardy, Italy
  • 2 Vita-Salute San Raffaele University, Milan, Lombardy, Italy

The final, formatted version of the article will be published soon.

    Despite allogeneic hematopoietic stem cell transplant (allo-HCT) and the development of novel FLT3 inhibitors in both induction (midostaurin) and in the relapsed/refractory setting (gilteritinib), FLT3-ITD mutated leukemia (FLT3-ITD+ AML) still represents a challenge for modern hematology. Sorafenib is to this date the only inhibitor that demonstrated efficacy in improving both progression free and overall survival as post HCT maintenance therapy, even if its use in this setting has not been approved so far by regulatory agencies. The aim of our study was to evaluate the feasibility, safety and efficacy of sorafenib maintenance in preventing early relapse in FLT3-ITD+ AML after HCT in a single center experience. We analyzed 26 consecutive patients who received post HCT 2-year maintenance with sorafenib at our center between 2017 and 2023. Median time from HCT to sorafenib start was 130 days and median dosage was 200 mg per day. Two (8%) and three (12%) patients discontinued maintenance due to toxicity and disease relapse, respectively. 8 (31%) patients terminated the 2-year maintenance and stopped sorafenib, while 13 patients are still under treatment. Overall, 21/26 patients (81%) are alive and in stable complete remission as outlined by a 2 year disease free survival of 83.61%. No major long-term toxicity were reported at last follow up. Our real-world experience supports the use of sorafenib as a feasible and effective therapeutic option in post HCT maintenance for FLT3-ITD+ AML.

    Keywords: Sorafenib, Maintenance, Allogeneic stem cell transplanation, FLT3 ITD, Acute Myeloid Leukemia

    Received: 26 Feb 2024; Accepted: 21 May 2024.

    Copyright: © 2024 Diral, Funari, Bruno, Greco, Clerici, Marktel, Farina, Mastaglio, Vago, Piemontese, Peccatori, Corti, Bernardi, Ciceri and Lupo Stanghellini. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.

    * Correspondence: Elisa Diral, San Raffaele Scientific Institute (IRCCS), Milan, 20132, Lombardy, Italy

    Disclaimer: All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.