Among patients with FLT3 internal tandem duplication (FLT3-ITD)-mutated acute myeloid leukemia (AML) undergoing allogeneic hemopoietic stem cell transplantation (HSCT), maintenance sorafenib appears to improve long-term overall survival (OS), according to research published in The Lancet Haematology.

Although HSCT has improved outcomes among patients with AML with FLT3 ITD, maintenance therapies are needed. Sorafenib, a multikinase inhibitor, has previously been shown to be safe and potentially effective in this population, according to initial results from a randomized phase 3 study ( Identifier: NCT02474290). In the present paper, researchers give the results of a post-hoc analysis on 5-year follow-up data from the phase 3 study.

Overall, 202 patients were randomly assigned to receive sorafenib maintenance after HSCT (100 patients) or no maintenance (102 patients); 1 patient from the latter group withdrew consent.

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The median follow-up was 60.4 months. Analysis showed that, compared with the no maintenance group, patients who received sorafenib had improved OS (55.9% vs 72%, respectively; hazard ratio, 0.55; P =.011).

In the control vs sorafenib groups, leukemia-free survival (49% vs 70%, respectively; P =.0007), graft-vs-host disease–free survival (39.2% vs 58%; P =.003), and cumulative relapse incidence (36.3% vs 15%; P =.003) were all improved in the sorafenib group.

The 5-year chronic graft vs host disease incidence rates were, however, similar in the sorafenib (54%) and control (51%) groups (P =.73). No treatment-related deaths were noted.

“Sorafenib maintenance after transplantation should be recommended for patients with FLT3-ITD acute myeloid leukaemia, and other FLT3 inhibitors warrant further investigation in larger prospective studies,” the authors wrote in their report.


Xuan L, Wang Y, Yang K, et al. Sorafenib maintenance after allogeneic haemopoietic stem-cell transplantation in patients with FLT3-ITD acute myeloid leukaemia: long-term follow-up of an open-label, multicentre, randomised, phase 3 trial. Lancet Haematol. Published online July 3, 2023. doi:10.1016/S2352-3026(23)00117-5