Cancer Treatment Today

There is now a randomized blinded phase III trial supporting this therapy.
In an early‐phase study of 22 patients with FLT3‐ITD AML treated with sorafenib following HCT, PFS and OS rates at 1 year were 85% and 95%, respectively.32 In a retrospective analysis, patients with FLT3‐ITD AML treated with sorafenib maintenance after HCT during the first CR (n = 26) were compared with matched patients who were not treated with sorafenib (n = 55). The treated patients showed significantly improved 2‐year OS (81% vs 62%, P = .029), PFS (82% vs 53%, P = .0081), and a lower relapse rate (8.2% vs 37.7%, P = .0077) compared with patients not treated with sorafenib.
A randomized, double blind, placebo‐controlled study (SORMAIN) compared sorafenib maintenance vs placebo in patients (n = 83), with FLT3‐ITD AML in complete hematologic remission after HCT.36 At a median follow up of 41.8 months after randomization, median relapse‐free survival was 30.9 months in the placebo group vs not reached in the sorafenib group. This corresponded to a 2‐year relapse‐free survival rate of 53% with placebo vs 85% with sorafenib (P = .0135).

A recent placebo-controlled, double-blind phase II trial (SORMAIN; German Clinical Trials Register: DRKS00000591)concluded that Sorafenib maintenance therapy reduces the risk of relapse and death after HCT for FLT3-ITD–positive AML. After this trial, Reliable Evidence shows that the prevailing opinion among experts regarding the drug, device, medical treatment or procedure is that further studies or clinical trails
are not necessary to determine its maximum tolerated dose, its toxicity, its safety, its
efficacy or its efficacy as compared with a standard means of treatment or diagnosis.

A Burchert et al, Sorafenib Maintenance After Allogeneic Hematopoietic Stem Cell Transplantation for Acute Myeloid Leukemia With FLT3–Internal Tandem Duplication Mutation (SORMAIN) Journal of Clinical Oncology 38, no. 26 (September 10, 2020) 2993-3002.

Brunner AM, Li S, Fathi AT, et al. Haematopoietic cell transplantation with and without sorafenib maintenance for patients with FLT3‐ITD acute myeloid leukaemia in first complete remission. Br J Haematol. 2016;175:496‐504.

Metzelder SK1, Schroeder T, Finck A, Scholl S, Fey M, Götze K, Linn YC, Kröger M, Reiter A, Salih HR, Heinicke T, Stuhlmann R, Müller L, Giagounidis A, Meyer RG, Brugger W, Vöhringer M, Dreger P, Mori M, Basara N, Schäfer-Eckart K, Schultheis B, Baldus C, Neubauer A, Burchert A. High activity of sorafenib in FLT3-ITD-positive acute myeloid leukemia synergizes with allo-immune effects to induce sustained responses.
Leukemia. 2012 Nov;26(11):2353-9.

R. H Carson. AML: Sorafenib May Hit More than FLT3
Oncology Times:10 February 2015 – Volume 37 – Issue 3 – p 32–33