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. 2024 Nov;103(11):4801-4803.
doi: 10.1007/s00277-024-06012-3. Epub 2024 Sep 25.

15-year remission in refractory FLT3-mutated AML attained by sorafenib

Christoph Rummelt  1 , Dietmar Pfeifer  1 , Ralph Wäsch  1 , Robert Zeiser  1 , Justus Duyster  1 , Jürgen Finke  1 , Michael Lübbert  2
Affiliations

15-year remission in refractory FLT3-mutated AML attained by sorafenib

Christoph Rummelt et al. Ann Hematol. 2024 Nov.
No abstract available

Keywords: Acute myeloid leukemia; Donor lymphocyte infusion; FLT3-ITD; Sorafenib; WT1.

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Conflict of interest statement

The authors declare no competing interests.

Figures

Fig. 1
Fig. 1
Clinical course of a primary refractory FLT3-ITD-positive AML patient achieving a durable remission with a combination of sorafenib and donor lymphocyte infusions. The patient (38 year-old female) was diagnosed with AML with the following mutations: FLT3-ITD (high), NPM1D, DNMT3A and WT1 R462W. Therapy was initiated within the RATIFY trial with two 7 + 3 (+ placebo) inductions and one high-dose cytarabine consolidation, but was refractory with 15% BM blasts hereafter (*). HSCT from an HLA matched unrelated male donor was conducted after BuCy conditioning, and GvHD prophylaxis was performed with 10 mg campath (2x), cyclosporine and MM (**). She experienced full-blown hematologic progression only 5 weeks after HCT, so cyclosporine and MMF were stopped. At that time FLT3-ITD and NPM1 were positive and a small interstitial deletion on chromosome 11, bands p11p15 was acquired (***). Sorafenib 400 mg 1-0-1 was initiated which led to CR with mixed chimerism and NPM1 persistence (****). After two DLIs, she achieved a complete chimerism and a NPM1 3 log decrease (*****). After one more DLI, MRD was negative with no more detectable NPM1. Therapy was stopped after 7.5 years on continued sorafenib therapy and 10 additional DLIs. Since then, she remains in ongoing unmaintained MRD negative CR (7.5 years until today)
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