Olutasidenib Alone or in Combination With Azacitidine as a Bridge to Transplant in Relapsed/Refractory mIDH1 AML

Allogeneic hematopoietic stem cell transplantation (HSCT) remains the only curative option for adults with relapsed/refractory (R/R) acute myeloid leukemia (AML) [1, 2], but only a minority of patients with R/R AML achieve remission with conventional salvage regimens and can proceed to transplant [3–5]. The availability of novel targeted therapies directed against specific genetic alterations may enable more patients to achieve remission and become eligible for HSCT [4, 6]. Somatic mutations in the isocitrate dehydrogenase 1 gene (mIDH1) occur in 7%–14% of AML patients [2]. Olutasidenib (OLU) is a potent, selective, oral inhibitor of mIDH1 that is FDA-approved for treatment of R/R mIDH1 AML [7, 8]. Approval was based on a phase 2 pivotal cohort (N = 153) of the phase 1/2 open-label, multicenter, multicohort study (NCT02719574), in which 35% of patients with R/R mIDH1 AML achieved complete remission (CR) or CR with partial hematologic recovery (CRh), including 32% with CR, with a median duration of CR/CRh of 25.3 months [7]. We conducted a post hoc analysis of adults in this study with R/R mIDH1 AML who were treated with OLU and subsequently underwent HSCT [1, 7, 9].