Durable remissions with ivosidenib in IDH1-mutated relapsed or refractory AML

Mutations in the gene encoding isocitrate dehydrogenase 1 (IDH1) are implicated in as many as 10% of acute myeloid leukemia (AML) cases, raising the prospect of treatment with an IDH1 inhibitor.

Mutations in the gene encoding isocitrate dehydrogenase 1 (IDH1) are implicated in as many as 10% of acute myeloid leukemia (AML) cases, raising the prospect of treatment with an IDH1 inhibitor. To test this possibility, researchers evaluated safety and efficacy endpoints in a sample population of patients with IDH1-mutated AML who took oral ivosidenib 500 mg once a day. The regimen appeared to generate favorable outcomes. In the safety analysis of 258 participants, the drug was associated with a low frequency of grade 3 or higher treatment-related adverse events, as well as transfusion independence, in patients with relapsed or refractory IDH1-mutated AML. The efficacy cohort, meanwhile, consisted of 125 participants, 30.4% of whom achieved complete remission or complete remission with partial hematological recovery. Among those 34 patients, 7 had no residual detectable IDH1 mutations on digital PCR assay, signifying molecular remission.