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Trial of Enasidenib Maintenance Post Allogeneic Hematopoietic Cell Transplant in Patients With IDH-2 Mutated AML.

Trial of Enasidenib Maintenance Post Allogeneic Hematopoietic Cell Transplant in Patients With IDH-2 Mutated AML. Transplantation and cellular therapy Salhotra, A., Bejanyan, N., Yang, D., Mokhtari, S., Knobler, D., Park, G., Malki, M. M., Sandhu, K. S., Faramand, R., Aldoss, I., Artz, A. S., Aribi, A., Elmariah, H., Sahebi, F., Mansour, J., Ball, B. J., Agrawal, V., Li, L., Pullarkat, V. A., Forman, S. J., Marcucci, G., Stein, A. S., Nakamura, R. 2025

Abstract

In AML patients with IDH-2 mutation who undergo allogeneic hematopoietic cell transplantation (alloHCT), the relapse rate at 1 year is around 30%. We designed a two-center, open-label, single arm pilot trial to investigate the safety and tolerability of enasidenib as post-HCT maintenance in AML patients carrying IDH-2 mutation (NCT03728335).Recipients of first alloHCT who had AML with IDH2 mutation at the time of diagnosis were eligible for maintenance if in complete remission (CR) at day +30 post-HCT, had ECOG PS =2, adequate hematopoietic recovery, patients with grade =2 acute graft-versus-host disease (GVHD) were excluded. Enasidenib was given at dose of 100 mg/day between days 50-120 post-HCT for 2 years in 28 days cycles. The primary objective was to evaluate the safety and tolerability of enasidenib as post-HCT maintenance therapy and secondary objectives were to assess clinical outcomes.Total of 15 patients were enrolled to this pilot study with median age of 58 years and median follow up of 24 months. Pre-transplant remission status was CR1 in majority of patients (73%) and 26.7% were adverse risk AML by 2022 ELN classification. Most patients (80%) received reduced intensity conditioning from HLA matched related/unrelated donor (73%) and post-transplant cyclophosphamide-based GVHD prophylaxis was given in 60% of the patients. Study patients completed a median of 24 (range: 1-24) enasidenib cycles. Enasidenib maintenance therapy was well tolerated, discontinuation rate was 20% and 12 patients completed 2-year maintenance of enasidenib. Grade =3 adverse effects were mainly hematologic and GI toxicities. The 2-year overall survival (OS) and leukemia-free survival (LFS) was 100%- and 2-year chronic GVHD-free and relapse-free survival (CRFS) was 80%.Post-HCT enasidenib maintenance therapy was safe and well-tolerated, resulting in favorable relapse control and survival outcomes in AML patients carrying IDH2 mutations. Phase 2 multicenter study investigating effectiveness of enasidenib maintenance as a relapse-prevention strategy after allo-HCT is ongoing.

View details for DOI 10.1016/j.jtct.2025.09.013

View details for PubMedID 40976487