Guadecitabine improved relapse-free survival in high-risk acute myeloid leukemia and myelodysplastic syndrome patients after transplant: phase II results from a single center

Abstract

This phase 2, single-center clinical trial evaluated the efficacy and safety of guadecitabine, with or without donor lymphocyte infusion, following allogeneic stem cell transplantation (allo-SCT) in adult patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). The study had three treatment cohorts based on disease status post-transplant. Cohort 1 included patients with hematological relapse after transplant (n=13). Cohort 2 consisted of patients with minimal residual disease (MRD) detected after transplant (n=18). Cohort 3 compromised of patients in remission without MRD within 100 days post-transplant (n=24). The primary objectives were to achieve morphological complete remission (CR) in cohort 1 and MRD eradication in cohort 2 within six cycles of guadecitabine. Cohort 3 patients received 12 cycles to improve relapse-free survival (RFS).

In cohort 1, 21.4% of patients achieved morphological CR. In cohort 2, 47.1% achieved MRD eradication. Those who cleared MRD had a 2-year RFS of 62.5%. Cohort 3 patients had a 2-year RFS rate was 62.5% with a median follow-up of 48 months.

No unexpected adverse events (AEs) occurred, and no graft failures were observed. Guadecitabine demonstrated efficacy and a favorable safety profile across all cohorts, supporting the investigations of hypomethylating agents.