Guadecitabine Improved Relapse-Free Survival in High-Risk Acute Myeloid Leukemia and Myelodysplastic Syndrome Patients After Transplant: Phase II Results From a Single Center

Authors

Betül Oran
Peter Thall
Amin Alousi
Gheath Al-Atrash
Rohtesh Mehta
David Marin
Partow Kebriaei
Uday Popat
Roland Bassett
Qaiser Bashir
Jin Im
Amanda Olson
Jessica Jewell
Portia Smallbone
Elizabeth Shpall
Richard Champlin

Language

English

Publication Date

1-1-2026

Journal

Haematologica

DOI

10.3324/haematol.2025.287628

PMID

40702903

PMCID

PMC12775770

PubMedCentral® Posted Date

7-24-2025

PubMedCentral® Full Text Version

Post-print

Abstract

This phase II, single-center clinical trial evaluated the efficacy and safety of guadecitabine, with or without donor lymphocyte infusion, following allogeneic stem cell transplantation in adult patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). The study had three treatment cohorts based on post-transplant disease status. Cohort 1 included patients with hematologic relapse after transplant (N=13). Cohort 2 consisted of patients with minimal residual disease (MRD) detected after transplant (N=18). Cohort 3 compromised patients in remission without MRD within 100 days after transplant (N=24). The primary objectives were achievement of morphological complete remission 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 complete remission. 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 of 62.5% with a median follow-up of 48 months. No unexpected adverse events occurred and no graft failures were observed. Guadecitabine demonstrated efficacy and a favorable safety profile across all cohorts, supporting the investigations of hypomethylating agents.

Keywords

Humans, Myelodysplastic Syndromes, Leukemia, Myeloid, Acute, Middle Aged, Male, Female, Adult, Hematopoietic Stem Cell Transplantation, Aged, Azacitidine, Young Adult, Treatment Outcome, Transplantation, Homologous, Antimetabolites, Antineoplastic, Neoplasm, Residual

Published Open-Access

yes

Recommended Citation

Oran, Betül; Thall, Peter; Alousi, Amin; et al., "Guadecitabine Improved Relapse-Free Survival in High-Risk Acute Myeloid Leukemia and Myelodysplastic Syndrome Patients After Transplant: Phase II Results From a Single Center" (2026). Faculty, Staff and Student Publications. 5251.
https://digitalcommons.library.tmc.edu/uthgsbs_docs/5251