why choose us

300×250 Ad Slot

Research Article: Ruxolitinib and decitabine plus a busulfan–cyclophosphamide conditioning regimen for relapse prophylaxis in patients with high-risk acute myeloid leukemia or myelodysplastic syndromes

Date Published: 2025-08-18

Abstract:
Relapse remains the leading cause of treatment failure in high-risk acute myeloid leukemia (AML) or myelodysplastic syndrome-IB (MDS-IB) patients after allogeneic hematopoietic stem cell transplantation (allo-HSCT). Ruxolitinib has demonstrated antileukemic activity in vitro , and decitabine has been found to be tolerable when combined with modified busulfan–cyclophosphamide (mBu/Cy) conditioning regimen. Here, we investigated the efficacy of ruxolitinib and decitabine plus a mBu/Cy conditioning regimen (Rux-Dec-mBu/Cy) in reducing relapse in high-risk AML/MDS patients ( ClinicalTrials.gov identifier: NCT04582604 ). This prospective investigational study enrolled 58 patients between May 2020 and July 2023. These patients had either a relapsed/refractory status, remission status with adverse genetic abnormalities or positive measurable residual disease (MRD+) prior to conditioning. Ruxolitinib (days –15 to –1) and decitabine (days –15 to –10) were administered, followed by mBu/Cy conditioning. The outcomes of a historical cohort of 58 patients (matched 1:1) who received mBu/Cy are described for reference. All 58 patients achieved engraftment. With a median follow-up of 967 (464–1597) days, the 2-year cumulative incidence of relapse was 19.0%. The probabilities of 2-year overall survival (OS), disease-free survival (DFS) and graft-versus-host disease-free, relapse-free survival (GRFS) were 70.3%, 70.6% and 65.2%, respectively. The cumulative incidence of grade II-IV acute graft-versus-host disease (aGVHD) was 44.1%. The most common grade ?3 adverse event was oropharyngeal mucositis (8.6%, n=5). Within 6 months post-transplantation, the cumulative incidence of cytomegalovirus (CMV) reactivation was 34.5%, and that of Epstein–Barr virus (EBV) reactivation was 62.1%. This investigational study revealed that the Rux-Dec-mBu/Cy conditioning was tolerable and reduced relapse in high-risk AML/MDS patients.

Introduction:
Relapse is the major cause of treatment failure, contributing to 40–50% of mortality rates in patients with high-risk acute myeloid leukemia (AML) or myelodysplastic syndrome-IB (MDS-IB) after allogeneic hematopoietic stem cell transplantation (allo-HSCT) ( 1 , 2 ). Relapsed/refractory status, adverse risk disease features and measurable residual disease (MRD) before allo-SCT increase the risk of relapse and are consistently associated with shortened survival ( 1 , 3 ). An increased intensity of conditioning…

Read more

300×250 Ad Slot