Session: 732. Allogeneic Transplantation: Disease Response and Comparative Treatment Studies: Poster II
Hematology Disease Topics & Pathways:
Research, Acute Myeloid Malignancies, AML, Combination therapy, Clinical Research, Diseases, Therapy sequence, Real-world evidence, Treatment Considerations, Registries, Myeloid Malignancies
In this study, we aimed to evaluate the safety and efficacy of ASCT in AML patients treated with AZA/VEN in both ND and R/R settings.
We conducted a multicenter retrospective cohort study using data from the VENAURA registry, which includes 585 AML patients from 12 centers in the Auvergne Rhône Alpes (AURA) region of France treated with AZA/VEN between 2019 and 2024.
This study analyzed 80 AML patients who underwent ASCT after receiving AZA/VEN in either ND (25; 31%) or R/R (55; 69%) settings. The cohort included 40 patients (52%) with de novo AML, 13 (17%) with secondary AML, 10 (13%) with AML with Myelodysplasia-Related Changes (AML-MRC), and 14 (18%) with MDS/AML. The median age was 57 years (y) (range 18-73). Besides age (61 y vs. 56 y, p=0.048), there were no significant differences between the ND and R/R groups in other clinical characteristics, molecular features, or ELN 2022 risk groups.
No significant differences were observed between the groups for median number of AZA/VEN cycles before HSCT (2 cycles, range 1-6, p=1), median time from AZA/VEN initiation to ASCT (3 months, range 1-14, p=0.85), pre-ASCT response (complete remission (CR) in 61 patients, 76%; CR with incomplete hematologic recovery (CRi) in 7 patients, 9%; morphologic leukemia-free state (MLFS) in 7 patients, 9%; no response (NR) in 5 patients, 6%, p=0.75), or minimal residual disease (MRD) status (36 MRD-negative, 60%; 24 MRD-positive, 40%, p=0.58).
With a median follow-up period of 13 months (IQR 11-16), no significant differences were observed between ND and R/R groups regarding 1-year overall survival (72 vs 79%, p=0.34), non-relapse mortality at 1 year (4.3 vs 6.2%, p=0.86), or relapse rate at 1 year (21% vs 25%, p=0.91).
No significant differences were observed between groups regarding acute graft-versus-host disease (GVHD) (28% overall, 37% ND, 23% R/R, p=0.12), severe acute GVHD (9.6% overall, 8.1% ND, 10% R/R, p=0.98), or chronic GVHD incidence at 1 year (22% overall, 18% ND, 23% R/R, p=0.97).
In multivariate overall survival analysis, factors significantly associated with worse survival were age (HR 1.08 per y, 95% CI: 1.00-1.15, p=0.043), higher HCT-CI score (≥3) (HR 8.69, 95% CI: 2.27-33.3, p=0.002) and ELN 2022 adverse risk classification (HR 12.5, 95% CI: 1.52-103, p=0.019).
Concerning cumulative incidence of relapse, MRD positivity pre-ASCT remained significant (HR 4.53, 95% CI: 1.34-15.3, p=0.015), and donor type (matched vs mismatched) was nearly significant (HR 3.03, 95% CI: 0.99-9.35, p=0.053) in multivariate analysis.
In conclusion, our data suggest that ASCT for AZA/VEN treated AML patients is feasible, with promising transplant outcomes and an encouraging post-ASCT treatment-related mortality. Additionally, it is important to note that our results, collected in a real-world setting, involve older patients with inherently poor leukemia characteristics and a high number of mismatched donors. To establish the benefit of AZA/VEN pre-ASCT, a matching comparison with a CIBMTR historical control cohort treated with conventional IC will be presented during the meeting.
Disclosures: Tavernier: Pfizer: Other; BMS: Honoraria. Contejean: Novartis: Honoraria; Pfizer: Honoraria; Abbvie: Honoraria; BMS: Honoraria; Janssen: Honoraria. Santana: Abbvie: Other; BMS: Other; Astrazeneca: Other. Rocher: ASTRAZENECA: Consultancy; BRISTOL MYERS SQUIBB: Research Funding; Pierre OUDOT hospital: Current Employment. Lamure: Janssen: Other, Research Funding; Gilead: Other; Roche Pharma: Other; Abbvie: Other; Sanofi: Other; Novartis: Other; Actelion: Other; Pfizer: Other. Heiblig: Servier: Honoraria; Jazz pharmaceutical: Honoraria; Abbvie: Honoraria; Pfizer: Honoraria; Astellas: Honoraria. Aspas Requena: Janssen: Honoraria; BMS: Honoraria; Abbvie: Honoraria; NOVARTIS: Honoraria; Servier: Honoraria.