denotes an abstract that is clinically relevant.
denotes that this is a recommended PHD Trainee Session.
denotes that this is a ticketed session.Session: 908. Outcomes Research: Myeloid Malignancies: Poster I
Hematology Disease Topics & Pathways:
Clinical Practice (Health Services and Quality), Therapy sequence, Treatment Considerations, Measurable Residual Disease
Methods: This was a retrospective study of AML patients who were treated at the Moffitt Cancer Center between 9/2020 and 6/2024 who had received Oral-AZA after achieving CR/CRi. Patients who started Oral-AZA after alloHCT were excluded. Descriptive statistics were performed. Kaplan-Meier analysis with log-rank test was used to estimate RFS and OS from the time of Oral-AZA initiation.
Results: There were 65 patients (39M/25F) identified with a median age of 65 (range: 21-75) at Oral-AZA initiation. Majority of the patients were diagnosed with de novo AML (78%). According to 2022 European LeukemiaNet (ELN) classification, 30 (46.2%), 14 (21.5%), and 21 (32.3%) had favorable, intermediate, and adverse risk disease, respectively. Frequently mutated genes included NPM1 (44.6%), FLT-ITD/TKD (27.7%), NRAS (24.6%), and IDH1/2 (13.8%). Prior to Oral-AZA initiation, 72.3% had 1 line of therapy (range: 1-3). Over 90% of patients received cytarabine-based induction. A total of 90.8% (59/65) received consolidation with a median of 2 cycles. The median time to Oral-AZA initiation from induction or consolidation chemotherapy was 63 days.
Median duration of Oral-AZA was 4 cycles and 16.9% remained on the drug for ≥12 months. Notably, 17 out of 65 patients (26.1%) in our cohort received alloHCT after Oral-AZA maintenance (median 3 cycles). Median follow-up was 15.5 months and OS from Oral-AZA initiation was not reached with OS rates of 93.3% (95% CI: 83.1-97.4) and 84.8% (95% CI: 71.6-92.2) at 6 and 12 months, respectively. Median RFS was 17.9 months with 6- and 12-month RFS rates of 79.4% (95% CI: 66.5-87.7) and 63.6% (95% CI: 48.6-75.3), respectively.
When patients who ended up receiving alloHCT after Oral-AZA were excluded (n=48), median OS was also not reached and OS rate at 12 months was 82.3% (95% CI: 66.1–91.2 %). In the subset of patients who had NPM1 at diagnosis (n=29), percent survival at 12 months was 95.2% (95% CI: 70.7-99.3 %). The median RFS in NPM1 positive patients who did not receive alloHCT was 26.9 months with RFS rates of 80.0% (95% CI: 49.98-93.07) and 53.3% (95% CI: 23.39 - 76.19) at 6 and 12 months respectively. There were 17 patients age < 55 and their survival was 100% at 12 months and 85.7% at 24 months.
Measurable residual disease (MRD) by flow cytometry was available for 22/65 (33.8%) patients prior to Oral-AZA initiation, and 11/22 (50%) of these patients had subsequent flow MRD assessments. All patients remained MRD negative by flow while on Oral-AZA. Four of the 22 patients did eventually relapse.
Conclusions: Our results affirm the survival benefits observed with the use of Oral-AZA maintenance in the QUAZAR AML-001 study in the real-world setting. The outcome of those who harbor NPM1 is especially favorable. In the subset of patients who ended up receiving alloHCT, maintenance therapy with Oral-AZA was used as bridging therapy for relapse prevention prior to transplant. The benefit of this strategy will need to be confirmed with larger dataset and be validated prospectively.
Disclosures: Chan: Abbvie: Honoraria, Research Funding; Syndax: Membership on an entity's Board of Directors or advisory committees; Jazz: Research Funding; Novartis: Honoraria; Aptitude Health: Honoraria; BMS: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees. Tobon: Eli Lilly: Consultancy, Honoraria, Other: Consultant/Advisory Board; Abbvie: Consultancy, Honoraria, Other: Consultant/Advisory Board; Bristol Myers Squibb: Consultancy, Honoraria, Other: Completed non-CE panel discussion. Lancet: Prelude Therapeutics: Consultancy, Other: Bristol Myers Squibb; Bristol Myers Squibb: Consultancy, Other: Consultant/Advisory Board; Tradewell Therapeutics: Consultancy, Other: Consultant/Advisory Board. Sallman: Abbvie: Consultancy; Agios: Consultancy; Axiom: Consultancy; Gilead: Consultancy; Celyad: Consultancy; Froghorn: Consultancy; Incyte: Consultancy; Intellisphere, LLC: Consultancy; Johnson & Johnson: Consultancy; Kite: Consultancy, Membership on an entity's Board of Directors or advisory committees; Magenta Therapeutics: Consultancy, Membership on an entity's Board of Directors or advisory committees; NextTech: Consultancy; Novartis: Consultancy, Membership on an entity's Board of Directors or advisory committees; AvenCell: Membership on an entity's Board of Directors or advisory committees; Astellas: Membership on an entity's Board of Directors or advisory committees; BlueBird Bio: Membership on an entity's Board of Directors or advisory committees; BMS: Membership on an entity's Board of Directors or advisory committees; Dark Blue Therapeutics: Membership on an entity's Board of Directors or advisory committees; Intellia: Membership on an entity's Board of Directors or advisory committees; Jasper Therapeutics: Membership on an entity's Board of Directors or advisory committees; NKARTA: Membership on an entity's Board of Directors or advisory committees; Orbital Therapeutics: Membership on an entity's Board of Directors or advisory committees; Rigel Pharmaceuticals: Membership on an entity's Board of Directors or advisory committees; Shattuck Labs: Membership on an entity's Board of Directors or advisory committees; Servier: Membership on an entity's Board of Directors or advisory committees; Syndax: Membership on an entity's Board of Directors or advisory committees; Syros: Membership on an entity's Board of Directors or advisory committees; Apera: Research Funding; Jazz: Research Funding. Komrokji: CTI biopharma: Membership on an entity's Board of Directors or advisory committees; Servio: Membership on an entity's Board of Directors or advisory committees; BMS: Research Funding; Servier: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; AbbVie: Consultancy, Membership on an entity's Board of Directors or advisory committees; Celgene/BMS: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Geron: Consultancy, Membership on an entity's Board of Directors or advisory committees; Jazz Pharmaceuticals: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; PharmaEssentia: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Rigel: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Sobi: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Sumitomo Pharma: Consultancy, Membership on an entity's Board of Directors or advisory committees; DSI: Consultancy, Membership on an entity's Board of Directors or advisory committees; Genentech: Consultancy; Keros: Membership on an entity's Board of Directors or advisory committees; Janssen: Consultancy; BMS: Honoraria, Membership on an entity's Board of Directors or advisory committees; DSI: Honoraria, Membership on an entity's Board of Directors or advisory committees; Servio: Honoraria; Taiho: Membership on an entity's Board of Directors or advisory committees; Novartis: Membership on an entity's Board of Directors or advisory committees. Kuykendall: Novartis: Research Funding; Protagonist Therapeutics: Honoraria, Research Funding; PharmaEssentia: Honoraria; Incyte: Honoraria.
OffLabel Disclosure: Off-Label use of Oral Azacitidine for maintenance in AML after first remission in HSCT eligible patients. This was used as a bridge to transplant for relapse prevention.
See more of: Oral and Poster Abstracts