Session: 906. Outcomes Research—Malignant Conditions (Myeloid Disease): Poster II
Hematology Disease Topics & Pathways:
Leukemia, AML, Diseases, Lymphoid Malignancies, Myeloid Malignancies
Methods: The AML Real world evidenCe (ARC) Initiative is a multicenter chart review study of adult pts with ND AML treated with VEN ≥ 4-11-2016 (VEN cohort) or non-VEN-based regimens ≥ 5-15-2015 (control cohort) from different academic sites in the US. Pts in the control cohort were matched to pts in the VEN cohort based on age (<60; 60-74; ≥75) and ELN risk. Interim descriptive results are presented from data as of 6-26-2020; data collection is ongoing, with a targeted sample size of over 500 pts. First and best responses to therapy, overall survival (OS), and event-free survival (EFS) were analyzed. A subgroup of pts receiving high-intensity chemotherapy regimens in the control cohort and their matched pts in the VEN cohort was analyzed separately (high-intensity therapy matched subgroup).
Results: At time of interim data analysis, 33 VEN and 33 control ND AML pts were included. Combinations in the VEN cohort included HMA (AZA [26 pts, 78.8%], DEC [6 pts, 18.2%]) or LDAC (1 pt, 3.0%); the control cohort received high- (18 pts, 54.5%) or low-intensity regimens (15 pts, 45.5%; HMA: 12 pts; enasidenib: 2 pts; LDAC: 1 pt). Median age was 75 years in both VEN and control cohorts, 39.4% and 42.4% were female, respectively, and 63.6% had de novo AML in both cohorts. The majority had ECOG performance status of 1 (VEN: 66.7%; control: 63.3%) and adverse-risk ELN classification (66.7% in both cohorts). All pts with favorable-risk in the VEN cohort (100.0%) had mutated NPM1 without FLT3-ITD or with low allelic ratio (<0.5) of FLT3-ITD, while 50.0% of pts with favorable-risk in the control cohort had inv(16)(p13.1;q22) or t(16;16)(p13.1;q22); CBFB-MYH11 (Table 1). IDH1 and IDH2 mutations were present in 9.1% and 21.2%, respectively, in the VEN cohort and 12.9% and 12.9% in the control cohort; TP53 mutation was present in 21.2% and 6.5% of the VEN and control cohort, respectively.
In the high-intensity therapy matched subgroup (VEN: n=18; control: n=18), median age was 74 and 69 in VEN versus control, respectively. The majority of pts had ECOG performance status of 1 (VEN: 76.5%; control: 75.0%) and adverse-risk ELN classification (61.1% in both cohorts). High-intensity treatment regimens used for the control group were: CPX-351 (n=10; 55.6%), 7+3 (n=4; 22.2%), 7+3 + gemtuzumab ozogamicin (n=3; 16.7%), cytarabine + mitoxantrone (n=1; 5.6%). In the VEN matched high-intensity subgroup, 15 (83.3%) received VEN + AZA, 2 (11.1%) received VEN + DEC, and 1 (5.6%) received VEN + LDAC. Ten (55.6%) initiated VEN as an inpatient treatment.
Outcomes for the full sample and subgroup are presented in Table 1. In the full sample, the overall response rate (ORR: CR+CRi+CRh) was 69.7% in the VEN cohort and 45.5% in the control cohort. The 1-year OS was 67.0% in the VEN cohort and 44.2% in the control cohort for the full sample; and 71.3% and 55.4%, respectively, in the high-intensity therapy matched subgroups.
Conclusions: The ongoing ARC Initiative demonstrates real-world treatment effectiveness of VEN combinations in clinical practice. The real-world outcomes for VEN combinations for ND AML pts appear consistent with results reported from clinical trials, though a small number of pts were assessed. Further analyses of comparisons to matched controls, both overall and in the high-intensity therapy matched subgroup, and continuing data collection will allow a more robust assessment of the comparative effectiveness of VEN combinations in the real-world setting.
Disclosures: Pollyea: Syndax: Consultancy; Syros: Consultancy; Novartis: Consultancy; Karyopharm: Consultancy; Janssen: Consultancy; 47: Consultancy, Research Funding; Genentech: Consultancy; Takeda: Consultancy; Celgene/BMS: Consultancy; Agios: Consultancy; Glycomimetics: Other; Amgen: Consultancy; Abbvie: Consultancy, Research Funding; Daiichi Sankyo: Consultancy; Pfizer: Consultancy. Talati: BMS: Honoraria; Jazz: Speakers Bureau; AbbVie: Honoraria; Pfizer: Honoraria; Astellas: Speakers Bureau. Lai: Abbvie: Consultancy; Jazz: Speakers Bureau; Astellas: Speakers Bureau; Macrogenics: Consultancy; Agios: Consultancy. Abedin: Actinium Pharmaceuticals: Research Funding; Helsinn Healthcare: Research Funding; Pfizer: Research Funding; Jazz Pharmaceuticals: Honoraria; Agios: Honoraria; Helsinn Healthcare: Honoraria. Zeidner: Sumitomo Dainippon Pharma Oncology, Inc.: Research Funding; Merck: Research Funding; Forty-Seven: Other: Travel Reimbursement, Research Funding; AROG: Research Funding; AsystBio Laboratories: Consultancy; Daiichi Sankyo: Honoraria; Takeda: Consultancy, Honoraria, Other: Travel Reimbursement, Research Funding; Pfizer: Honoraria; Genentech: Honoraria; Celgene: Consultancy, Honoraria, Research Funding; AbbVie: Honoraria, Other: Independent Review Committee; Agios: Honoraria. Xavier: Kite/Gilead: Honoraria, Membership on an entity's Board of Directors or advisory committees; AbbVie: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; AstraZeneca: Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Takeda: Honoraria, Membership on an entity's Board of Directors or advisory committees; Morphosys: Honoraria, Membership on an entity's Board of Directors or advisory committees; TG therapeutics: Honoraria, Membership on an entity's Board of Directors or advisory committees; Verastem: Honoraria, Membership on an entity's Board of Directors or advisory committees; Acrotec: Consultancy, Speakers Bureau; Biegene: Speakers Bureau; Genentech: Speakers Bureau; Seattle Genetics: Speakers Bureau; Celgene/BMS: Speakers Bureau; Epizyme: Speakers Bureau; Astellas: Speakers Bureau; Incyte: Speakers Bureau. Wolach: AbbVie: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Other: Fees for lectures and Consultancy, Research Funding; Pfizer: Consultancy, Honoraria; Janssen: Other: Fees for lectures and Consultancy; Amgen: Other: Fees for lectures and Consultancy; Astellas: Consultancy, Honoraria, Other: Fees for lectures and Consultancy; Novartis: Consultancy, Honoraria, Other: Fees for lectures and Consultancy. Moshe: Novartis: Membership on an entity's Board of Directors or advisory committees, Other: Lecturer; Astellas: Membership on an entity's Board of Directors or advisory committees, Other: lecturer; AbbVie: Membership on an entity's Board of Directors or advisory committees, Other: Fee for lec. Choi: AbbVie: Current Employment, Current equity holder in publicly-traded company. Bui: AbbVie: Current Employment, Current equity holder in publicly-traded company. Svensson: AbbVie: Current Employment, Current equity holder in publicly-traded company. Kye: AbbVie: Current Employment, Other: may hold stock or other options. Burne: Analysis Group, Inc., which has received consultancy fees from AbbVie: Current Employment. Guerin: Abbvie: Consultancy, Other; Sanofi Genzyme: Consultancy, Other: Annie Guerin is an employee of Analysis Group, Inc. which received consultancy fees from Sanofi Genzyme.; Novartis Pharmaceuticals Corporation: Consultancy, Other: Annie Guerin is an employee of Analysis Group, Inc. which received consultancy fees from Novartis.. Ma: Genentech, Inc.: Current Employment, Current equity holder in publicly-traded company. Montez: Genentech, Inc.: Current Employment; F. Hoffmann-La Roche: Current equity holder in publicly-traded company. Goldberg: Dava Oncology: Honoraria; Pfizer: Research Funding; Celularity: Research Funding; AROG: Research Funding; Aprea: Research Funding; ADC Therapeutics: Research Funding; Genentech: Consultancy, Membership on an entity's Board of Directors or advisory committees; Daiichi Sankyo: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Celgene: Consultancy; AbbVie: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Aptose: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding.
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