Real-World Treatment Patterns Among Patients With Relapsed or Refractory (R/R) NPM1 Mutated (NPM1m) Acute Myeloid Leukemia (AML) in the United States (US)
Author(s)
Grace Gahlon, ScM1, Shailja Vaghela, MPH2, Lorena Lopez-Gonzalez, PhD3, Craig Freyer, PharmD, BCOP3, Shelby Corman, PharmD, MS1, Huan Huang, PhD3.
1Precision AQ, Bethesda, MD, USA, 2HealthEcon Consulting Inc., Ancaster, ON, Canada, 3Syndax Pharmaceuticals, Inc., New York, NY, USA.
1Precision AQ, Bethesda, MD, USA, 2HealthEcon Consulting Inc., Ancaster, ON, Canada, 3Syndax Pharmaceuticals, Inc., New York, NY, USA.
OBJECTIVES: Approximately 30% of patients with AML have NPM1 mutations, half of whom relapse or are refractory to initial treatment. There are no US FDA-approved therapies for R/R NPM1m AML. This retrospective analysis described real-world treatment patterns among US patients with R/R NPM1m AML.
METHODS: Using electronic health records data from COTA Healthcare, patients with an initial AML diagnosis between 2009 and 2024, documented NPM1m, and who experienced a first relapse or became refractory were followed from initial diagnosis to death or loss to follow-up. R/R AML salvage regimens were classified into hierarchical categories: intensive chemotherapy (IC), low-intensity therapy (LIT), and investigational agents only. A subgroup analysis was conducted for patients with FLT3 co-mutation (FLT3com) diagnosed between 2017 and 2024.
RESULTS: Among 326 patients with R/R NPM1m AML (median age at diagnosis, 62 years; median follow-up from first R/R, 11 months), 48 (15%) did not receive salvage treatment, of whom 81% died (median time to death, 21 days). Following the first R/R, 51%, 24%, 6%, and 5% of patients received salvage therapy lines 1, 2, 3, and 4+, respectively. IC/LIT/investigational agents only use was 52%/34%/10%, 31%/47%/15%, and 32%/41%/24% in salvage lines 1, 2, and 3, respectively. Among IC recipients (all salvage lines), IC only (49%-91%) was the most common regimen, followed by IC+FLT3 inhibitor (FLT3i; 18%-30%) and IC+venetoclax (8%-20%). No clear utilization trend by salvage line was observed among LIT recipients, with LIT+venetoclax (29%-60%), LIT+FLT3i (29%-40%), and LIT only (20%-38%) being the most common regimens. In the FLT3com subgroup (86 patients), 79% received ≥1 salvage therapy, among whom 62% received FLT3i and 21% received investigational agents (alone or with another treatment).
CONCLUSIONS: These results suggest no consistent standard of care exists for patients with R/R NPM1m AML. Further research is recommended to understand the considerations for treatment decisions.
METHODS: Using electronic health records data from COTA Healthcare, patients with an initial AML diagnosis between 2009 and 2024, documented NPM1m, and who experienced a first relapse or became refractory were followed from initial diagnosis to death or loss to follow-up. R/R AML salvage regimens were classified into hierarchical categories: intensive chemotherapy (IC), low-intensity therapy (LIT), and investigational agents only. A subgroup analysis was conducted for patients with FLT3 co-mutation (FLT3com) diagnosed between 2017 and 2024.
RESULTS: Among 326 patients with R/R NPM1m AML (median age at diagnosis, 62 years; median follow-up from first R/R, 11 months), 48 (15%) did not receive salvage treatment, of whom 81% died (median time to death, 21 days). Following the first R/R, 51%, 24%, 6%, and 5% of patients received salvage therapy lines 1, 2, 3, and 4+, respectively. IC/LIT/investigational agents only use was 52%/34%/10%, 31%/47%/15%, and 32%/41%/24% in salvage lines 1, 2, and 3, respectively. Among IC recipients (all salvage lines), IC only (49%-91%) was the most common regimen, followed by IC+FLT3 inhibitor (FLT3i; 18%-30%) and IC+venetoclax (8%-20%). No clear utilization trend by salvage line was observed among LIT recipients, with LIT+venetoclax (29%-60%), LIT+FLT3i (29%-40%), and LIT only (20%-38%) being the most common regimens. In the FLT3com subgroup (86 patients), 79% received ≥1 salvage therapy, among whom 62% received FLT3i and 21% received investigational agents (alone or with another treatment).
CONCLUSIONS: These results suggest no consistent standard of care exists for patients with R/R NPM1m AML. Further research is recommended to understand the considerations for treatment decisions.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HSD97
Topic
Health Service Delivery & Process of Care
Disease
Oncology