Abstract
Acute myeloid leukemia (AML) is a relatively rare, yet aggressive myeloid neoplasm with an incidence rate of about 4.1 new cases per 100,000 people annually in the US. The standard first-line therapeutic strategy for fit patients with AML relies on intensive chemotherapy, however many patients are ineligible for this due to comorbidities, advanced age or performance status. These patients have limited options for treatment and thus a far poorer prognosis. The objective of this study, beginning in 2019, was to conduct a retrospective chart review to collect real world outcomes and treatment patterns of patients with AML treated with the client’s drug (drugX). An online data collection portal allowed physicians at site centers in the US and Israel to randomly select one of their patients diagnosed with AML on or after April 11, 2016 and whom received a drugX-based regimen. A control patient diagnosed with AML on or after May 15, 2015 was matched 1:1 on the following criteria: line of therapy of interest, age categories (<60 years, 60-74 years, ≥75 years) and cytogenetic risk according to the European Leukemia Network (favorable, intermediate, adverse). Physicians were then able to input information from the selected patient charts until the date of last follow-up or death. Interim response rates, and Kaplan-Meier estimates of overall survival, event-free survival, and duration of response were analyzed however only results for response rates were available for this. Sample size for overall drugX-based regimen patients was 169 and 172 for overall control patients. Sample size of the unfit subgroup of drug X-based regimen patients was 112 and 114 for unfit subgroup of control patients. Higher complete composite remission (CRc) rate was achieved over the follow-up period amongst the newly diagnosed patients treated with drugX than those in the control group: 65.7% vs 53.5%. Among the unfit subgroup, a higher CRc rate was found for newly diagnosed patients treated with drugX than those treated with a non-drugX-based regimen: 66.1% vs 45.6%. The ongoing study will aim to provide evidence for physician and clinical guidelines of drugX treatment to make it as safe and effective as possible.Â