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Association Between Targeted Therapy and Survival in Patients With Acute Myeloid Leukemia

Precision oncology innovatively addresses cancer care by targeting treatments to patients with specific biomarker alterations. However, in the U.S., nearly one-third of eligible patients miss out on these life-saving therapies. Operational barriers, physician awareness gaps, slow test result turnaround times (TATs) and reimbursement challenges create significant obstacles, leaving many without treatments that could dramatically improve their chances of living longer. For acute myeloid leukemia (AML)—a blood cancer with a poor prognosis—these disparities are especially dire. High-risk patients face limited options, but even without complete remission, targeted therapies can extend their lives. 

In a study conducted by Pierantonio Russo, MD, Ramaa Nathan, PhD, Daniel Pfeffer, PhD, and Alex Moore, PhD, they shine a light on the transformative power of these therapies. Analyzing data from over 2,200 AML patients, their research explored the impact of targeted treatments for IDH1, IDH2, and FLT3 mutations. The results found that targeted therapies reduced mortality by up to 94% within the first month and maintained significant survival benefits over the next year. Older patients, often the most vulnerable, also experienced substantial improvements. 

However, the research also revealed a troubling statistic: 77% of AML patients with actionable mutations never received targeted therapies. Slow genetic testing processes and logistical gaps often stood in the way. Comprehensive panel tests proved more efficient, delivering faster results than single-gene tests, but systemic changes are needed to ensure equitable access. 

By addressing practice gaps, improving provider awareness and streamlining access to genetic testing, the divide between innovation and patient care can be bridged. Targeted therapies are not just treatments; they are lifelines that can turn cutting-edge science into real-world survival stories. 

Download the poster to view the full research and findings. 

Author
Pierantonio Russo, MD, FCPP, FAAP, STS
Corporate Chief Medical Officer

After leaving the Mayo Clinic, from 1988 to 2007, he held academic, clinical and administrative leadership positions as Chief of Cardiac Surgery, Pediatric Cardiac Surgery and Heart Transplantation  at several Academic Hospitals in the…

Ramaa Nathan, PhD
Vice President, Data Science and RWE Insights

Ramaa Nathan is a Director of Data Science at EVERSANA with several years of experience in the financial and healthcare industries. Ramaa is a data scientist and statistician with extensive programming, statistical and data…

Alex Moore, PhD
Data Science Manager

Alex holds a PhD in chemical engineering from University of Pennsylvania with a focus on molecular simulation of organic glasses utilizing predictive modeling techniques. At University of Pennsylvania, he gained years of hands-on experience…

Daniel Pfeffer, PhD
Senior Data Science Manager

Daniel has a background in cosmology where he developed tools to apply machine learning techniques to the field. At EVERSANA, he has worked extensively with medical insurance claims and electronic health records (EHR) on many…