Each year, over 20,000 Americans are diagnosed with an aggressive blood cancer called acute myeloid leukemia (AML). This cancer can start due to a mutation in isocitrate dehydrogenase (IDH), a group of proteins in your body that control how blood cells grow.
IDH mutations are seen in about 20% of patients with AML, and may portend a favorable or unfavorable prognosis depending on what other mutations are present. Treatments known as “IDH inhibitors” have been developed to treat AML by targeting these IDH mutations. However, there is a lack of data on the efficacy and safety of these treatments in patient subgroups not well-represented in clinical trials (e.g., demographic, cytogenetic, and molecular subgroups). In addition, the pivotal trials that led to approval of these treatments (AG120-C-001 and AG120-C-009) did not include a representative sample from the US population.
Project Description and Goals
This study aims to evaluate the safety and effectiveness of IDH inhibitors in routine clinical care among patients from various demographic, cytogenetic, and molecular subgroups that may not have been well represented in clinical trials. Using electronic health record (EHR) data from multiple academic and community hospitals, this study will analyze patients with AML treated with IDH inhibitors. This study will examine the association between clinical and biologic factors with patient outcomes. Additionally, this study will evaluate the safety of IDH inhibitors, including further understanding of risk of adverse events in routine clinical practice.
Anticipated Outcomes of Interest
This study aims to assemble one of the largest de-identified EHR–derived real-world databases of AML patients treated with an IDH inhibitor to date. This analysis aligns with the FDA OCE research goals to develop approaches to evaluate, integrate, and facilitate the use of oncology real-world data (RWD) to generate high-quality real-world evidence (RWE). The results may also provide important insights into enrolling patients representative of the US population into clinical trials. Finally, the database may serve as a resource to evaluate other important future questions related to AML treatment. The results of the study will be presented at scientific meetings and published in peer-reviewed journals to disseminate findings to health care professionals treating patients with AML. A report of these results will be made available to patients and other interested parties through online journals openly available to the public.
Publications
- Chenyu Lin, Ashley Woods, Zhiguo Li, Sami Leone, Emily Dworkin, Justin Kahla, Moiez Ali, Nepheli Raptis, Yasmin Abaza, Alifya Lokhandwala, Cilomar Martins de Oliveira Filho, Michelle Lee, Cristian Taborda, Jan Philipp Bewersdorf, Jordan Ritchie, Alexander Coltoff, Sean Ormond, Madelyn Burkart, Dominika Dulak, Annie Im, Mahesh Kumar, Amy Song, Sunil Iyer, Guru Subramanian Guru Murthy, Katherine Zhou, Micaela Scobie, Jessica Lewis-Gonzalez, Charles Foucar, Ehab Atallah, Eric Winer, Harry Erba, Thomas LeBlanc, E. Dianne Pulte, Joseph Wynne, Anand Patel, Talha Badar, Kelly Norsworthy; Real-world practice patterns of IDH inhibitors and outcomes of on-label IDH inhibitor use for Acute Myeloid Leukemia in the United States. Blood 2025; 146 (Supplement 1): 1614. doi: https://doi.org/10.1182/blood-2025-1614
