Acute myeloid leukemia (AML) is the most common acute leukemia in adults. The incidence rate is rising each year. Key factors include the aging of the population and the fact that AML can be linked to chemotherapy for other cancers. The conventional treatment approach for a younger patient with newly diagnosed AML is induction chemotherapy followed by consolidation or intensification treatment. For older patients, there is no standard of care. There is also currently no standard of care for relapsed AML. Younger patients can re-attempt induction chemotherapy regiments or go into a clinical trial. Due to high unmet patient needs, multiple forms of novel therapy are currently in clinical trials.
In patients diagnosed with AML, molecular biomarkers are used for prognosis and, as a companion diagnostic for approved targeted therapies, to predict drug response. Well-known chromosomal abnormalities may be identified in approximately 50% of all AML patients. FLT3 alterations were first identified as the most common oncogenic driver as well as a strong poor prognostic factor for long-term survival in AML. These have since been the leading drug target in this indication. Leveraging the IQVIA CORE™ (Figure 1), IQVIA applies a unique combination of data, machine learning and domain expertise to produce more predictable results and to offer innovation to the AML community. This approach has four elements: domain expertise, transformative technology, unparalleled data and advanced analytics.
CORE-enabled clinical development brings realworld and commercial data insights into the clinicaldevelopment and planning process for novel drugs targeting AML, facilitating identification of potential alternative paths to shorten timelines, decrease costs and maximize asset value.
Authored by an IQVIA team with expertise across translational medicine, clinical trials and data science, this paper provides a comprehensive review of AML disease pathology and biomarkers, and highlights how the CORE-enabled approach can support the successful delivery of trials in this indication.