Menu Back to Poster-Presentations-Details

SP02-31: Evaluation of Scientific Evidence and Implementation Challenges to Precision Medicine for Acute Myeloid Leukemia Patients

Poster Presenter

      Yejin Lee

      • Rutgers University- Ernest Mario School of Pharmacy
        United States


Evaluating the growing body of scientific evidence and identifying possible steps and solutions necessary to implement the use of targeted cancer therapy as an acceptable alternative or a complement to the current standard of care in adult Acute Myeloid Leukemia (AML) patients.


A literature review was performed in PubMed, The Cochrane Library and various grey literature. We analyzed current and investigational targeted therapies for AML. Then various implementation plans were compared to formulate possible changes to current guidelines and to address key challenges.


The most frequently altered genes in AML are DNMT3A, NPM1 and on. Of the list, FLT3, IDH1, and IDH2 are the most utilized predictive biomarkers for AML treatment and have shown general positive effects in improving the standard of care when coupled with corresponding therapies. There are 4 U.S. Food and Drug Administration (FDA) approved companion diagnostic coupled therapies and 1 additional therapy according to the National Comprehensive Cancer Network guideline. These medications include enasidenib, gilteritinib, ivosidenib, midostaurin, and sorafenib. Gemtuzumab has been FDA approved for some AML patients as well. Completed and ongoing clinical trials in various areas such as CAR-T, molecular diagnostics, and monoclonal antibodies were interpreted to depict the rapid progression of precision medicine (PM) in the treatment of AML. After reviewing the current implementers of PM, common steps were identified as important catalysts in successfully implementing PM in real-world practice. The initiation steps include creating a molecular tumor board. Institutional support must be gained through utility-based evidence collection and dissemination. Then, proper genetic test ordering and interpretation processes should be established. Reimbursement sources or processes for testing and counseling should be instilled. Once this has been completed, the integration of genetic data into electronic health records should be completed by informatics. Patient and provider education programs should be created and the implementation workflow must be organized before the start of implementation. We found organizations offering various resources and guidance for future implementations for previously noted challenges. Some of the suggestions addressing frequently faced challenges list: administrative support for prior authorization and clinical trial matching, prior coordination with payers and labs, and better technical support to facilitate utilization and navigation.


Precision medicine is gradually becoming more widely used for patients with AML. Many implementers’ collaborations and clinical data demonstrate the growing effectiveness of targeted cancer therapy in treating cancer patients. There has been promising evidence in the improvement of various outcomes endpoints, such as overall survival and progression-free survival. However, there still remains additional barriers to maximize its benefit in a clinical setting. Some of these challenges include physician comfort in prescribing diagnostics and interpreting the results for the individualized treatment plans. With the complexity in adding the targeted aspect of treating AML patients, physicians and patients may undervalue the benefits. In addition to these complexities, cost and reimbursements remain a key challenge. More real-world evidence is necessary to justify targeted therapy as front-line therapy in AML.