General AML

AML World Awareness Day 2019 | Latest updates from clinical trials

Acute myeloid leukemia (AML) World Awareness Day, on 21 April 2019, marks an important event in the AML calendar allowing the community to unite, share knowledge, and raise awareness of the advances in the prevention, management, and treatment of AML.

Clinical trials provide a monitored and controlled environment to test novel agents for the treatment of disease. The aim of clinical trials vary, but can include assessment of efficacy and safety, and a comparison to the standard-of-care (SoC) regimen.1 Patients with AML may receive novel therapeutic regimens in a clinical trial setting, which are otherwise unavailable clinically. The results from clinical trials provide important data to assess treatment strategies for the effective prevention, management, and/or treatment of patients with AML.1

At the 60th American Society of Hematology (ASH) Annual Meeting in 2018, Ellen Ritchie discussed the accrual of patients to clinical trials in the United States:

The analysis of clinical trial results is critical to allow for advancements to patient care and continual improvements in treatment pathways.

At the ASH 2018, Andreas Burchert discussed the findings of the SORMAIN trial, which evaluated the use of sorafenib, a tyrosine kinase inhibitor, as a maintenance therapy in patients with FLT3-ITD positive AML following allogeneic stem cell transplantation (allo-SCT):

In addition to this, the primary results from the phase II RADIUS study, which examined the role of midostaurin in combination with SoC therapy following allo-SCT, was discussed by Richard Maziarz:

The use of combination therapies have shown growing importance in the trend of recent clinical trial results. The use of venetoclax, an oral selective inhibitor of BCL-2, has shown promising therapeutic potential in a number of studies, including in combination with chemotherapy (CAVEAT study), with low-dose cytarabine (NCT02287233), and with decitabine or azacitidine (NCT02203773).

Moreover, selective FLT3 inhibitors are being extensively tested in the clinical trial setting. The final analysis from the QuANTUM-R study, which assessed quizartinib, an oral, potent and selective FLT3 inhibitor, demonstrated a survival benefit compared to salvage chemotherapy for the treatment of patients with relapsed/refractory FLT3-ITD mutant AML. Keith Pratz discussed the results from a phase I trial examining gilteritinib, a potent FLT3/AXL inhibitor, in combination with intensive chemotherapy as a treatment approach for patients with newly diagnosed AML:

In patients with FLT3-mutated AML, the use of midostaurin, a multi-targeted tyrosine kinase inhibitor, in combination with chemotherapy was analyzed in the RATIFY trial, with the results demonstrating the negative prognostic impact of insertion site mutations.

Furthermore, results from the Beat AML Master trial was presented by Eytan Stein at ASH 2018, which assessed the use of enasidenib, an oral inhibitor of IDH2, for the treatment of newly diagnosed patients with IDH2-mutant AML:

The use of gemtuzumab ozogamicin (GO) continues to be explored in clinical trials, with the results of the prospective AMLSG 09–09 trial presented at ASH 2018. The study concluded that the combination of GO and intensive induction therapy in patients with NPM1-mutated AML was associated with a higher death rate. This demonstrates the vital nature of exploring new treatment approaches in the clinical trial setting.

In conclusion, these developments in clinical trials have demonstrated the importance of sharing knowledge within the AML community in order to continually improve understanding and provide patients with the latest available treatment options. Initiatives such as the AML Global Portal and Know AML are helping to share advances in AML amongst the community. To find out how you can participate in AML World Awareness Day, visit for further information.

  1. Zwierzyna M. et al. Clinical trial design and dissemination: comprehensive analysis of and PubMed data since 2005. BMJ. 2018 Jun 6; 361: k2130. DOI: 10.1136/bmj.k2130
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