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Sharing advances in the treatment and management of AML

Apr 17, 2018

The 21st of April marks an important day in the AML calendar, a day when the AML community unites to mark AML World Awareness Day and raise awareness of the advances in AML and the support available to Patients, Carers & Families as well as Healthcare Professionals.

More than ever, sharing advances in acute myeloid leukemia (AML) amongst those managing the disease is becoming increasingly important. Over recent years, our knowledge and management of AML have changed significantly. The classification of AML has changed from purely focusing on morphological features, cytogenetics, and phenotypic characteristics to a greater emphasis on molecular genetic abnormalities.

 An interview on the AML Global Portal with North American steering committee co-chair, Prof. Gail Roboz during ASH 2016, explains how AML is now being subcategorized into separate diseases which all have a very different prognosis from one another.

Prof. Gail Roboz | ASH 2016 |New classification of AML

In recent years, we have seen many important breakthroughs in the definition of the AML genomic landscape. This rapid progress in identifying different types of mutations and their clinical significance has resulted in many instances in the development of specific therapies directed at such abnormalities. Both the European LeukemiaNet and the National Comprehensive Cancer Network guidelines now recommend screening patients to identify abnormalities at diagnosis as this can inform treatment decisions.

During ASH 2017, Dr. Elli Papaemmanuil shared with the AML Global Portal on how the identification of cytogenetic and molecular abnormalities to understand a patient’s risk has played a vital role in helping clinicians deliver patient-tailored decisions and improve clinical outcomes in AML. Moreover, we have seen exponential growth in identifying the genomic underpinnings in AML allowing us to further appreciate which mutations are relevant and how they come together to define distinct molecular subgroups in AML. This now means AML treatment and management is deviating from the “One-Size-Fits-All" approach.

Dr. Elli Papaemmanuil | ASH 2017 | Classification and risk assessment in AML

Recent advances in research have also identified new treatment pathways in AML including signaling pathways, epigenetic regulation of DNA and chromatin, nuclear export of proteins, as well as antigens expressed on leukemic cells.

As a consequence, we have recently seen four new drugs being approved with further approvals anticipated later this year. The standard of care induction regimen 7+3 has been improved in terms of administration and efficacy with CPX-351 (Vyxeos) for newly diagnosed (ND) tAML or AML-MRC. Identification of CD33 on leukemic cells has led to approval of a specific antibody-drug conjugate, gemtuzumab ozogamicin (Mylotarg) in ND adult CD33+ AML or relapsed/refractory (R/R) CD33+ adults and children with AML (the first drug approved for pediatric AML). The identification of two major molecular abnormalities, FLT3 and IDH2, has brought on the market two specific drugs, midostaurin (Rydapt) and enasidenib (Idhifa), for ND FLT3+ AML in combination with the 7+3 regimen and for R/R IDH2+ AML patients, respectively.

In an interview titled “New hope in AML” during ASH 2017, Gail Roboz highlighted that AML physicians are now becoming accustomed with the need to know the mutational profile of their patients at diagnosis; demonstrating real progress and the importance of sharing our advances in AML. However, all these mutations will not clear during remission and as such most AML patients present with measurable residual disease (MRD). With validation of multiparameter flow cytometry, real-time quantitative polymerase chain reaction or next-generation sequencing (NGS) to measure MRD, there is scope to harness these methods to gain further quantifiable data to help inform future post-induction treatment decisions.

Prof. Gail Roboz | ASH 2017 | New hope in AML

In conclusion: as healthcare professionals, it is becoming increasingly important to share our advances in AML as a community to ensure we are all able to keep up-to-date with the latest advances. Although the 21st April provides a great opportunity to raise awareness of the disease, we should continuously strive to increase knowledge and awareness of AML on a daily basis. Initiatives like the AML Global Portal and Know AML are helping us share our advances in AML, ensuring patients have access to the latest treatments available. To find out how you can participate in AML World Awareness Day, visit for further information.


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