18th Congress of EHA

Challenges facing haematologists treating acute myeloid leukaemia

Dr Dalia Mahmoud - Celgene Corporation, Summit, USA

Dr Mahmoud, thank you for joining us at the EHA meeting this year. Can you first of all describe the main challenges facing haematologists who treat patients with AML in 2013 and are there any obvious differences between the United States and the UK?

One of the key challenges is really treating patients with high risk into MDS. Now, in Europe azacitidine has been available for patients for quite some time within to high risk MDS as well as patients with 20-30% blasts. The great news there is that azacitidine has been available for quite some time to treat patients and to provide them with an overall survival advantage. In the clinical trial setting, for example, azacitidine delivered 9.4 months of incremental overall survival compared to conventional care regimens. So this has now become a standard of care, really worldwide. It has been available in the US for many years and also in Europe.

One recent change that has happened in Europe is, just this past year, the availability of Dacogen, decitabine, also it’s been just recently approved from a regulatory perspective for patients with AML as well. So now physicians have what was previously the standard of care, azacitidine, available to treat and also decitabine as an alternative as well.

Now you’ve presented two AML studies at this meeting, one to do with real world outcomes and one to do with burden of disease. Can you describe these studies and just highlight the main findings for us?

So in looking at the real world outcomes, we’ve done an exciting analysis of patients that have been treated with both products, azacitidine and decitabine, in the US since 2006 through 2013, mid-2013. This is using a US health insurance database, retrospective database analysis, and the great finding there is that it really confirms the results that we saw for azacitidine in the clinical trials setting which is, as I mentioned, that overall survival benefit. When we looked at real world data we saw, at a statistically significant level, that azacitidine had a real world survival benefit, a median of 10.1 months compared to 6.9 months for decitabine. So again this was really confirmatory of the results that we saw, as I said, in the clinical trial setting.

When you dig a little bit further into that and try to understand how does that impact the health care system in terms of resource utilisation, we actually also saw that azacitidine had an advantage over decitabine in terms of the incidence of hospitalisations that occurred on an annual basis. So these are very important endpoints or analyses to bring forward to especially the payer community and those that are very focussed nowadays on budget impact to understand that there is definitely a medical cost offset that these drugs provide to the healthcare systems.

So looking again at the second of those studies, I think you did use UK and US data in that. So, again, were there any obvious differences between the two?

I actually have a few presentations here and in that separate analysis that actually is looking at patients that are eligible for induction consolidation therapy and taking a look at once they receive induction and consolidation what are the costs that are incurred in the healthcare system thereafter in terms of they achieve a complete response but once they relapse, what are the costs that they would incur? We did see a difference between the US and UK data, we think that is just endemic to the nature of the unique healthcare systems that are in place. But what it does communicate or emphasise for us is the need for new therapies to come into this space to further increase the CR that patients experience and to delay relapse for those patients. So we really need to focus on patients that are eligible for induction consolidation therapy, what can we do to push out that relapse as long as possible?

And, finally, you’ve mentioned the need for new therapies. Do you think we’ve reached a therapeutic plateau in any sense in AML or do you think we can look forward to some real advances in the near future?

I think we can certainly look forward to some new advances, especially in the space of AML. When you think about the hypomethylating failures that are currently available and the fact that there are many patients that over time will eventually fail on those hypomethylating agents, what other therapies can we now bring into this space to further extend life for these patients? It’s something that we ask ourselves every day and we’re looking into testing new novel agents with patients to see what would be the benefit that could be derived in some of these later lines. So there is an exciting future ahead of us in terms of new agents and we just need to keep chipping away at the burden of disease that these patients face.

Dr Mahmoud, thank you very much indeed.