EHA 2019 | MDS: treating high and low-risk disease

MDS are a complex group of disease. They have very different nature, very different prognosis. Of course we need to have solutions, both for high risk and for lower risk MDS, meaning that the goal of treatment will be different for higher risk and lower risk.

For high risk MDS, that have a propensity to develop

MDS are a complex group of disease. They have very different nature, very different prognosis. Of course we need to have solutions, both for high risk and for lower risk MDS, meaning that the goal of treatment will be different for higher risk and lower risk.

For high risk MDS, that have a propensity to develop leukemia have cytopenias. We need to eradicate the disease possibly and block progression to AML. During this meeting in EHA, there were not so many new data regarding high risk MDS, the standard and the golden treatment, the golden starter treatment is still hypomethylating agents. There have been several attempts to add other agents, but I must say that not many of the attempts have been successful. So we stand in a sort of limbo now, because this patient do respond to hypomethylating agents, but this response has a duration that is not always extremely long, and after that we have to change therapy.

Now there are new hypomethylating agents proposed, like oral decitabine that is going to be studied in AML and MDS, high risk MDS. There is a phase three study, that is going to be finished soon, with the guadecitabine. That is the long acting decitabine, and is a randomized study, with the standard of care which is the choice of the physician. We don’t know the results yet, but we are still walking in the same path, let’s say, with hypomethylating agents.

There are the drugs, like Enasidenib, Ivosidenib, that are mainly now approved for AML, but not for MDS, and probably they will be very useful for a subset of MDS as well. Here at EHA, there have been data presented regarding the association of hypomethylating agents and an oral polo 3-kinase inhibitor, which is Rigosertib. Data are interesting, with a high overall response rate, but there is also a toxicity to add to the efficacy. So I think we have a long way to go in this very moment for high risk MDS in comparison, especially to AML, for which we have had very many drugs approved in the last year. And therefore, I think there is a lot of work, and a lot of trials are going on, so we are looking forward to seeing the results.