Clinical Insights on the Treatment of Patients with High-Risk MDS

EP: 1.An Introduction to MDS: Initial Diagnosis and Patient Risk Stratification

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EP: 2.Clinical Insights on the Treatment of Patients with High-Risk MDS

EP: 3.Targeting CD47 in MDS: Reviewing the CD47 Pathway and Key Trial Data

EP: 4.The Future of Treatment for Patients with MDS

Dr. Naval Daver: In our practice at MD Anderson, we see a very large volume closer to around 35400 MDS patients a year. About 50% to 60% of these present in what we call the higher risk group. This includes intermediate, high, very high risk scores from the IPSSR. So this is kind of the presentation for the majority of our patients coming to us.

For a newly diagnosed patient with higher risk MDS, which includes usually very high intermediate risk IPSSR risk groups, we usually are looking at different therapeutic modalities. The most commonly used are what we call hypomethylating agents or HMA. This includes two different drugs. One is Azacitidine, the other is Decitabine. These have been used in frontline treatment of MDS for the last two decades. They give about a 30% to 35% overall response rate and about a 15% to 20%, CR rate. There are certain patients where we may in fact consider using intensive cytarabine based induction chemotherapy in the first line setting and not hypomethylating agents. These are usually younger patients below 65 years of age, or those who have mutations such as NPM1, IDH1, IDH2, MLL, that could be sensitive to intensive induction chemotherapy, and they could have a very high response rate. The next big question is whether you use upfront hypomethylating agent-based therapy, either single agent or various combinations that are being looked at in clinical trials at this time, or you use cytarabine based intensive induction is whether this patient will then need to go into allergenic stem cell transplant. And in most of our patients who have higher risk disease, those who are below 73, 74 years of age, we are routinely considering allergenic stem cell transplant in the first remission after giving them three to five cycles of hypomethylating agent or intensive cytarabine based treatment.