Overview of Myelodysplastic Syndrome and the Importance of Biomarker Testing

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EP: 1.Overview of Myelodysplastic Syndrome and the Importance of Biomarker Testing

EP: 2.RARA Gene Expression in Higher-Risk MDS

EP: 3.A Potential Role for Tamibarotene in the Treatment of Higher-Risk MDS

EP: 4.SELECT-MDS-1: Review of Study Design and Objectives

EP: 5.Future Perspectives in Higher-Risk MDS

Gustavo Rivero, MD: I am Gustavo Rivero. I am an associate professor in hematology-oncology at Baylor College of Medicine [Houston, Texas]. I concentrate my efforts on treating acute myelogenous leukemia and myelodysplastic syndrome [MDS].

I want to emphasize that myelodysplastic syndrome is a very unique entity. It's mainly classified as an apoptotic-like disease and being more resistant to apoptosis. It is a spectrum of disorders that we normally can distinguish by classifying the entity via the Revised International Prognostic Scoring System. There are some items that we should keep in mind for the risk stratification, which include the depth of anemia, thrombocytopenia, neutropenia, the number of blasts, and specifically the cytogenetic abnormalities that the patient might have at disease initiation. So the risk stratification allows a subcategorization for these entities into a low-risk biology and a high-risk biology. Points are assigned in an increasing mode; the higher the score, the more likely the patient will have high-risk disease vs with a low score, the patient will be categorized as having a low-risk disease. This is normally what we do in clinical practice every day.

Biomarker testing is becoming extremely relevant for acute myelogenous leukemia and myelodysplastic syndrome. There is an increasing necessity for detecting what type of drugs might be implemented in subcategories of patients with MDS. Specifically, we have retained a very agnostic therapy, which is an intravenous hypomethylating agent. Hypomethylating agents are azacitidine and decitabine, which are medications that have been associated with an overall response rate of 45%. However, there is a group of patients who won’t respond to the intervention, and these subgroups of patients are categorized as primary refractory. Almost every patient responding to the medication will develop secondary failure.

So biomarker testing is especially important because it will select the patients who might respond to normal interventions. Among them, we have recent evidence that subcategorization for MDS is grouped into monocytic-like and myeloid-like. These MDS groups might reveal a path for incorporation of novel therapies like RARA inhibition, and even a subgroup of patients in the high-risk setting who might respond better to BCL2 inhibition.

Transcript edited for clarity.