We all know that the prognosis of patients who have myelodysplastic syndromes but are, quote-unquote, classified as lower risk is, in fact, very heterogeneous. There are many possibilities to upgrade the way we stratify the prognosis of these patients but these are very costly manners. We thought of designing very simple, almost costless, strategies to refine the prognostic assignment of these patients. We thought that cytopenias are key prognostic markers in these patients but they are always assessed as steady state variables – your haemoglobin level on a given day, say at diagnosis. So we instead looked at the pace of these cytopenias in the first six months of follow-up of lower risk MDS patients in a European registry that has included a little more than 2,000 patients. What we saw is that the velocity of platelets decrease over the first six months of follow-up is a very strong indicator of adverse outcome. Patients who lose more than 25% of their baseline platelet count over these first six months have a higher risk of progression to high risk MDS and a higher risk of death of all causes.
Based on that we designed a very simple classifier that can be used after six months of follow-up in lower risk MDS. This six month follow-up period is a relatively short follow-up for these patients and most often there is no reassessment of bone marrow parameters after such a short follow-up. So our classifier is simply based on the follow-up of cytopenias, relies on platelet decrease and persistence of red blood cell transfusion dependency and can refine the prognosis of lower risk MDS patients without the need for sophisticated biology.
Would you have future plans for it?
Obviously, for the time being, we have analysed this in a unique cohort which although it is very large and has been submitted to robust internal validation would be strengthened by external validation. So we’re in the process of checking whether our findings are relevant in an independent dataset.
From a patient’s point of view, why would they be interested in this?
They are all interested in knowing, with some degree of precision, what the future is made of. If they are elderly they may have plans according to that. From a therapeutic perspective identifying early patients who would be good candidates for disease modifying therapeutic strategies such as stem cell transplantation for the youngest of them or even hypomethylating agent therapy would be relevant.