For patients with lower risk MDS there’s really no current therapy that improves survival and current therapies are focussed on improvement in quality of life. There are several options – lenalidomide is used, erythropoiesis stimulating agents, transfusions, sometimes even hypomethylating agents, although they’re mainly more for the higher risk patients. I would say for immunosuppressive therapies that are a more rarely used form of therapy and often only examined in smaller studies. So the goal of our study was to look at a bigger patient cohort, combining data from multiple centres in the United States and in Europe and look at outcomes as well as predictors of the therapy.
Were there any markers that you found to be particularly predictive?
Yes, we looked at a bunch of clinical as well as molecular markers that have been classically reported with predicting a response to immunosuppressive therapy including age, sex, prior transfusions, bone marrow cellularity as well as molecular markers like HLA-DR15 positivity, PNH clones etc. What we detected is that the one marker that panned out in univariate and multivariate analyses was a hypocellular bone marrow, so a bone marrow cellularity less than 20% predicted a better response to therapy. While the other markers that were classically predicted like HLA-DR15 positivity, prior transfusion dependence, as well as some mutations we looked at, in particular SF3B1, did not predict response in multivariate analysis.
What did that mean for overall survival?
Overall survival for patients, keeping in mind those were all lower risk MDS patients, but for patients who received transfusion independence mean overall survival was not reached. So it was relatively good. We looked at predictors of overall survival too and the classic predictors panned out like a high IPSS score and more than 5% blasts being associated with a worse overall survival. But no other predictors predicted overall survival, including bone marrow cellularity did not predict overall survival.