MPN Workshop of the Carolinas 2024 | The potential use of molecular response as a surrogate marker for survival outcomes in MPNs

In the panel discussion, I think there’s a number of important questions that came up. One of them is molecular response and what it really means. I think there’s more and more data now to support the the idea that molecular response could be a good surrogate for event-free survival. Could it be a surrogate for overall survival? We don’t really know that yet, but the MAJIC-PV study has really shown, for instance, that not ropeg, but ruxolitinib, achieved molecular responses and achieved event-free survival advantage, and when you looked specifically at the cohort of patients who achieved a molecular response, those patients also had the better or the higher event-free survival. So there is now clinical trial evidence that molecular response really correlates to event-free survival.

I would say that the CONTINUATION PV study showed similar findings in that the patients who primarily were ropeginterferon-treated had a molecular response, which translated to a better event-free survival. So I think it’s an important endpoint to look at. It’s an important surrogate or biomarker for survival to look at. It is probably not the best out there. I’m sure that one day we will discover a better biomarker for survival, but so far it’s, you know, it’s what we have and there is some correlation there that might take a couple of years to appear. So it’s not necessarily an early predictor or it’s not necessarily a very reliable predictor, but there’s a general association there that I think we can capitalize on when running clinical trials that are powered ultimately to study survival as a primary endpoint.