MPN Workshop of the Carolinas 2025 | The importance of measuring and understanding MPN symptom burden in clinical practice and trials

What we’ve learned from MPN symptoms is one, they’re very prevalent and they’re a major driver of a challenge for the quality of life in patients with MPNs. And two, they are one key aspect of evaluating both how the patient is affected, but also response in terms of therapy. You know, I don’t think it’s the only thing, but it’s also not something to be ignored. You know, I view the whole portfolio of what the patient is experiencing: the control of the blood counts, risk of thrombosis, molecular risk for progression, burden from splenomegaly or cytopenias, and their symptom burden in having a very much individualized approach to understanding what the burden that they face. 

Now, assessing symptoms is very important, I think, both in clinical practice as well as in clinical trials. What is that at baseline? That might be modest. That might be non-existent. Some patients don’t have symptoms. They’re not universal, but they are common. Or they might be severe. And then how are they really impacting the patient? And that’s a factor in terms of deciding on therapy. Do we need to initiate therapy? And if so, do we need to adjust the dose? And if we start a therapy, is it really effective for what we’re trying to achieve? So I think it’s an important endpoint in clinical trials. I think it is nuanced in terms of what we expect for a therapy to achieve to obtain clinical benefit for a patient. 

In the end, patients can really do just one of two things as we have a medical intervention. They can live longer or they can live better. Symptoms are part of that living better part of the calculus. And indeed, there is evidence to say that changes in symptoms sometimes might even be predictors of that living longer. You know, in that, in many ways, symptoms are, as a manifestation of the disease, are just one more overt biomarker of the underlying disease with inflammation or other changes that again may, if symptoms are better, there may be less inflammation, there may be less pressure for additional somatic mutations to form that may lead to prolongation of survival. So in the end, what I want folks to take away – one, symptoms are common, we can measure them, and they’re relevant to be measured as part of our decision making, both in the conduct of clinical trials, but clearly in how we care for patients on a day-to-day basis.

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