Tag Archive for: abnormal genes

How Are MPN Treatments Changing for Low-Risk vs High-Risk Patients?

How Are MPN Treatments Changing for Low-Risk vs High-Risk Patients? from Patient Empowerment Network on Vimeo.

How do MPN treatments vary for low-risk and high-risk patients? MPN expert Dr. Claire Harrison from Guy’s and St. Thomas’ Hospital in London explains how treatment differs for these patients and changes that she would like to see for care of some patients. 

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Transcript:

Dr. Nicole Rochester: 

How are treatment strategies changing for low-risk and high-risk patients with MPN?

Dr. Claire Harrison: 

It’s complicated because we need to think across the entities, and we don’t have an answer to that for patients with MPN unclassified. And we don’t actually have a good answer to that for this entity called pre-fibrotic myelofibrosis, which does appear and is strongly recognized in the new diagnostic criteria. But for ET, for example, low-risk patients I mentioned triple-negative, calreticulin, M-positive, young patients, platelets less than 1500, not too much changing their queries about aspirin or not, and then for PV patients, we haven’t really changed all kind of high-risk criteria and for both ET and PV, the questionnaire is, should we use the treatment above aspirin or above aspirin and venesection. 

And for the most part, that would be hydroxycarbamide, hydroxyurea (Hydrea), which is the commonest treatment used worldwide or interferon, and these are the right treatment for some patients and not the right treatment for other patients, so some patients can be very fixated on interferon is the absolute best, but there is no clear evidence of that, and there are some patients who interferon is not the right treatment, but low versus high risk becomes even more important for myelofibrosis patients.

And here, we’re thinking about using a risky strategy like transplantation for those patients who have higher risk disease, and we’re using, as I mentioned to you, these molecular markers and newer prognostic tools to stratify patients. And it is important to remember as a patient if someone puts your data into a prognostic tool and that comes up with five years, but it doesn’t mean to say five years on the dot your time’s up, that’s an average. And if we put your data into a slightly different tool, we might get something else. So for the most part, we make decisions like transplants, we are learning more about transplantation and outcomes from that, and then in some countries, some treatments are used for patients who fall into intermediate or high-risk categories, and some clinical trials are based on that as well. I would want to say about myelofibrosis, and something I think I would really like to see changed, not changing yet, but changed, is that we should be able to intervene for patients with a low-risk disease. If my myelofibrosis patients have breast cancer, we would not be going there, then you’ve got low-risk disease, we’ll put you on watch and wait, watch and wait is really hard for our patients, we know that I can see you nodding.

You know that too, right? So if these were patients with breast cancer we would not say, we’ll just watch and wait. So I would really like to see in the next five to 10 years a treatment that we could use earlier in the disease course, but there is nothing at the moment, but we’re looking at that. The other thing we’re looking at, if we’ve got a minute or so is the different endpoint, so we’re trying to understand what does it mean if your allele burden, so the amount of abnormal genes you’ve got goes down, the amount of bone marrow fibrosis you’ve got goes down. And again, this is something we’ll collect in a clinical trial, but also from real-world data.