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New Data Challenge Traditional Treatment Paradigm in MPNs, Says Dr Raajit Rampal

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Traditionally, low-risk patients with myeloproliferative neoplasms (MPNs) are not given medication, but new data challenge that thinking, said Raajit Rampal, MD, PhD, hematologic oncologist, associate attending physician, Memorial Sloan Kettering Cancer Center.

New data challenge the traditional thinking that low-risk patients with myeloproliferative neoplasms are largely just treated with phlebotomy and aspirin and have shown the benefits of medication, such as ropeginterferon, said Raajit Rampal, MD, PhD, hematologic oncologist, associate attending physician, Memorial Sloan Kettering Cancer Center.

Transcript

Interferons have been around for decades, but what unanswered questions remain about their use?

I think it really is about: when to start, who starts, and for how long should they be treated? Those, to me, are kind of key questions. There's relatively recent data that looked at treating patients with polycythemia vera, who are low risk with ropeginterferon vs what we traditionally do, which is to use things like phlebotomy and aspirin. There at least seems to be some signal to suggest that those patients may derive a benefit. Our traditional thinking is we leave the patients alone except for phlebotomy and aspirin, and if they have a blood clot or symptoms or something, maybe we put them on medication. If not, we only treat them if they're high risk. But this data was actually provocative in the sense that it said, “Well, if you take these low-risk patients, there may be some clinical benefits to them by starting early.”

It raises the question, which I think is not a results question: Should we be treating patients earlier in their disease course with drugs like interferon that may have the ability to deplete some of the stem cells causing the disease, but also to put patients in a position where their disease is well controlled clinically? I think that's got to be resolved.

There is data that has emerged from France about discontinuing interferon after a number of years of therapy. So, is that something that should be explored as part of a paradigm shift? Or you can say, “Listen, we treat patients for a limited time; if the disease is under control, maybe we can stop it and monitor them and retreat as needed?”

I mean, that would be a complete change from the current paradigm of how we treat. These I think, are broadly unanswered questions that should be explored.

How do you determine strong enough disease control that warrants treatment discontinuation?

We're not there yet. The study that has been published was retrospective in nature and essentially looked at how durable were patients’ responses if they had discontinued based on a number of different clinical factors. But at this point, is there guidance to be given in terms of who we should consider for this? No, I think we need prospective data to tell us the answer.

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