Published on April 21, 2016
As part of our â??Ask the Expertâ?? series, Dr. Naveen Pemmaraju of MD Anderson Cancer Center responds to Patient Power Community member Markâ??s question about the pros and cons of beginning myelofibrosis treatment to prevent progression. Listen as Dr. Pemmaraju discusses the DIPSS scoring system and how, and if, treatment is beneficial and recommended.
The Ask the Expert series is sponsored through an educational grant to the Patient Empowerment Network from Incyte Corporation.
Transcript | My MPN Is Stable: What Are the Pros and Cons of Starting Treatment?
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Here’s a question we got from Mark. Mark writes in, “I have myelofibrosis and I take no medication. I have an enlarged spleen, a few symptoms—not too many—just mostly intermittent itching, and have stayed off drugs the last 15 years since I actually started with PV. Wow. I often wonder if I should start treatment to prevent progression. What are the pros and cons?”
This is one of the most important areas I think moving forward over the next five years. The first part of the question is important. Myelofibrosis, as you know and a lot of our viewers know but not everyone comes in different stages and different flavors. While we don’t use the same staging system as the solid tumors: sort of stage
I, II III, IV; in 2009 our colleague in Europe, Cervantes et al in Blood found that hey, there is a staging system. We call it the IPSS or International Prognostic Scoring System, and since that time many other groups including the Mayo Clinic group have refined it.
But using the scoring system there is such a thing as low-risk for myelofibrosis, intermediate I, intermediate II, and high risk. Obviously, we don’t know the details of every patient including our very nice question here, but in general for a low-risk patient we think of their survival as measured in decades. I love saying that, and you know I love saying that to you here in our interview. So that’s great. So hopefully it sounds like with our patient’s question they may fit into this new low-risk category.
The standard of care has been and still is that if you fit into these lower risk categories, yes it’s okay that you don’t have a treatment. You’re probably being followed by a doctor observation, and we think this is wonderful, and I hope it stays like that forever. If a patient is either diagnosed or moves into the intermediate or the high-risk categoriesm that’s where you start to think about therapeutic options. Importantly, this JAK inhibitor, ruxolutinib (Jakafi), is actually only approved for intermediate to high-risk patients. I think it’s something very important, and I want to make sure that we mention that here.
For the early-risk patients, there is a large clinical trial actually that’s starting in Europe to answer the very question that our questioner has asked. Is there a role for JAK inhibitors as an early, let’s say “preventive” strategy? So that trial is now underway in Europe. It’s a big trial, and hopefully it will be enough to answer this question, so we’ll see.