Tag Archive for: myelofibrosis treatment options
How Are Prognostic Scoring Systems Used in Myelofibrosis Care?
How are scoring systems such as DIPSS used in myelofibrosis care? Dr. Pemmaraju explains how these tools assess myelofibrosis prognosis and guide treatment decisions.
Dr. Naveen Pemmaraju is Director of the Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) Program and Professor in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. Learn more about Dr. Pemmaraju.
See More from Elevate Myelofibrosis
Related Resources:
Myelofibrosis Care | Impact of Diet & Lifestyle Modifications |
Myelofibrosis Symptoms and Side Effects | Why Speaking Up Is Vital |
|
Transcript:
Katherine Banwell:
“Can you explain the dynamic international prognostic scoring system or DIPSS?” Thank goodness there’s an acronym for that.
Dr. Naveen Pemmaraju:
Yeah, no, it’s a great question, scoring systems, right?
Katherine Banwell:
Yeah, and Cliff wants to know how he can ask his doctor about it.
Dr. Naveen Pemmaraju:
Right, so the easiest way to talk about it, the good news is everything we’ve been talking about is incorporated in the scoring system. So, said in another way, we’ve been talking about it subjectively, the scoring systems try to make the subject objective. So, quick history, these started in 2009 with the IPSS, International Prognostic Scoring System. The concept there were a thousand patients in Europe and basically trying to observe the natural history of the progression of myelofibrosis. This was just before, just as the JAK inhibitor era was starting. What we found is that the four groups nicely separate.
So, the lowest of the low-risk group potentially can be measured in decades for overall survival. Intermediate one, intermediate two, and high risk, again, all separated by overall survival and AML leukemia transformation risk. Now, that’s evolved over time as the questioner is asking for more sophisticated scoring systems. So, that’s all you need to know. So, DIPSS Plus just means Dynamic International Prognostic Scoring System.
Then there’s DIPSS plus, and can you believe it? There’s even the MIPSS now, the Molecular International Prognostic Scoring System. All right. So, at least there’s a rhyme and reason there. I think each iteration is telling you that we are dynamically understanding more about the disease. Two, the IPSS, the original one, was meant to be only at diagnosis, and the DIPSS by definition, dynamic scoring, is any time during the course of the disease, that’s interesting. Then three, they’re incorporating new factors each time.
So, from the time of the IPSS to the DIPSS and now the MIPSS, you’re incorporating all these factors that we couldn’t before. Cytogenetics, molecular findings, anemia, transfusion, burn, thrombocytopenia, etc. So, that’s basically it. You can ask your doctor. I mean, basically, in the course of what we do in the non-clinical trial standard of care, even if somebody doesn’t hand stop and calculate these risk scores, we’re talking about the same thing, right? The subjective or the objective matchup.
However, of interest to the patients, there are calculators that are available, you know, obviously rather than doing it in isolation in your house. Yes, it is better, I agree to do it with your doctor, with your provider team, and see what it means for you. The goal of these is twofold. In clinical trials to help stratify patients so you can understand who’s high risk versus lower. However, in the standard of care, sure it may help with transplant decisions, referrals for clinical trials, etc.
What Are Treatment Options for Myelofibrosis?
What Are Treatment Options for Myelofibrosis? from Patient Empowerment Network on Vimeo.
MPN specialist Dr. Abdulraheem Yacoub shares and overview of treatment options, including therapies in development, for patients living with myelofibrosis (MF).
Dr. Abdulraheem Yacoub is a hematologist oncologist at the University of Kansas Cancer Center. Dr. Yacoub is an active researcher and is an Associate Professor of Hematologic Malignancies and Cellular Therapeutics. Learn more about Dr. Yacoub, here.
Related Programs:
|
|
How Should You Participate in MPN Care and Treatment Decisions? |
Transcript:
Katherine:
And since myelofibrosis is a progressive condition, I imagine it’s more difficult to manage. So, what else is available for patients with myelofibrosis?
Dr. Yacoub:
Correct So, myelofibrosis is the higher end of this spectrum of cancers.
It is a cancer that is associated with much higher symptom burden and impact on daily life. It is also associated with low blood counts, and some patients will require transfusions. It’s a major morbidity to our patients. And in addition, it’s a cancer that is associated with shortened life. So, patients with myelofibrosis will not live as long as their health would have allowed them. And some of them will live actually a much shorter life than they want or deserve.
So, myelofibrosis treatment requires a lot more considerations. So, for patients who are in good health, who have a cancer that is more aggressive, that would be imminently impacting their longevity, we start a discussion about a curative role of allogeneic stem cell transplantation very early in their course.
Because bone marrow transplantation can be curative, and those patients can live a long life after a successful transplant. So, this is a treatment modality that should be brought up very early for patients with higher risk myelofibrosis. There are approved JAK inhibitors, ruxolitinib (Jakafi) and fedratinib (Inrebic). And we know that ruxolitinib which has been approved for over 10 years can improve symptoms, can improve the spleen volume, can actually prolong lives for patients on it, and also makes the transplant more successful.
So, we should be offering that to the appropriate patients also early in their diagnosis, in a strategy where, in addition to that, we get them to a transplant. Fedratinib is approved in that setting. And we are very optimistic that by the end of this calendar year, we will have two other JAK inhibitors approved.
[Editor’s Note: As of February 28, 2022, pacritinib (Vonjo) has been approved for the treatment of myelofibrosis patients with severe thrombocytopenia.]
So, we look forward to those two drugs. Momelotinib and pacritinib for patients with special disease features. And hopefully, by the end of this year, we will have a list of JAK inhibitors that we can choose from, which is great news for our patients.
Katherine:
Oh, we’re still fighting.
Dr. Yacoub:
Yes, absolutely.
