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Unmet Needs and Future Directions in Treating MPNs

Panelists: Harry P. Erba, MD, PhD, Duke University; Rami Komrokji, MD, Moffitt Cancer Center; Ruben A. Mesa, MD, FACP, The Mays Cancer Center, the newly named center of UT Health San Antonio MD Anderson Cancer Center; Jamile M. Shammo, MD, FASCP, FACP, Rush University Medical Center; Srdan Verstovsek, MD, PhD, MD Anderson Cancer Center; Srdan Verstovsek, MD, PhD, MD Anderson Cancer Center
Published: Thursday, Aug 30, 2018



Transcript: 

Harry P. Erba, MD, PhD: Srdan, what do you think of the unmet needs for the near future, at least in PV [polycythemia vera] and MF [myelofibrosis]?

Srdan Verstovsek, MD, PhD: For myelofibrosis, it’s relatively easier, because we all agree that after ruxolitinib, life is short and very bad for patients. That’s an area of unmet need, and so our efforts are in developing something after ruxolitinib. But No. 2 would be anemia. Everybody is anemic and gets worse over time, and we don’t have any medication approved for that. Occasionally, we have response to danazol, prednisone, or erythropoietin, but nothing really there. Then you would like to extend or improve ruxolitinib’s activity itself by adding something to it. That will be No. 3 for myelofibrosis: Make it better, longer lasting, or add an anemia drug to it. The combination will be better frontline.

And then, in my lifetime, my goal would be to get something to prevent progression and not wait for patients to get sick and then intervene. What we are doing are intervention studies. How about prevention studies? We haven’t done it yet, but, hopefully, we will in myelofibrosis. For PV, we should up the bar. We are controlling the signs and symptoms. We are not talking about progression or prevention of the progression. We are talking about prevention of thrombosis but not progression, and certainly not about elimination of the disease. We need to up the bar in PV.  

Harry P. Erba, MD, PhD: Well, Srdan, I spend most of my day taking care of patients with acute myeloid leukemia [AML], so anything you guys could do to prevent progression of myeloproliferative neoplasms or AML will be greatly appreciated by my patients and by me. After this, get back to work, OK?

Ruben A. Mesa, MD, FACP: There we go.

Harry P. Erba, MD, PhD: We’ve just covered a lot of information. Before we end this discussion, I’d like to ask each of you to provide a takeaway from today’s program. Let’s start with you, Dr Komrokji.

Rami Komrokji, MD: I think in recent years, we’ve learned a lot about the disease and its biology. We are redefining the criteria for those diseases and how to be diagnosed or risk stratified. I think we have options for treatment that were not available the past few years for patients. I think the message for community physicians is basically that those patients can sometimes benefit from seeing experts in the field, at least for a second opinion, where we can partner with them, see those patients, do all that, then return the patients to be treated in the community. I think it’s getting complicated enough that the patients will benefit from that. As a message for the patients, I think this is a promising time. For the first time, we have some drugs approved, and we have talked about several drugs coming. We’ve learned in the last 5 or 10 years about the disease biology more than probably the last 50 years, so, hopefully, within the coming few years we’ll have more effective treatments for patients.

Harry P. Erba, MD, PhD: With that expert hematology review comes expert pathology review, too, which is a key point.

Rami Komrokji, MD: Absolutely a key point.

Harry P. Erba, MD, PhD: Dr Mesa?

Ruben A. Mesa, MD, FACP: I would add on to that and agree that I’m incredibly hopeful. During my career, I’ve really witnessed a change where in MPNs, we’ve largely used agents for which we did not have a strong, scientific rationale. They were empirically being used from the development from other indications. We have now seen really a robust pipeline of new hypothesis-driven drugs being tested in well-done randomized phase III clinical trials, as well as a much-expanded understanding of what clinical benefit really does mean in MPNs at the different phases of disease. I think we still have a way to go, but I’m very hopeful for the future.

Harry P. Erba, MD, PhD: Dr. Shammo?

Jamile M. Shammo, MD, FASCP, FACP: I think that patients who have PV and MF are those who have myeloid malignancies, and they deserve to have to be taken care of as such. My point is to say that those patients should actually be monitored just as anybody else who has any kind of hematologic malignancies would be, have their symptoms evaluated, and be considered for treatment when they require it.

Harry P. Erba, MD, PhD: I agree with that, and a lot of it is symptom control, right? Having somewhere in your clinic to have patients report their symptoms and thinking about how they’re related to these diseases and intervening. And Dr. Verstovsek?

Srdan Verstovsek, MD, PhD: Just to add a note on the actual drug developments, I agree with the summaries so far, and I’m very encouraged where we are now compared with 15 years ago, where the introduction of the JAK2 mutation happened. But in terms of where we are heading, I’m very pleased to see, like Ruben said, new ways of tackling the disease. In the next 3 years, I would expect that we’re going to have a medication for anemia and medication to treat patients in the second line: fedratinib, luspatercept, the drugs that we mentioned. Ropeginterferon is certainly a very active medication and easy to tolerate in patients with ET [extended thrombosis] and PV. Perhaps we’ll even see ruxolitinib in patients with ET, not just in PV. I think within 3 to 5 years, a number of new drugs will be approved and help us in management of our patients.

Harry P. Erba, MD, PhD: Thank you. Thank you all for your contributions to this discussion. On behalf of our panel, we thank you for joining us for this Peer Exchange discussion.

Transcript Edited for Clarity 

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Transcript: 

Harry P. Erba, MD, PhD: Srdan, what do you think of the unmet needs for the near future, at least in PV [polycythemia vera] and MF [myelofibrosis]?

Srdan Verstovsek, MD, PhD: For myelofibrosis, it’s relatively easier, because we all agree that after ruxolitinib, life is short and very bad for patients. That’s an area of unmet need, and so our efforts are in developing something after ruxolitinib. But No. 2 would be anemia. Everybody is anemic and gets worse over time, and we don’t have any medication approved for that. Occasionally, we have response to danazol, prednisone, or erythropoietin, but nothing really there. Then you would like to extend or improve ruxolitinib’s activity itself by adding something to it. That will be No. 3 for myelofibrosis: Make it better, longer lasting, or add an anemia drug to it. The combination will be better frontline.

And then, in my lifetime, my goal would be to get something to prevent progression and not wait for patients to get sick and then intervene. What we are doing are intervention studies. How about prevention studies? We haven’t done it yet, but, hopefully, we will in myelofibrosis. For PV, we should up the bar. We are controlling the signs and symptoms. We are not talking about progression or prevention of the progression. We are talking about prevention of thrombosis but not progression, and certainly not about elimination of the disease. We need to up the bar in PV.  

Harry P. Erba, MD, PhD: Well, Srdan, I spend most of my day taking care of patients with acute myeloid leukemia [AML], so anything you guys could do to prevent progression of myeloproliferative neoplasms or AML will be greatly appreciated by my patients and by me. After this, get back to work, OK?

Ruben A. Mesa, MD, FACP: There we go.

Harry P. Erba, MD, PhD: We’ve just covered a lot of information. Before we end this discussion, I’d like to ask each of you to provide a takeaway from today’s program. Let’s start with you, Dr Komrokji.

Rami Komrokji, MD: I think in recent years, we’ve learned a lot about the disease and its biology. We are redefining the criteria for those diseases and how to be diagnosed or risk stratified. I think we have options for treatment that were not available the past few years for patients. I think the message for community physicians is basically that those patients can sometimes benefit from seeing experts in the field, at least for a second opinion, where we can partner with them, see those patients, do all that, then return the patients to be treated in the community. I think it’s getting complicated enough that the patients will benefit from that. As a message for the patients, I think this is a promising time. For the first time, we have some drugs approved, and we have talked about several drugs coming. We’ve learned in the last 5 or 10 years about the disease biology more than probably the last 50 years, so, hopefully, within the coming few years we’ll have more effective treatments for patients.

Harry P. Erba, MD, PhD: With that expert hematology review comes expert pathology review, too, which is a key point.

Rami Komrokji, MD: Absolutely a key point.

Harry P. Erba, MD, PhD: Dr Mesa?

Ruben A. Mesa, MD, FACP: I would add on to that and agree that I’m incredibly hopeful. During my career, I’ve really witnessed a change where in MPNs, we’ve largely used agents for which we did not have a strong, scientific rationale. They were empirically being used from the development from other indications. We have now seen really a robust pipeline of new hypothesis-driven drugs being tested in well-done randomized phase III clinical trials, as well as a much-expanded understanding of what clinical benefit really does mean in MPNs at the different phases of disease. I think we still have a way to go, but I’m very hopeful for the future.

Harry P. Erba, MD, PhD: Dr. Shammo?

Jamile M. Shammo, MD, FASCP, FACP: I think that patients who have PV and MF are those who have myeloid malignancies, and they deserve to have to be taken care of as such. My point is to say that those patients should actually be monitored just as anybody else who has any kind of hematologic malignancies would be, have their symptoms evaluated, and be considered for treatment when they require it.

Harry P. Erba, MD, PhD: I agree with that, and a lot of it is symptom control, right? Having somewhere in your clinic to have patients report their symptoms and thinking about how they’re related to these diseases and intervening. And Dr. Verstovsek?

Srdan Verstovsek, MD, PhD: Just to add a note on the actual drug developments, I agree with the summaries so far, and I’m very encouraged where we are now compared with 15 years ago, where the introduction of the JAK2 mutation happened. But in terms of where we are heading, I’m very pleased to see, like Ruben said, new ways of tackling the disease. In the next 3 years, I would expect that we’re going to have a medication for anemia and medication to treat patients in the second line: fedratinib, luspatercept, the drugs that we mentioned. Ropeginterferon is certainly a very active medication and easy to tolerate in patients with ET [extended thrombosis] and PV. Perhaps we’ll even see ruxolitinib in patients with ET, not just in PV. I think within 3 to 5 years, a number of new drugs will be approved and help us in management of our patients.

Harry P. Erba, MD, PhD: Thank you. Thank you all for your contributions to this discussion. On behalf of our panel, we thank you for joining us for this Peer Exchange discussion.

Transcript Edited for Clarity 
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