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Emerging Treatments, New Guidelines Advance MPN Care

Gina Columbus @ginacolumbusonc
Published: Wednesday, Dec 14, 2016

Ruben Mesa, MD

Ruben Mesa, MD

The NCCN recently published new guidelines for myeloproliferative neoplasms (MPNs), with an emphasis on diagnosis, treatment, and supportive care strategies for myelofibrosis. Subsequent editions will focus more on other MPNs, including essential thrombocythemia (ET), polycythemia vera (PV).

During the 2016 OncLive State of the Science Summit on Treatment of Hematologic Malignancies, Ruben Mesa, MD, provided a lecture on MPNs, with a focus on the newly released recommendations.

“My focus was really for our colleagues here on the new guidelines for the therapy for MPNs,” said Mesa. “This is the first time there have been US-based guidelines through the NCCN for the diagnosis and treatment for MPNs, so we’re trying to get this cutting-edge information across to our colleagues.”

In an interview, Mesa, who is chair of the Division of Hematology and Medical Oncology at Mayo Clinic, provided his expert insight on these guidelines; recent advancements for patients with ET, PV, and myelofibrosis; and ongoing clinical trials with the potential to change practice in the management of MPNs.

OncLive: What do the new guidelines state?

Mesa: For the first time ever there are guidelines for MPNs, which we hope will have an impact on equality, as well as trying to have care be more homogenous across the United States. We begin with guidelines regarding the diagnosis and prognosis of ET, PV, and myelofibrosis, and then it really goes into the treatment guidelines for myelofibrosis for this first iteration. We plan, in the beginning of 2017, to have treatment guidelines for ET and PV.

The fundamental of the guidelines are trying to describe with primarily 1 FDA-approved therapy of ruxolitinib (Jakafi) and determining the role for ruxolitinib for patients with myelofibrosis. Both of the on-label indications are in intermediate-2 and high-risk patients, which are now in a clear and mature set of data with ruxolitinib. However, what are the situations for which we consider it for patients with low- and intermediate-1 risk?

We also try to flesh out where transplantation is to be considered, as well as the factors that impact transplantation. We need to look at both risk, quality of the candidate for transplant in terms of their health, as well as where the new molecular mutations fit into play.

Finally, we highlight the importance of many steps in consideration of clinical trials, but discuss a variety of off-label therapies, as well. Where we consider interferons and other therapies that can be supportive, particularly anemia.

What do we know about the role of ruxolitinib in myelofibrosis thus far, and what do we still need to determine with it?

It’s been a very impactful drug for myelofibrosis. It’s been approved for about 5 years, and we clearly see its impact in improving splenomegaly, symptoms, and survival. Without question, as someone who focuses on myelofibrosis, my patients are living longer on ruxolitinib. Our long-term data suggest that there are no long-term, hidden, negative, unexpected side effects, so that’s very reassuring. Despite how beneficial ruxolitinib is, there is still room for improvement without question.

There are patients who benefit from ruxolitinib but eventually have progression of disease, or have room for further benefit, whether it is reduction of splenomegaly and symptoms, or improvement in cytopenia such as anemia or thrombocytopenia.

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