MPN Expert On How New Guidelines Will Affect Disease Treatment

The National Comprehensive Cancer Network (NCCN) recently issued new guidelines for myeloproliferative neoplasms (MPNs). They specifically outline diagnosis, treatment and supportive care strategies for myelofibrosis (MF).

Rubin Mesa, M.D., professor of Hematology at the Mayo Clinic in Arizona, explained the changes and what lies ahead in the treatment of MPNs during a presentation at the 34th Annual Chemotherapy Foundation Symposium™.

Can you give a recap of your talk on the evolving guidelines for MPNs?

In an interview with CURE, Mesa provided an overview of the new NCCN guidelines and what they mean for MPNs are managed and treated.We focused first on diagnosis, prognosis and symptom burden, validating and endorsing the criteria for diagnosis, the already recognized prognostic scores in these diseases, as well as the importance of symptom assessment.

The guidelines have really begun first with the therapy for myelofibrosis to clarify risk assessment and disease burden in patients. We then stratify really by the risk itself and then stratify patients between those that we observe, such as low-risk asymptomatic; those that we consider for medical therapy with Jakafi (ruxolitinib); as well as trying to clarify where we can consider clinical trials, which really occurs all along the way. Also, the guidelines address the importance of stem cell transplantation, in particular for intermediate 1 with high-risk molecular features, intermediate 2 and high-risk in good transplant candidates and crucially for individuals that are evolving toward acute leukemia.

Will these guidelines also be updated to include recommendations for essential thrombocythemia (ET) and polycythemia vera (PV) as well?

I hope these new guidelines will provide greater clarity and uniformity of care for myelofibrosis in the U.S.That is correct. There is a lot to tackle to begin with, so we anticipate the next step will include treatment guidelines for PV and ET. And then subsequently, guidelines for the less common MPNs of hypereosinophilic syndrome, systemic mast cell disease, etc.

How will these guidelines be used in clinical practice?

Additionally, the myelofibrosis guidelines will be routinely updated as we have potentially other drugs that are currently in the pipeline. If they’re approved, the guidelines will be updated very shortly thereafter, so that we can see where those best fit.We hope the guidelines will be useful. We recognize the challenges involved in trying to keep track of so many different diseases — just keeping abreast of the number of new drugs approved in myeloma alone would be enough to be a full-time job for anyone.

The guidelines also address some of the key — and more challenging — decision points. Sometimes, for patients for whom there is uncertainty about appropriate treatment, that can be a time to bounce options off individuals who focus on the disease or utilize platforms such as the American Society of

As an expert in the field, how would you describe what having NCCN guidelines means for MPNs?

What kind of role do you think transplantation will continue to have in this disease?

Hematology’s “Ask an Expert” platform. Challenging cases involving transplantation, in particular, is an area where conferring with other experts can be helpful. MPNs were a group of diseases that had lived on this border between benign and malignant disease, and that really led to a lack of clarity, resources for research and confusion. We clearly recognize that they are chronic leukemias, and being considered in parallel with other hematologic malignancies is very important. It’s important for patients in terms of the resources and the support that they receive. These guidelines provide clarity, so I think it’s very helpful for the field — for patients and for treating physicians.I think it will continue to play an important role. Key questions that remain are: What is the optimal timing, and how do we continue to decrease the morbidity that comes along with transplantation?

Finally, when we identify that there are individuals with high-risk disease who have a high risk of relapse post-transplantation, how do we continue to decrease that risk?

So the era of “you have your transplant and just hope for the best” — I think that’s evolving. It will be refined with more intensive pre- and post-transplantation therapy.