ORLANDO (FRONTLINE MEDICAL NEWS) – A guideline published late last year for the diagnostic work-up of myeloproliferative neoplasms and for the management of myelofibrosis in particular is just the first in a series of National Comprehensive Cancer Network guidelines on this “family of myeloid neoplasms,” according to the guideline panel chair, Ruben A. Mesa, MD.

The myeloproliferative neoplasm (MPN) guideline panel first worked to develop a framework based on existing understanding of the MPNs. Members consulted with two other panels working in the area of chronic myeloid diseases, including chronic myeloid leukemia and myelodysplastic syndrome.

“We were in agreement that these are different entities, and our treatments are different, our guidelines are different,” Dr. Mesa of the Mayo Clinic Cancer Center, Phoenix, said at the annual conference of the National Comprehensive Cancer Network.

That said, there are also some shared circumstances. For example, all three sets of diseases can progress to acute myeloid leukemia.

“Indeed, I view this very much as pieces in a jigsaw puzzle. … It is important that we recognize their interdependencies as well as those aspects that are disease specific,” he said.

In essence, however, the MPN guideline development is a from-scratch effort, as these are the first guidelines for these disorders, he noted.

The effort is timely, as the diagnosis and management of patients with MPNs have rapidly evolved since the identification of mutations that activate the JAK pathway, including JAK2, CALR, and MPL mutations. Further, the development of targeted therapies – such as the JAK1 and JAK2 inhibitor ruxolitinib, which was the first drug approved for the treatment of myelofibrosis – has resulted in significant improvements in disease-related symptoms and quality of life.

The panel is focusing first on the “core classic” Philadelphia chromosome–negative MPNs: myelofibrosis, polycythemia vera, and essential thrombocythemia. The first piece to be placed in the MPN puzzle was the guideline for the diagnostic work-up of these entities and for risk stratification, treatment, and supportive care strategies for the management of myelofibrosis, which the panel considered “the greatest unmet need and the most urgent in terms of guidance,” Dr. Mesa said.

This initial MPN guideline was published in December (J Natl Compr Canc Netw. 2016;14:1572-611) and, because of the evolving understanding of MPNs, updates are already under consideration as additional MPN guidelines are being developed.

“We have been actively working … to develop the next set of treatment guidelines, which are the treatment guidelines for polycythemia vera and essential thrombocythemia. Finally, we will work to include the atypical MPNs,” he said, noting that the latter include hypereosinophilic disease, systemic mast cell disease, and other atypical illnesses.

These represent a small number of patients, but “their management is key, it’s distinct from the others, and there is no good guidance,” Dr. Mesa said.

“Once this is fleshed out, we will then have a fully developed set of guidelines that then will be maintained along the traditional process that NCCN follows, which is first an annual review, but second, a monitoring in real time of key developments that could impact the guidelines during the off-cycle,” he said.

Dr. Mesa disclosed that he has received consulting fees, honoraria, and/or grant/research support from ARIAD Pharmaceuticals, Celgene, CTI BioPharma, Galena Biopharma, Gilead Sciences, Incyte, Novartis Pharmaceuticals, and Promedior.


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