Dr. Mesa on NCCN Guidelines for MPNs

Ruben A. Mesa, MD, chair, Division of Hematology and Medical Oncology, deputy director, professor of Medicine, Mayo Clinic, discusses the NCCN guidelines to guide physicians for treatment of patients with myeloproliferative neoplasms (MPNs).

Mesa says he hopes these guidelines for MPNs will have an impact on quality, as well as lead to more homogenous treatment across the United States. In the set of guidelines, there are recommendations for prognosis, diagnosis, treatment, and supportive care strategies for essential thrombocythemia, polycythemia vera, and myelofibrosis. Regarding treatment, the NCCN describes the role of ruxotlinib (Jakafi) for patients with myelofibrosis.

While the on-label indications in intermediate-2 and high-risk patients are clear, researchers are still figuring out what the contexts are to use the drug in low-risk patients. The NCCN guidelines for patients with low-risk myelofibrosis who are asymptomatic recommend observation or a clinical trial.

In June 2016, final 5-year efficacy and safety data from the phase III COMFORT-I trial confirmed previous findings that ruxolitinib was associated with a significant benefit in patients with intermediate-2 and high-risk myelofibrosis. The JAK2 inhibitor also resulted in durable reductions in splenomegaly and an improvement in survival. The median follow-up was 268.4 weeks for the ruxolitinib group and 269 weeks for the placebo arm. The median overall survival was not yet reached for patients receiving ruxolitinib compared with 200 weeks for the control group. Patients ultimately did not experience any new or unexpected adverse events as a result of the long-term treatment. The FDA approved ruxolitinib in November 2011 for the treatment of patients with myelofibrosis.

Experts are also determining the optimal time for transplantation and considerable risk factors. Clinical trial data should also be considered for other therapies, such as interferon and others that can be supportive of anemia.