New York, NY—Myeloproliferative neoplasms (MPNs) include myelofibrosis, polycythemia vera, and essential thrombocytopenia, and affect an estimated 295,00 individuals in the United States. MPNs represent a diverse array of diseases. This has led to the generation of various treatment guidelines that are often contradictory and difficult to interpret. However, the National Comprehensive Cancer Network (NCCN) is developing a unified field theory on the diagnosis and treatment of MPNs.
On September 23, 2016, the NCCN published its guidelines on the diagnosis, treatment, and management of myelofibrosis to provide healthcare professionals with up-to-date, evidence-based recommendations. Although the majority of the guidelines are focused on the management of myelofibrosis, several sections that complete recommendations for the management of essential thrombocytopenia and polycythemia vera are expected to be published in later versions of the guidelines. These are the first guidelines to be published for MPNs by the NCCN.
At the 2016 NCCN Congress on Hematologic Malignancies, Ruben Mesa, MD, Mayo Clinic College of Medicine, Scottsdale, AZ, and Co-Chair of the NCCN’s MPN Panel, discussed the NCCN’s new guidelines for the diagnosis, treatment, and management of myelofibrosis.
“The NCCN guidelines for myelofibrosis were published on September 23, so they’re certainly up-to-date. The NCCN Panel on Myeloproliferative Neoplasms included many of the key opinion leaders in the United States, and we think that the guidelines represent a superlative standard of care. We decided to tackle the treatment guidelines for myelofibrosis first, because it is the most severe of the MPNs, and it has the greatest unmet need in terms of standardized guidelines,” said Dr Mesa.
The NCCN guidelines on myelofibrosis endorse the World Health Organization (WHO)’s 2016 criteria for the diagnosis of myelofibrosis. The NCCN guidelines, however, incorporate features into the diagnosis of myelofibrosis that are not included in the WHO criteria, including diagnostic features that affect patient outcomes, such as vascular events, disease progression, cytopenias, splenomegaly, burden of symptoms, and baseline health and comorbidities.
When assessing the disease burden of myelofibrosis, Dr Mesa said that the NCCN endorsed the MPN Symptom Assessment Form total symptom score, which includes 10 items from 2 previously validated scoring systems. As for the prognosis of myelofibrosis, he noted that the NCCN endorses the International Prognostic Scoring System (IPSS) for myelofibrosis at the time of diagnosis, and the Dynamic International Prognostic Scoring System (DIPSS) for primary myelofibrosis subsequent to diagnosis.
In addition, Dr Mesa said that the NCCN incorporated the Mutation-Enhanced International Prognostic Scoring System for primary myelofibrosis, because mutations play an integral role in myelofibrosis progression and outcomes.
According to the new NCCN guidelines, patients with IPSS or DIPSS scores of 0 have low-risk myelofibrosis. If these patients are asymptomatic, the NCCN recommends observation or participation in a clinical trial. Conversely, patients with symptomatic myelofibrosis have 3 treatment options, including ruxolitinib (Jakafi), possibly interferons, or participation in a clinical trial.
Patients with IPSS or DIPSS scores of 1 have intermediate risk-1 myelofibrosis. These patients are typically the most challenging in terms of decision-making for treating physicians, because they are a very heterogeneous group. Patients can have intermediate risk-1 myelofibrosis for a variety of reasons, including age, symptoms, or anemia.
“For intermediate risk-1 patients, the NCCN guidelines empower treating physicians with a variety of options, including observation, ruxolitinib, a clinical trial, or hematopoietic-cell transplantation. The NCCN recommends intermediate risk-1 patients be monitored for symptoms of disease progression every 3 to 6 months,” Dr Mesa told attendees.
In the new NCCN guidelines for myelofibrosis, an IPSS score of 2 or a DIPSS score of 3 or 4 delineates intermediate risk-2 myelofibrosis, and an IPSS score of 3 or a DIPSS score of 5 or 6 delineates high-risk patients.
The guidelines recommend that these patients receive an allogeneic stem-cell transplantation if they are suitable candidates. If the patients are not suitable candidates for stem-cell transplantation, then they are stratified by their platelet counts.
Patients with platelet counts <50,000 are directed to participate in a clinical trial, and patients with platelet counts >50,000 are directed to participate in a clinical trial or to receive ruxolitinib therapy.
“If the patients’ myelofibrosis progresses to [acute myeloid leukemia], the NCCN guidelines state that the patients should receive bone marrow aspiration and biopsies, bone marrow cytogenetic testing, and also molecular testing for AML-associated mutations,” said Dr Mesa.
