For the first time, NCCN has published guidelines specifically geared toward treating myeloproliferative neoplasms (MPNs). The first set of guidelines was developed for myelofibrosis (MF), and was presented at the NCCN 22nd Annual Conference. Future guidelines will be issued for polycythemia vera, essential thrombocytopenia, and atypical MPNs. Patients with MF can have an unpredictable course, one that is largely dependent on the presence of certain molecular alterations. Models are currently emerging that take into account molecular factors. Only one drug is currently approved for MF, the oral JAK1/2 inhibitor ruxolitinib, which has been shown to significantly reduce splenomegaly and improve symptoms.
Brady L. Stein, Susan O’Brien, Peter Greenberg and Ruben A. Mesa
Ruben Mesa, Catriona Jamieson, Ravi Bhatia, Michael W. Deininger, Aaron T. Gerds, Ivana Gojo, Jason Gotlib, Krishna Gundabolu, Gabriela Hobbs, Rebecca B. Klisovic, Patricia Kropf, Sanjay R. Mohan, Stephen Oh, Eric Padron, Nikolai Podoltsev, Daniel A. Pollyea, Raajit Rampal, Lindsay A. M. Rein, Bart Scott, David S. Snyder, Brady L. Stein, Srdan Verstovsek, Martha Wadleigh, Eunice S. Wang, Mary Anne Bergman, Kristina M. Gregory and Hema Sundar
Myelofibrosis (MF), polycythemia vera (PV), and essential thrombocythemia (ET) are a group of heterogeneous disorders of the hematopoietic system collectively known as Philadelphia chromosome–negative myeloproliferative neoplasms (MPNs). The diagnosis and the management of patients with MPNs have evolved since the identification of mutations that activate the JAK pathway (JAK2, CALR, and MPL mutations) and the development of targeted therapies has resulted in significant improvements in disease-related symptoms and quality of life. This manuscript discusses the recommendations outlined in the NCCN Guidelines for the diagnostic workup of MPN (MF, PV, and ET), risk stratification, treatment, and supportive care strategies for the management of MF.
Brady L. Stein, Jason Gotlib, Murat Arcasoy, Marie Huong Nguyen, Neil Shah, Alison Moliterno, Catriona Jamieson, Daniel A. Pollyea, Bart Scott, Martha Wadleigh, Ross Levine, Rami Komrokji, Rebecca Klisovic, Krishna Gundabolu, Patricia Kropf, Meir Wetzler, Stephen T. Oh, Raul Ribeiro, Rita Paschal, Sanjay Mohan, Nikolai Podoltsev, Josef Prchal, Moshe Talpaz, David Snyder, Srdan Verstovsek and Ruben A. Mesa
The classical Philadelphia chromosome–negative myeloproliferative neoplasms (MPN), which include essential thrombocythemia, polycythemia vera, and myelofibrosis (MF), are in a new era of molecular diagnosis, ushered in by the identification of the JAK2V617F and cMPL mutations in 2005 and 2006, respectively, and the CALR mutations in 2013. Coupled with increased knowledge of disease pathogenesis and refined diagnostic criteria and prognostic scoring systems, a more nuanced appreciation has emerged of the burden of MPN in the United States, including the prevalence, symptom burden, and impact on quality of life. Biological advances in MPN have translated into the rapid development of novel therapeutics, culminating in the approval of the first treatment for MF, the JAK1/JAK2 inhibitor ruxolitinib. However, certain practical aspects of care, such as those regarding diagnosis, prevention of vascular events, choice of cytoreductive agent, and planning for therapies, present challenges for hematologists/oncologists, and are discussed in this article.
Ruben A. Mesa, Catriona Jamieson, Ravi Bhatia, Michael W. Deininger, Christopher D. Fletcher, Aaron T. Gerds, Ivana Gojo, Jason Gotlib, Krishna Gundabolu, Gabriela Hobbs, Brandon McMahon, Sanjay R. Mohan, Stephen Oh, Eric Padron, Nikolaos Papadantonakis, Philip Pancari, Nikolai Podoltsev, Raajit Rampal, Erik Ranheim, Vishnu Reddy, Lindsay A.M. Rein, Bart Scott, David S. Snyder, Brady L. Stein, Moshe Talpaz, Srdan Verstovsek, Martha Wadleigh, Eunice S. Wang, Mary Anne Bergman, Kristina M. Gregory and Hema Sundar
Myeloproliferative neoplasms (MPNs) are a group of heterogeneous disorders of the hematopoietic system that include myelofibrosis (MF), polycythemia vera (PV), and essential thrombocythemia (ET). PV and ET are characterized by significant thrombohemorrhagic complications and a high risk of transformation to MF and acute myeloid leukemia. The diagnosis and management of PV and ET has evolved since the identification of mutations implicated in their pathogenesis. These NCCN Guideline Insights discuss the recommendations outlined in the NCCN Guidelines for the risk stratification, treatment, and special considerations for the management of PV and ET.