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.
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
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.
Patrick A. Brown, Bijal Shah, Amir Fathi, Matthew Wieduwilt, Anjali Advani, Patricia Aoun, Stefan K. Barta, Michael W. Boyer, Teresa Bryan, Patrick W. Burke, Ryan Cassaday, Peter F. Coccia, Steven E. Coutre, Lloyd E. Damon, Daniel J. DeAngelo, Olga Frankfurt, John P. Greer, Hagop M. Kantarjian, Rebecca B. Klisovic, Gary Kupfer, Mark Litzow, Arthur Liu, Ryan Mattison, Jae Park, Jeffrey Rubnitz, Ayman Saad, Geoffrey L. Uy, Eunice S. Wang, Kristina M. Gregory, and Ndiya Ogba
The prognosis for patients with newly diagnosed acute lymphoblastic leukemia (ALL) has improved with the use of more intensive chemotherapy regimens, tyrosine kinase inhibitors, targeted agents, and allogeneic hematopoietic cell transplantation. However, the management of relapsed or refractory (R/R) ALL remains challenging and prognosis is poor. The NCCN Guidelines for ALL provide recommendations on standard treatment approaches based on current evidence. These NCCN Guidelines Insights summarize treatment recommendations for R/R ALL and highlight important updates, and provide a summary of the panel's discussion and underlying data supporting the most recent recommendations for R/R ALL management.
Joseph C. Alvarnas, Patrick A. Brown, Patricia Aoun, Karen Kuhn Ballen, Stefan K. Barta, Uma Borate, Michael W. Boyer, Patrick W. Burke, Ryan Cassaday, Januario E. Castro, Peter F. Coccia, Steven E. Coutre, Lloyd E. Damon, Daniel J. DeAngelo, Dan Douer, Olga Frankfurt, John P. Greer, Robert A. Johnson, Hagop M. Kantarjian, Rebecca B. Klisovic, Gary Kupfer, Mark Litzow, Arthur Liu, Arati V. Rao, Bijal Shah, Geoffrey L. Uy, Eunice S. Wang, Andrew D. Zelenetz, Kristina Gregory, and Courtney Smith
Treatment of acute lymphoblastic leukemia (ALL) continues to advance, as evidenced by the improved risk stratification of patients and development of newer treatment options. Identification of ALL subtypes based on immunophenotyping and cytogenetic and molecular markers has resulted in the inclusion of Philadelphia-like ALL and early T-cell precursor ALL as subtypes that affect prognosis. Identification of Ikaros mutations has also emerged as a prognostic factor. In addition to improved prognostication, treatment options for patients with ALL have expanded, particularly with regard to relapsed/refractory ALL. Continued development of second-generation tyrosine kinase inhibitors and the emergence of immunotherapy, including blinatumomab and chimeric antigen receptor T-cell therapy, have improved survival. Furthermore, incorporation of minimal residual disease (MRD) monitoring has shown insight into patient outcomes and may lead to treatment modification or alternative treatment strategies in select populations. This excerpt focuses on the sections of the ALL guidelines specific to clinical presentation and diagnosis, treatment of relapsed/refractory ALL, and incorporation of MRD monitoring. To view the most recent complete version of these guidelines, visit NCCN.org.