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Updated: Apr 15, 2026

Use of Hematopoietic Stem Cell Transplantation to Assess the Origin of Myelodysplastic Syndrome
Published on: October 3, 2018
Historical views, conventional approaches, and evolving management strategies for myeloproliferative neoplasms
Brady L Stein1, Jason Gotlib1, Murat Arcasoy1
1From Northwestern University Feinberg School of Medicine, Chicago, Illinois; Stanford Cancer Institute, Stanford University, Stanford, California; Duke Cancer Institute, Duke University, Durham, North Carolina; University of Michigan Comprehensive Cancer Center, Ann Arbor, Michigan; University of California, San Francisco, San Francisco, California; Johns Hopkins University School of Medicine, Baltimore, Maryland; Moores Cancer Center, UC San Diego, San Diego, California; University of Colorado Cancer Center, Aurora, Colorado; Fred Hutchinson Cancer Research Center, Seattle, Washington; Dana Farber Cancer Institute, Boston, Massachusetts; Memorial Sloan Kettering Cancer Center, New York; New York; Moffitt Cancer Center and Research Institute, Tampa, Florida; The Ohio State University, Columbus, Ohio; Fred & Pamela Buffett Cancer Center, Omaha, Nebraska; Fox Chase Cancer Center, Philadelphia, Pennsylvania; Roswell Park Cancer Institute, Buffalo, New York; Washington University-Siteman Cancer Center, St Louis, Missouri; St. Jude Children's Research Hospital, Memphis, Tennessee; University of Alabama at Birmingham, Birmingham, Alabama; Vanderbilt-Ingram Cancer Center, Nashville, Tennessee; Yale University School of Medicine; New Haven, Connecticut; University of Utah, Salt Lake City, Utah; University of Michigan Comprehensive Cancer Center, Ann Arbor, Michigan; City of Hope Cancer Center, Los Angeles, California; The University of Texas MD Anderson Cancer Center, Houston, Texas; and Mayo Clinic Cancer Center, Scottsdale, Arizona.
Abstract:
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 JAK2(V617F) 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.
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