title: Hematopoietic Cell Transplantation as Curative Therapy for Patients with Myelofibrosis: Long-Term Success in all Age Groups.
abstract: Myeloproliferative neoplasms (MPN) are chronic marrow disorders with variable prognoses. Most patients with polycythemia vera, essential thrombocythemia, or even primary myelofibrosis (PMF) are successfully treated with conservative strategies for years or even decades, and recent data suggest that even in patients with high-risk disease, in particular those with PMF, life expectancy can be extended by treatment with janus kinase (JAK2) inhibitors. However, none of those modalities are curative, and after marrow failure develops, the disease "accelerates," or transforms to acute leukemia, the only option able to effectively treat and, in fact, cure MPN is allogeneic hematopoietic cell transplantation (HCT). Outcome is superior if HCT is performed before leukemic transformation occurs. Several reports document survival in unmaintained remission beyond 10 years. The most recent analyses show reduced regimen-related mortality (less than 10% or even 5% at day 100) and progressively improved survival with both HLA-identical sibling and unrelated donors. The development of low/reduced-intensity conditioning regimens has contributed to the improved success rate and has allowed successful HCT in patients in their seventh and even eighth decade of life. We propose, therefore, that HCT should be offered to fit patients in these age groups and should be covered by their respective insurance carriers.
authors.affiliation: Fred Hutchinson Cancer Research Center and the University of Washington School of Medicine, Seattle, Washington. Electronic address: jdeeg@fredhutch.org.
authors.forename: H Joachim
authors.initials: HJ
authors.lastname: Deeg
authors.affiliation: Ottawa Hospital Research Institute at University of Ottawa, Ottawa, Canada.
authors.forename: Christopher
authors.initials: C
authors.lastname: Bredeson
authors.affiliation: National Marrow Donor Program, Minneapolis, Minnesota.
authors.forename: Stephanie
authors.initials: S
authors.lastname: Farnia
authors.affiliation: Hematology/Oncology Division, Massachusetts General Hospital, Boston, Massachusetts.
authors.forename: Karen
authors.initials: K
authors.lastname: Ballen
authors.affiliation: Medical Oncology and Hematology, Princess Margaret Cancer Center, University of Toronto, Toronto, Canada.
authors.forename: Vikas
authors.initials: V
authors.lastname: Gupta
authors.affiliation: Division of Hematology and Medical Oncology, Mayo Clinic Cancer Center, Scottsdale, Arizona.
authors.forename: Ruben A
authors.initials: RA
authors.lastname: Mesa
authors.affiliation: Department of Stem Cell Transplantation and Cellular Therapy, University of Texas MD Anderson Cancer Center, Houston, Texas.
authors.forename: Uday
authors.initials: U
authors.lastname: Popat
authors.affiliation: Division of Hematology/Oncology, Medical College of Wisconsin, Milwaukee, Wisconsin.
authors.forename: Parameswaran
authors.initials: P
authors.lastname: Hari
authors.affiliation: Division of Hematology/Oncology, Medical College of Wisconsin, Milwaukee, Wisconsin.
authors.forename: Wael
authors.initials: W
authors.lastname: Saber
authors.affiliation: Section of Hematology/Oncology, University of Manitoba, Winnipeg, Canada.
authors.forename: Matthew
authors.initials: M
authors.lastname: Seftel
authors.affiliation: Adult Bone Marrow Transplantation Service, Memorial Sloan Kettering Cancer Center, New York, New York; Department of Medicine, Weill Cornell Medical College, New York, New York.
authors.forename: Roni
authors.initials: R
authors.lastname: Tamari
authors.affiliation: Fred Hutchinson Cancer Research Center and the University of Washington School of Medicine, Seattle, Washington.
authors.forename: Effie W
authors.initials: EW
authors.lastname: Petersdorf
mesh_terms: D000208:Acute Disease; D000328:Adult; D000368:Aged; D018450:Disease Progression; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007938:Leukemia / Q000209:etiology / Q000401:mortality / Q000473:pathology / Q000517:prevention & control*; D008875:Middle Aged; D019653:Myeloablative Agonists / Q000627:therapeutic use*; D055728:Primary Myelofibrosis / Q000150:complications / Q000401:mortality / Q000473:pathology / Q000628:therapy*; D035781:Siblings; D016019:Survival Analysis; D019172:Transplantation Conditioning / Q000379:methods*; D014184:Transplantation, Homologous; D061349:Unrelated Donors
chemical_list: D019653:Myeloablative Agonists
keywords: Essential thrombocythemia; Hematopoietic cell transplantation; Myeloproliferative neoplasms; Polycythemia vera; Primary myelofibrosis