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Hematopoietic Cell Transplantation as Curative Therapy for Patients with Myelofibrosis: Long-Term Success in all Age Groups. Biol Blood Marrow Transplant 2015 Nov;21(11):1883-7

Date

09/16/2015

Pubmed ID

26371371

Pubmed Central ID

PMC4604067

DOI

10.1016/j.bbmt.2015.09.005

Scopus ID

2-s2.0-84943418147 (requires institutional sign-in at Scopus site)   36 Citations

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.

Author List

Deeg HJ, Bredeson C, Farnia S, Ballen K, Gupta V, Mesa RA, Popat U, Hari P, Saber W, Seftel M, Tamari R, Petersdorf EW

Authors

Parameswaran Hari MD Adjunct Professor in the Medicine department at Medical College of Wisconsin
Wael Saber MD, MS Professor in the Medicine department at Medical College of Wisconsin




MESH terms used to index this publication - Major topics in bold

Acute Disease
Adult
Aged
Disease Progression
Hematopoietic Stem Cell Transplantation
Humans
Leukemia
Middle Aged
Myeloablative Agonists
Primary Myelofibrosis
Siblings
Survival Analysis
Transplantation Conditioning
Transplantation, Homologous
Unrelated Donors