Allo-SCT for myelofibrosis: Reversing the chronic phase in the JAK inhibitor era? Journal Article


Authors: Tamari, R.; Mughal, T. I.; Rondelli, D.; Hasserjian, R.; Gupta, V.; Odenike, O.; Fauble, V.; Finazzi, G.; Pane, F.; Mascarenhas, J.; Prchal, J.; Giralt, S.; Hoffman, R.
Article Title: Allo-SCT for myelofibrosis: Reversing the chronic phase in the JAK inhibitor era?
Abstract: At present, allo-SCT is the only curative treatment for patients with myelofibrosis (MF). Unfortunately, a significant proportion of candidate patients are considered transplant ineligible due to their poor general condition and advanced age at the time of diagnosis. The approval of the first JAK inhibitor, ruxolitinib, for patients with advanced MF in 2011 has had a qualified impact on the treatment algorithm. The drug affords substantial improvement in MF-associated symptoms and splenomegaly but no major effect on the natural history. There has, therefore, been considerable support for assessing the drug's candidacy in the peritransplant period. The drug's precise impact on clinical outcome following allo-SCT is currently not known; nor are the drug's long-term efficacy and safety known. Considering the rarity of MF and the small proportion of patients who undergo allo-SCT, well designed collaborative efforts are required. In order to address some of the principal challenges, an expert panel of laboratory and clinical experts in this field was established, and an independent workshop held during the 54th American Society of Hematology Annual Meeting in New Orleans, USA on 6 December 2013, and the European Hematology Association's Annual Meeting in Milan, Italy on 13 June 2014. This document summarizes the results of these efforts. © 2015 Macmillan Publishers Limited.
Keywords: signal transduction; myelofibrosis; gene mutation; overall survival; busulfan; hydroxyurea; janus kinase inhibitor; allogeneic stem cell transplantation; histopathology; review; drug efficacy; drug safety; drug targeting; alpha interferon; cytarabine; outcome assessment; follow up; gene; anemia; mitogen activated protein kinase inhibitor; transcription initiation; thrombocytopenia; melphalan; practice guideline; cell differentiation; drug effect; chronic myeloid leukemia; dna methylation; history; acute graft versus host disease; chronic graft versus host disease; bone marrow biopsy; epigenetics; janus kinase; splenomegaly; acute myeloblastic leukemia; graft versus host reaction; medical society; natural killer cell; immunomodulation; antiinflammatory activity; clinical effectiveness; age distribution; bone marrow cell; immunosuppressive treatment; malignant transformation; cell activation; phenotypic variation; genetic marker; clofarabine; alemtuzumab; lymphocyte function; tet2 gene; thymocyte antibody; premedication; granulocytic sarcoma; randomized controlled trial (topic); peginterferon; workshop; myeloablative agent; ruxolitinib; tofacitinib; prognosis; human; priority journal; myeloid dendritic cell; health impact assessment; flucytosine arabinoside
Journal Title: Bone Marrow Transplantation
Volume: 50
Issue: 5
ISSN: 0268-3369
Publisher: Nature Publishing Group  
Date Published: 2015-05-01
Start Page: 628
End Page: 636
Language: English
DOI: 10.1038/bmt.2014.323
PROVIDER: scopus
PUBMED: 25665047
PMCID: PMC6394215
DOI/URL:
Notes: Export Date: 3 June 2015 -- Source: Scopus
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  1. Sergio Andres Giralt
    1050 Giralt
  2. Roni Tamari
    208 Tamari