Hematopoietic Cell Transplantation as Curative Therapy for Patients with Myelofibrosis: Long-Term Success in all Age Groups

H. Joachim Deeg, Christopher Bredeson, Stephanie Farnia, Karen Ballen, Vikas Gupta, Ruben A. Mesa, Uday Popat, Parameswaran Hari, Wael Saber, Matthew Seftel, Roni Tamari, Effie W. Petersdorf

Research output: Contribution to journalReview articlepeer-review

25 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)1883-1887
Number of pages5
JournalBiology of Blood and Marrow Transplantation
Issue number11
StatePublished - Nov 2015


  • Essential thrombocythemia
  • Hematopoietic cell transplantation
  • Myeloproliferative neoplasms
  • Polycythemia vera
  • Primary myelofibrosis

ASJC Scopus subject areas

  • Hematology
  • Transplantation


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