TY - JOUR
T1 - Hematopoietic Cell Transplantation as Curative Therapy for Patients with Myelofibrosis
T2 - Long-Term Success in all Age Groups
AU - Deeg, H. Joachim
AU - Bredeson, Christopher
AU - Farnia, Stephanie
AU - Ballen, Karen
AU - Gupta, Vikas
AU - Mesa, Ruben A.
AU - Popat, Uday
AU - Hari, Parameswaran
AU - Saber, Wael
AU - Seftel, Matthew
AU - Tamari, Roni
AU - Petersdorf, Effie W.
N1 - Publisher Copyright:
© 2015 American Society for Blood and Marrow Transplantation.
PY - 2015/11
Y1 - 2015/11
N2 - 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.
AB - 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.
KW - Essential thrombocythemia
KW - Hematopoietic cell transplantation
KW - Myeloproliferative neoplasms
KW - Polycythemia vera
KW - Primary myelofibrosis
UR - http://www.scopus.com/inward/record.url?scp=84943418147&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=84943418147&partnerID=8YFLogxK
U2 - 10.1016/j.bbmt.2015.09.005
DO - 10.1016/j.bbmt.2015.09.005
M3 - Review article
C2 - 26371371
AN - SCOPUS:84943418147
SN - 1083-8791
VL - 21
SP - 1883
EP - 1887
JO - Biology of Blood and Marrow Transplantation
JF - Biology of Blood and Marrow Transplantation
IS - 11
ER -