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Allogeneic hematopoietic cell transplantation for myelofibrosis in the era of JAK inhibitors. Blood 2012 Aug 16;120(7):1367-79 PMID: 22700718 PMCID: PMC5800543

Pubmed ID





The discovery of JAK2617F mutation paved the way for the development of small molecule inhibitors of JAK1/2 resulting in first approved JAK1/2 inhibitor, ruxolitinib, for the treatment of patients with myelofibrosis (MF). Although JAK1/2 inhibitor therapy is effective in decreasing the burden of symptoms associated with splenomegaly and MF-related constitutional symptoms, it is neither curative nor effective in reducing the risk of leukemic transformation. Presently, allogeneic hematopoietic cell transplantation (HCT) is the only curative therapy for MF. A significant risk of regimen-related toxicities, graft failure, and GVHD are major barriers to the success of HCT in MF. Because of significant HCT-associated morbidity and mortality, divergent opinions regarding its appropriate role in this clinical situation have emerged. In this review, the risk-benefit ratios of modern drug therapy compared with HCT in MF patients are analyzed. A risk-adapted approach individualized to each patient's biologic characteristics and comorbidities is described, which is currently warranted in determining optimal treatment strategies for patients with MF. Inclusion of JAK1/2 inhibitor therapy in future transplant conditioning regimens may provide an opportunity to overcome some of these barriers, resulting in greater success with HCT for MF patients.

Author List

Gupta V, Hari P, Hoffman R


Parameswaran Hari MD Chief, Professor in the Medicine department at Medical College of Wisconsin


2-s2.0-84865174824   58 Citations

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

Hematopoietic Stem Cell Transplantation
Janus Kinase 1
Primary Myelofibrosis
Protein Kinase Inhibitors
Transplantation, Homologous
Treatment Outcome
jenkins-FCD Prod-331 a335b1a6d1e9c32173c9534e6f6ff51494143916