Allogeneic stem cell transplant vs.Janus kinase inhibition in the treatment of primary myelofibrosis or myelofibrosis after essential thrombocythemia or polycythemia vera
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Allogeneic stem cell transplant vs.Janus kinase inhibition in the treatment of primary myelofibrosis or myelofibrosis after essential thrombocythemia or polycythemia vera. / Alchalby, Haefaa; Kröger, Nicolaus.
In: CL LYMPH MYELOM LEUK, Vol. 14 Suppl, 01.09.2014, p. S36-41.Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review
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TY - JOUR
T1 - Allogeneic stem cell transplant vs.Janus kinase inhibition in the treatment of primary myelofibrosis or myelofibrosis after essential thrombocythemia or polycythemia vera
AU - Alchalby, Haefaa
AU - Kröger, Nicolaus
N1 - Copyright © 2014 Elsevier Inc. All rights reserved.
PY - 2014/9/1
Y1 - 2014/9/1
N2 - Primary myelofibrosis is one of the Philadelphia chromosome-negative myeloproliferative neoplasms and is the member of that group with the worst survival and the most significant limitations in quality of life. Hepatosplenomegaly due to extramedullary hematopoiesis, constitutional symptoms, and cytopenias are the main manifestations. The natural history is highly variable, and up to 30% of patients can experience acceleration to acute myelogenous leukemia. Conventional therapy is only palliative and not always effective. However, huge advances have been achieved in the past 2 decades toward a better understanding of the pathogenesis of this disease, as well as improved management. Powerful risk stratification systems are now available and can reliably separate the patients into different prognostic categories to aid clinical management. Allogeneic stem cell transplant can offer cure but is still not universally applicable owing to the treatment-related mortality and toxicity. Nevertheless, outcomes of transplant are improving, owing to the introduction of reduced-intensity conditioning regimens and the optimization of remission monitoring techniques and relapse prevention strategies. The discovery of the V617F mutation of JAK2 (Janus kinase 2) and some other molecular aberrations has shed more light on the molecular pathogenesis of the disease and has led to the introduction of novel therapies such as JAK2 inhibitors. In fact, JAK inhibitors have shown promising symptomatic efficacy, and the JAK inhibitor ruxolitinib has also shown a potential survival benefit. Future effort should be made to combine allogeneic stem cell transplant with JAK inhibition.
AB - Primary myelofibrosis is one of the Philadelphia chromosome-negative myeloproliferative neoplasms and is the member of that group with the worst survival and the most significant limitations in quality of life. Hepatosplenomegaly due to extramedullary hematopoiesis, constitutional symptoms, and cytopenias are the main manifestations. The natural history is highly variable, and up to 30% of patients can experience acceleration to acute myelogenous leukemia. Conventional therapy is only palliative and not always effective. However, huge advances have been achieved in the past 2 decades toward a better understanding of the pathogenesis of this disease, as well as improved management. Powerful risk stratification systems are now available and can reliably separate the patients into different prognostic categories to aid clinical management. Allogeneic stem cell transplant can offer cure but is still not universally applicable owing to the treatment-related mortality and toxicity. Nevertheless, outcomes of transplant are improving, owing to the introduction of reduced-intensity conditioning regimens and the optimization of remission monitoring techniques and relapse prevention strategies. The discovery of the V617F mutation of JAK2 (Janus kinase 2) and some other molecular aberrations has shed more light on the molecular pathogenesis of the disease and has led to the introduction of novel therapies such as JAK2 inhibitors. In fact, JAK inhibitors have shown promising symptomatic efficacy, and the JAK inhibitor ruxolitinib has also shown a potential survival benefit. Future effort should be made to combine allogeneic stem cell transplant with JAK inhibition.
U2 - 10.1016/j.clml.2014.06.012
DO - 10.1016/j.clml.2014.06.012
M3 - SCORING: Journal article
C2 - 25486953
VL - 14 Suppl
SP - S36-41
JO - CL LYMPH MYELOM LEUK
JF - CL LYMPH MYELOM LEUK
SN - 2152-2650
ER -