PMID- 22160038
OWN - NLM
STAT- MEDLINE
DCOM- 20120404
LR  - 20161021
IS  - 1520-4383 (Electronic)
IS  - 1520-4383 (Linking)
VI  - 2011
DP  - 2011
TI  - Management of myelofibrosis.
PG  - 222-30
LID - 10.1182/asheducation-2011.1.222 [doi]
AB  - Myelofibrosis (MF), either primary or arising from previous polycythemia vera 
      (PV) or essential thrombocythemia (ET), is the worst among the chronic 
      myeloproliferative neoplasms in terms of survival and quality of life. Patients 
      with MF have to face several clinical issues that, because of the poor 
      effectiveness of medical therapy, surgery or radiotherapy, represent largely 
      unmet clinical needs. Powerful risk stratification systems, applicable either at 
      diagnosis using the International Prognostic Scoring System (IPSS) or during the 
      variable course of illness using the Dynamic International Prognostic Scoring 
      System (DIPSS) and DIPSS Plus, allow recognition of categories of patients with 
      survival times ranging from decades to < 2 years. These scores are especially 
      important for therapeutic decisions that include allogeneic stem cell 
      transplantation (allogeneic SCT), the only curative approach that still carries a 
      nonnegligible risk of morbidity and mortality even with newest reduced intensity 
      conditioning (RIC) regimens. Discovery of JAK2V617F mutation prompted the 
      development of clinical trials using JAK2 inhibitors; these agents overall have 
      resulted in meaningful symptomatic improvement and reduction of splenomegaly that 
      were otherwise not achievable with conventional therapy. Intriguing differences 
      in the efficacy and tolerability of JAK2 inhibitors are being recognized, which 
      could lead to a nonoverlapping spectrum of activity/safety. Other agents that do 
      not directly target JAK2 and have shown symptomatic efficacy in MF are 
      represented by inhibitors of the mammalian target of rapamycin (mTOR) and histone 
      deacetylases (HDACs). Pomalidomide appears to be particularly active against 
      MF-associated anemia. However, because these agents are all poorly effective in 
      reducing the burden of mutated cells, further advancements are needed to move 
      from enhancing our ability to palliate the disease to arriving at an actual cure 
      for MF.
FAU - Vannucchi, Alessandro M
AU  - Vannucchi AM
AD  - Section of Hematology, Department of Critical Care, University of Florence, 
      Florence, Italy. amvannucchi@unifi.it
LA  - eng
PT  - Journal Article
PT  - Review
PL  - United States
TA  - Hematology Am Soc Hematol Educ Program
JT  - Hematology. American Society of Hematology. Education Program
JID - 100890099
SB  - IM
MH  - Humans
MH  - Primary Myelofibrosis/diagnosis/*therapy
MH  - Risk Factors
MH  - Stem Cell Transplantation
MH  - Transplantation, Homologous
EDAT- 2011/12/14 06:00
MHDA- 2012/04/05 06:00
CRDT- 2011/12/14 06:00
PHST- 2011/12/14 06:00 [entrez]
PHST- 2011/12/14 06:00 [pubmed]
PHST- 2012/04/05 06:00 [medline]
AID - 2011/1/222 [pii]
AID - 10.1182/asheducation-2011.1.222 [doi]
PST - ppublish
SO  - Hematology Am Soc Hematol Educ Program. 2011;2011:222-30. doi: 
      10.1182/asheducation-2011.1.222.