Table 1.

Investigational medical therapies for the BCR-ABL–negative myeloproliferative disorders.

DiseaseAgentClass/type of drugRoutePhaseReference
Abbreviations: PV, polycythemia vera; ET, essential thrombocythemia; PMF, primary myelofibrosis; MF, myelofibrosis; IV, intravenous; SQ, subcutaneous; TGF, tumor growth factor; VEGF, vascular endothelial growth factor 
  Immunomodulatory agents    
PV/ET Pegylated interferon-2a Immunomodulatory agent SQ 22  
PMF/post-ET/PV MF Lenalidomide + prednisone Immunomodulatory agent Oral 43  
PMF/post-ET/PV MF CC-4047 ± prednisone Immunomodulatory agent Oral Ongoing 
  Targeting the stromal reaction    
PMF/post-ET/PV MF GC-1008 Pan-specific human IV 44  
  anti–TGF-β antibody    
PMF/post-ET/PV MF Bevacizumab Anti-VEGF monoclonal antibody IV 45  
  DNA hypomethylating agents    
PMF/post-ET/PV MF Azacitidine Hypomethylating agent SQ Ongoing 
PMF/post-ET/PV MF Decitabine Hypomethylating agent IV Ongoing 
  Targeting apoptosis agents    
PMF/post-ET/PV MF Bortezomib Proteasome inhibitor IV 46  
  Kinase inhibitory agents    
PMF/post-ET/PV MF Dasatinib Kinase inhibitor Oral 47  
  JAK2 inhibitors in development    
PMF/post-ET/PV MF (at first) TG 101209 JAK2 inhibitor Oral 1/2 37  
PMF/post-ET/PV MF (at first) Go6976 FLT3-JAK2 inhibitor Unknown Preclinical 38  
PMF/post-ET/PV MF (at first) Erlotinib JAK2 inhibitor Oral 1/2 39  
PMF/post-ET/PV MF (at first) MK 0457 Aurora kinase inhibitor IV 1/2 40  
PMF/post-ET/PV MF (at first) CEP-701 FLT3-JAK2 inhibitor Oral 1/2 41  
DiseaseAgentClass/type of drugRoutePhaseReference
Abbreviations: PV, polycythemia vera; ET, essential thrombocythemia; PMF, primary myelofibrosis; MF, myelofibrosis; IV, intravenous; SQ, subcutaneous; TGF, tumor growth factor; VEGF, vascular endothelial growth factor 
  Immunomodulatory agents    
PV/ET Pegylated interferon-2a Immunomodulatory agent SQ 22  
PMF/post-ET/PV MF Lenalidomide + prednisone Immunomodulatory agent Oral 43  
PMF/post-ET/PV MF CC-4047 ± prednisone Immunomodulatory agent Oral Ongoing 
  Targeting the stromal reaction    
PMF/post-ET/PV MF GC-1008 Pan-specific human IV 44  
  anti–TGF-β antibody    
PMF/post-ET/PV MF Bevacizumab Anti-VEGF monoclonal antibody IV 45  
  DNA hypomethylating agents    
PMF/post-ET/PV MF Azacitidine Hypomethylating agent SQ Ongoing 
PMF/post-ET/PV MF Decitabine Hypomethylating agent IV Ongoing 
  Targeting apoptosis agents    
PMF/post-ET/PV MF Bortezomib Proteasome inhibitor IV 46  
  Kinase inhibitory agents    
PMF/post-ET/PV MF Dasatinib Kinase inhibitor Oral 47  
  JAK2 inhibitors in development    
PMF/post-ET/PV MF (at first) TG 101209 JAK2 inhibitor Oral 1/2 37  
PMF/post-ET/PV MF (at first) Go6976 FLT3-JAK2 inhibitor Unknown Preclinical 38  
PMF/post-ET/PV MF (at first) Erlotinib JAK2 inhibitor Oral 1/2 39  
PMF/post-ET/PV MF (at first) MK 0457 Aurora kinase inhibitor IV 1/2 40  
PMF/post-ET/PV MF (at first) CEP-701 FLT3-JAK2 inhibitor Oral 1/2 41  
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