New-generation Drugs That Stimulate Platelet Production in Chronic Immune Thrombocytopenic Purpura

Lancet. 2009 May 2;373(9674):1562-9. doi: 10.1016/S0140-6736(09)60255-5. Epub 2009 Mar 25.

Abstract

Idiopathic thrombocytopenic purpura is an acquired disease characterised by a low platelet count. Development of autoantibodies is a main cause of the disease. Although many patients have few symptoms, life-threatening bleeding can arise and hence, when platelet counts fall to unacceptable values treatment should be initiated. However, conventional immunosuppressive approaches can fail, perhaps because of the heterogeneous nature of the disease. Newly developed agents that increase platelet production by stimulating megakaryocytes-such as drugs that bind to the thrombopoietin receptor c-MPL-offer an alternative treatment strategy. Although initial thrombopoietin analogues caused adverse immune reactions, second-generation thrombopoietin-receptor agonists that are in late-stage clinical development seem promising. In particular, eltrombopag and romiplostim safely increase and maintain platelet production in patients with refractory disease. However, long-term side-effects are being assessed and the exact role of these agents in the overall treatment strategy of chronic idiopathic thrombocytopenic purpura remains to be established.

Publication types

  • Review

MeSH terms

  • Benzoates / chemistry
  • Benzoates / pharmacology
  • Benzoates / therapeutic use*
  • Carrier Proteins / chemistry
  • Carrier Proteins / pharmacology
  • Carrier Proteins / therapeutic use*
  • Chronic Disease
  • Drug Approval
  • Hemorrhage / etiology
  • Humans
  • Hydrazines / chemistry
  • Hydrazines / pharmacology
  • Hydrazines / therapeutic use*
  • Megakaryocytes / drug effects
  • Methacrylates / pharmacology
  • Methacrylates / therapeutic use
  • Patient Selection
  • Platelet Count
  • Purpura, Thrombocytopenic, Idiopathic / blood
  • Purpura, Thrombocytopenic, Idiopathic / drug therapy*
  • Purpura, Thrombocytopenic, Idiopathic / etiology
  • Pyrazoles / chemistry
  • Pyrazoles / pharmacology
  • Pyrazoles / therapeutic use*
  • Receptors, Fc / chemistry
  • Receptors, Fc / therapeutic use*
  • Receptors, Thrombopoietin / agonists*
  • Recombinant Fusion Proteins
  • Safety
  • Thiazoles / pharmacology
  • Thiazoles / therapeutic use
  • Thiophenes / pharmacology
  • Thiophenes / therapeutic use
  • Thrombopoiesis / drug effects*
  • Thrombopoiesis / physiology
  • Thrombopoietin
  • Treatment Outcome
  • United States
  • United States Food and Drug Administration

Substances

  • Benzoates
  • Carrier Proteins
  • Hydrazines
  • Methacrylates
  • NIP-004
  • Pyrazoles
  • Receptors, Fc
  • Receptors, Thrombopoietin
  • Recombinant Fusion Proteins
  • Thiazoles
  • Thiophenes
  • butyzamide
  • MPL protein, human
  • Thrombopoietin
  • romiplostim
  • eltrombopag