Eltrombopag.
The current concepts and the management of ITP have significantly changed in the past decade. Decreased use of cytotoxic therapy and the introduction of new selective modalities of drug such as TPO-r mimetics are the landmarks of this change. Discovered in the middle of last decade, followed by experiments in mice and then approved in humans, Eltrombopag is the first TPO-r mimetic available. It has been used and validated in several clinical studies in different etiologies of thrombocytopenia, including primary ITP (chronic Immune ThrombocytoPenia) and secondary ITP, due to hepatitis C and more recently in bone marrow failure as myelodysplastic syndromes. Good tolerability and low side effects are the strengths of this drug, contrasted with issues regarding administration (it must be taken every day apart from specific meals containing high levels of calcium, which leads to problems with compliance). We review the first clinical studies with this agent, emphasizing the significant findings.
Division of Pediatric Hematology-Oncology, Department of Pediatrics in Obstetrics and Gynecology and in Medicine, Weill Cornell Medical College, New York Presbyterian Hospital, Weill Cornell Medical Center, New York, NY 10044, USA. jbussel@med.cornell.edu
James B
JB
Bussel
Mariana P
MP
Pinheiro
D000818:Animals; D001565:Benzoates / Q000627:therapeutic use*; D002986:Clinical Trials as Topic; D006801:Humans; D006834:Hydrazines / Q000627:therapeutic use*; D051379:Mice; D009369:Neoplasms / Q000150:complications* / Q000628:therapy; D011720:Pyrazoles / Q000627:therapeutic use*; D013921:Thrombocytopenia / Q000188:drug therapy* / Q000209:etiology
D001565:Benzoates; D006834:Hydrazines; D011720:Pyrazoles; C520809:eltrombopag