Eltrombopag: A Review in Paediatric Chronic Immune Thrombocytopenia

Drugs. 2016 May;76(8):869-78. doi: 10.1007/s40265-016-0581-4.


Eltrombopag (Promacta(®); Revolade(®)) is an orally active thrombopoietin receptor agonist recently approved in the USA and the EU for use in paediatric patients aged ≥1 year with chronic immune thrombocytopenia (ITP) who have had an insufficient response or are refractory to other ITP treatments (e.g. corticosteroids, immunoglobulins or splenectomy). The efficacy of 7 or 13 weeks' therapy with oral eltrombopag (up to 75 mg/day) was compared with that of placebo in patients aged 1-17 years with previously treated chronic ITP in randomized, double-blind, multicentre phase II and III trials (PETIT and PETIT-2). In these trials, the platelet response rate (primary endpoint of PETIT) and the sustained platelet response rate (primary endpoint of PETIT-2) were significantly higher with eltrombopag than with placebo. A clinical benefit was shown by a reduction in the need for rescue therapy with eltrombopag versus placebo in both trials and a reduction of clinically significant bleeding in PETIT. During longer-term therapy (open-label treatment period for ≥24 weeks), eltrombopag maintained platelet counts above 50 × 10(9)/L in the majority of patients and approximately one-half of patients were able to reduce or discontinue concurrent ITP drugs. Eltrombopag was generally well tolerated. Current evidence suggests that eltrombopag is a valuable addition to the limited treatment options available for the management of chronic ITP in paediatric patients with an inadequate response to first-line therapies.

Publication types

  • Review

MeSH terms

  • Adolescent
  • Benzoates / therapeutic use*
  • Blood Platelets / drug effects
  • Child
  • Child, Preschool
  • Chronic Disease
  • Clinical Trials, Phase II as Topic
  • Clinical Trials, Phase III as Topic
  • Double-Blind Method
  • Hemorrhage / prevention & control
  • Humans
  • Hydrazines / therapeutic use*
  • Infant
  • Multicenter Studies as Topic
  • Platelet Count
  • Purpura, Thrombocytopenic, Idiopathic / drug therapy*
  • Purpura, Thrombocytopenic, Idiopathic / immunology
  • Pyrazoles / therapeutic use*
  • Randomized Controlled Trials as Topic
  • Receptors, Thrombopoietin / agonists*


  • Benzoates
  • Hydrazines
  • Pyrazoles
  • Receptors, Thrombopoietin
  • MPL protein, human
  • eltrombopag