Drugs

, Volume 76, Issue 8, pp 869–878 | Cite as

Eltrombopag: A Review in Paediatric Chronic Immune Thrombocytopenia

  • Celeste B. Burness
  • Gillian M. Keating
  • Karly P. Garnock-Jones
Adis Drug Evaluation

Abstract

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 × 109/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.

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Copyright information

© Springer International Publishing Switzerland 2016

Authors and Affiliations

  • Celeste B. Burness
    • 1
  • Gillian M. Keating
    • 1
  • Karly P. Garnock-Jones
    • 1
  1. 1.SpringerAucklandNew Zealand

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