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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During the peer review process, the manufacturer of eltrombopag was also offered an opportunity to review this article. Changes resulting from comments received were made on the basis of scientific and editorial merit.
The preparation of this review was not supported by any external funding.
Conflicts of interest
Celeste B. Burness, Gillian Keating and Karly P. Garnock-Jones are salaried employee of Adis/Springer, are responsible for the article content and declare no relevant conflicts of interest.
The manuscript was reviewed by: D. B. Cines, Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA; J Despotovic, Texas Children’s Hematology Center, Baylor College of Medicine, Houston, TX, USA; J. Grainger, Faculty of Medical and Human Sciences, University of Manchester; Royal Manchester Children’s Hospital, Central Manchester University Hospitals NHS Foundation Trust, Manchester Academic Health Science Centre, Manchester, UK; F. Rodeghiero, Hematology Department, S Bortolo Hospital, Vincenza, Italy, J. Sevilla, Hemato-Oncología Pediátrica Hospital Infantil Universitario Niño Jesús, Madrid, Spain.
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Burness, C.B., Keating, G.M. & Garnock-Jones, K.P. Eltrombopag: A Review in Paediatric Chronic Immune Thrombocytopenia. Drugs 76, 869–878 (2016). https://doi.org/10.1007/s40265-016-0581-4
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