title: Eltrombopag: A Review in Paediatric Chronic Immune Thrombocytopenia.
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 × 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.
authors.affiliation: Springer, Private Bag 65901, Mairangi Bay, 0754, Auckland, New Zealand. demail@springer.com.
authors.forename: Celeste B
authors.initials: CB
authors.lastname: Burness
authors.affiliation: Springer, Private Bag 65901, Mairangi Bay, 0754, Auckland, New Zealand.
authors.forename: Gillian M
authors.initials: GM
authors.lastname: Keating
authors.affiliation: Springer, Private Bag 65901, Mairangi Bay, 0754, Auckland, New Zealand.
authors.forename: Karly P
authors.initials: KP
authors.lastname: Garnock-Jones
mesh_terms: D000293:Adolescent; D001565:Benzoates / Q000627:therapeutic use*; D001792:Blood Platelets / Q000187:drug effects; D002648:Child; D002675:Child, Preschool; D002908:Chronic Disease; D017322:Clinical Trials, Phase II as Topic; D017326:Clinical Trials, Phase III as Topic; D004311:Double-Blind Method; D006470:Hemorrhage / Q000517:prevention & control; D006801:Humans; D006834:Hydrazines / Q000627:therapeutic use*; D007223:Infant; D015337:Multicenter Studies as Topic; D010976:Platelet Count; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000188:drug therapy* / Q000276:immunology; D011720:Pyrazoles / Q000627:therapeutic use*; D016032:Randomized Controlled Trials as Topic; D053628:Receptors, Thrombopoietin / Q000819:agonists*
chemical_list: D001565:Benzoates; D006834:Hydrazines; D011720:Pyrazoles; D053628:Receptors, Thrombopoietin; C082218:MPL protein, human; C520809:eltrombopag