This is one of two parallel randomised, multicentre, placebo-controlled trials of eltrombopag in children with chronic immune thrombocytopenia. The other study, the phase II PETIT trial (n=15 dose finding and 67 randomised controlled phase) was published in The Lancet Haematology. These studies enrolled similar patient cohorts; most patients had received more than two previous treatments, and few had undergone splenectomy.
According to a commentary, PETIT and PETIT2 provide key data to help to inform decision making for paediatric immune thrombocytopenia, and with these data, the FDA has now approved eltrombopag for the treatment of children aged 6 years or older who have chronic immune thrombocytopenia with an insufficient response to corticosteroids, intravenous immunoglobulins, or splenectomy. Currently eltrombopag is not licensed in UK for use in children.
The commentators question how, with so many treatment choices and so few direct comparisons, should clinicians and patients select treatment. They note that adequately powered RCTs are difficult to do, especially in view of individual preference, cost, differences in routes of administration, and surgical considerations of splenectomy. They caution that although the PETIT and PETIT2 studies show efficacy of eltrombopag against placebo, further data are needed to establish its effectiveness in clinical practice where thought must be given to cost, availability of more curative options, and patient adherence to long-term daily medication. They add that continued long-term safety data are needed because paediatric patients with chronic refractory immune thrombocytopenia might need years of pharmacological therapy.