Use and Effectiveness of Eltrombopag in a Tertiary Hospital

Background Eltrombopag is authorised by the EMA for adult chronic immune thrombocytopenic purpura (ITP) splenectomised patients who are refractory to other treatments and as second-line treatment for non-splenectomised patients for whom surgery is contraindicated. Eltrombopag was effective in 59% of patients in a randomised controlled trial (Bussel, 2009). Purpose To determine whether eltrombopag is prescribed according to the approved indications. To observe the effect on platelet levels. Materials and methods Observational study. The authors included patients treated with eltrombopag from 01/01/2011 to 31/08/2011. Variables analysed: demographics, diagnosis, previous treatments, duration, rescue medication, changes in platelet levels, and reason for suspension (where applicable). Results Seven patients were treated with eltrombopag. Median age: 65 years, 4 males. Six had ITP and 1 had multifactorial essential thrombocytopenia. All patients with ITP had received first-line treatment with corticosteroids and immunoglobulins and were refractory to at least 2 second-line treatments, as follows: immunosuppressants (3 patients), rituximab (3), Vinca alkaloids (2), tranexamic acid (3), and romiplostim (2). One patient with ITP was splenectomised, while 5 were not (old age (3), multiple comorbidities, refusal (1 each)). Four of the 7 patients discontinued treatment before the end of the study (median duration, 87 days), while 3 continued with treatment (median interval from initiation, 46 days). The 3 patients who continued with treatment maintained increased platelet levels from baseline (>50 x 103/μL). Of the 4 who stopped treatment, 3 did not have increased platelet levels at any time during the study, while 1, despite reaching and maintaining platelet levels, discontinued treatment due to uncontrolled bleeding events. All non-responders required rescue with immunoglobulins. Conclusions Eltrombopag was prescribed according to the approved indication in 6 out of 7 patients and was effective in half of the patients with ITP. Despite our small study population, the percentage of responders was similar to that found by Bussel et al.