Treatment of immune thrombocytopenia (ITP) with Eltrombopag – results of the 3 rd interim analysis of the German non-interventional trial RISA. Focussing on steroid-pretreatment and fatigue

Objective Eltrombopag (EPAG) is a thrombopoietin-receptor agonist, which is proved to be effective and safe in the treatment of immune thrombocytopenia (ITP).

Material and Methods In the planned interim analysis of the study RISA we evaluated data from the routine treatment with EPAG over two years. Patients were included, if they received at least one dose of EPAG and completed one post baseline assessment. Fatigue was assessed at baseline and during the study using the FACIT-F questionnaire.

Results 210 patients were included in the analysis. Mean±SD age was 63.1±17.4 years, median (range) duration of ITP was 5.6 (0.0– 44.9) years. Comorbidity was present in 81.4 % of all cases. 47.6 % were male, median platelet count at baseline was 33.5x109/L. In 37.6 %, bleeding complications occurred within 12 months before baseline. In 10 %, splenectomy had been performed. 85.2 % of the patients were pretreated pharmacologically. 46.2 % received prednisolone, 9 % prednisone and 24.3 % dexamethasone. 14.8 % of all patients received corticosteroids for longer than 6 months as first-line therapy. In another 10.0 %, the total duration of corticosteroid therapy over first and second line of treatment exceeded 6 months. The longest treatment with corticosteroids lasted 108 months. 20.0 % of all patients received immune globulins, 2.9 % Rituximab.

Mean±SD daily dose of EPAG was 45.1±14.4 mg. Within the first month of treatment, 75 % of the patients responded in terms of raising the thrombocyte counts above 50x109/L. After 24 months, response rate was 89 %. Initially mean±SD FACIT-Fatigue score was 36.3±11.1. It did not change significantly during the two-year observation period. 31 % of all evaluable patients had severe fatigue (FACIT-F score ≤ 30). EPAG was generally well tolerated. No new signals concerning the safety or tolerability of the drug occurred.

Conclusion This 3 rd interim analysis confirmed, that treatment of ITP with EPAG is an effective and safe option. We found that severe fatigue is present in ITP patients to a similar extent as in cancer. A clinically significant improvement in fatigue could not be found in our study, but this finding is preliminary. A quarter of patients received corticosteroids for longer than 6 months, which tremendously exceeds the maximum duration of steroid treatment, as being recommended in evidence-based guidelines.

Publication History

Article published online:
18 June 2021