Efficacy and safety of BT595 (10% human intravenous immunoglobulin) in adult patients with chronic immune thrombocytopenia

Purpose This trial investigated the efficacy and safety of the new 10% human intravenous immunoglobulin (IVIg) BT595 (Yimmugo (R)). Methods Adult patients with chronic immune thrombocytopenia (ITP) received a total dose of 2 g/kg body weight (bw) IVIg either over 2 or 5 days. Results Response as defined by the European Medicines Agency (EMA) was achieved in 18 of 34 patients (52.9%) in the full analysis set (FAS), with a complete response in 11 patients (32.4%). The median time to response was 1.0 days (range 1-4); the median duration was 28.0 days. In a subgroup with a baseline platelet count < 20*10(9)/L evaluated according to FDA criteria, a platelet response >= 50*10(9)/L was achieved in 18 of 19 patients at day 8. No fatal case occured. One serious treatment-emergent adverse event (TEAE) (anaemia, not related) was reported (2.9%). The most frequent infusional adverse drug reaction (ADR) was headache, which was reported for 14.7% of all patients. All other infusional ADRs (pyrexia, [intravascular] haemolysis, skin reaction, tinnitus, and Coombs test positive) occurred in only one patient (2.9%). Premedication was administered only once. The 5-day schedule showed less side effects with similar efficacy. Conclusion The benefit-risk profile of BT595 is favourable.