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

Abstract

Purpose: This trial investigated the efficacy and safety of the new 10% human intravenous immunoglobulin (IVIg) BT595 (Yimmugo®).

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*10⁹ /L evaluated according to FDA criteria, a platelet response ≥50*10⁹ /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.

Trial registration number: Eudra CT Number 2015-003653-17, ClinicalTrials.gov NCT02859909.

Keywords: BT595; ITP; chronic immune thrombocytopenia; phase III trial; pivotal.