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.
