An open-label, multicenter study evaluating the efficacy and safety of a new 10% liquid intravenous immunoglobulin in patients with primary immune thrombocytopenia (ITP)

BACKGROUND AND AIMS This single-arm, open-label, multicenter study evaluated the efficacy and safety of a new 10% liquid intravenous immunoglobulin (Intragam® 10 NF), in patients with primary immune thrombocytopenia (ITP). METHODS Nineteen patients]18 years of age with a platelet count of <50×10⁹/L received 2 g/kg 10% liquid intravenous immunoglobulin (Intragam® 10 NF) over 2 days. The primary endpoint was the proportion of patients with a platelet response (≥50×10⁹/L and an increment≥30×10⁹/L over baseline within 7 days). RESULTS In the per protocol analysis, 88.2% of patients (n=15; 95% Confidence Intervals (CI): 63.6%, 98.5%) achieved the primary endpoint. The median duration of platelet response was 17.2 days and the median time to first platelet response was 4 days. External bleeding events decreased in number and intensity from Day 2. There were no episodes of internal bleeding. All patients experienced at least one treatment emergent adverse event (TEAE). The majority of TEAEs were mild or moderate intensity with no study withdrawals due to TEAEs and no deaths. Headache was the most common TEAE, attributed to treatment in 68.4% of patients. There were four severe TEAEs with a causal relationship to the study drug: three cases of headache and one aseptic meningitis syndrome (AMS). CONCLUSION The majority of ITP patients treated with 10% liquid intravenous immunoglobulin (Intragam® 10 NF) achieved a rapid platelet response accompanied by a decrease in bleeding events. The efficacy and safety profile of 10% liquid intravenous immunoglobulin (Intragam® 10 NF) is similar to other studies with Intragam P, with the added reassurance of a third dedicated virus removal step and the convenience of a 10% formulation.