Efficacy and safety of a new intravenous immunoglobulin (Panzyga®) in chronic immune thrombocytopenia

Objectives To assess the efficacy and safety of intravenous immunoglobulin (IVIG) 10% (Panzyga((R))), a novel human normal IVIG 10%, in patients with chronic immune thrombocytopenia (ITP). Background First-line treatment options in ITP include IVIGs. Methods In this prospective, open-label, non-controlled, multicentre, phase III study, patients received a daily dose of IVIG 10% (1 g kg(-1) body weight) for two consecutive days. The primary end point was clinical response rate; secondary end points included alternate response definitions, time to response, response duration, platelet counts, regression of bleeding and safety. Results Forty patients were enrolled (57 center dot 5% male, mean age 36 center dot 7 years); the full analysis set comprised 36 patients. A clinical response was seen for 29 of 36 patients (80 center dot 6%). Median time to response and response duration was 2 days and 14 days, respectively. IVIG 10% was well tolerated at a maximum infusion rate of 8 mg (kg min)(-1) in all but one patient; adverse events were mainly mild to moderate in severity, and the most frequent was headache (42 center dot 5%). Conclusion IVIG 10% is well tolerated even at a high infusion speed and induces a rapid platelet count increase, thus decreasing the bleeding rate and the severity of bleeding events. Trial registry: record: NCT01349790.