Efficacy and Safety of IQYMUNE®, a Ten Percent Intravenous Immunoglobulin in Adult Patients With Chronic, Primary Immune Thrombocytopenia

Francesco Rodeghiero; Dariusz Woszczyk; Borhane Slama; Anait Melikyan; Jean-Francois Viallard; Rabye Ouaja; Ousmane Alfa Cisse; Alain Sadoun; Abdulgabar Salama

doi:10.14740/jh385w

Efficacy and Safety of IQYMUNE®, a Ten Percent Intravenous Immunoglobulin in Adult Patients With Chronic, Primary Immune Thrombocytopenia

J Hematol. 2018 Sep;7(3):87-95. doi: 10.14740/jh385w. Epub 2018 Sep 1.

Authors

Francesco Rodeghiero¹, Dariusz Woszczyk², Borhane Slama³, Anait Melikyan⁴, Jean-Francois Viallard⁵, Rabye Ouaja⁶, Ousmane Alfa Cisse⁶, Alain Sadoun⁶, Abdulgabar Salama⁷

Affiliations

¹ Hematology Project Foundation, Vicenza-Affiliated to the Hematology Department, S. Bortolo Hospital, Vicenza, Italy.
² Hematology Department, University of Opole, Opole Provincial Hospital, Opole, Poland.
³ Hematology Department, Henri Duffaut Hospital, Avignon, France.
⁴ National Research Center for Hematology, Moscow, Russia.
⁵ Internal Medicine Department, Haut-Leveque Hospital, Pessac, France.
⁶ Medical Unit, LFB SA, Les Ulis, France.
⁷ Institute for Transfusion Medicine, Charite Campus Virchow-Klinikum, Berlin, Germany.

PMID: 32300420
PMCID: PMC7155829
DOI: 10.14740/jh385w

Abstract

Background: Intravenous immunoglobulin (IVIG) IQYMUNE® is a highly purified 10% IVIG that was assessed using the new stringent definition of response described in the revised guideline on the clinical investigation of IVIG. The efficacy and the safety of IQYMUNE® were investigated in adult patients with chronic primary immune thrombocytopenia (ITP).

Methods: In this phase III multinational, multicentre, prospective, uncontrolled, open-label, single-arm study, adult patients with a baseline platelet count < 30 × 10⁹/L were treated with IVIG 10% at a dose of 2 g/kg body weight administered over 2 consecutive days. The primary endpoint was Response over the study period and was defined according to the recent and most stringent European Medicines Agency guidelines (platelet count ≥ 30 × 10⁹/L and a ≥ 2-fold increase from baseline, no new bleeding, and no concomitant treatment with drugs that affect platelet count and/or induce bleeding cessation).

Results: Thirty-eight patients were enrolled; 73 infusions were administered (38 on Day 1 and 35 on Day 2). Response was reached by 24 patients corresponding to 63.2% of patients in the full analysis set (95% CI: 46.0; 78.2) and 68.6% of patients in the per-protocol set (95% CI: 50.7; 83.1). The median time to Response was 1 day. The median duration of Response was 13.5 days. Reasons for non-response were failure to reach the required platelet count (n = 12), a new bleeding event (n = 1), and forbidden medication use (n = 1). Among the 23 patients with a baseline platelet count ≤ 20 × 10⁹/L, 19 patients (82.6%) achieved a platelet count ≥ 50 × 10⁹/L at least once before Day 5 (previous European Medicines Agency definition of response). Treatment was well tolerated even in patients with a high flow rate (≥ 6 mL/kg/h in 40% of patients). Headache (34.2%), pyrexia (15.8%), and creatinine renal clearance decrease, including one case of decrease in glomerular filtration rate (10.5%) were the most frequently reported drug-related adverse events.

Conclusions: Administration of IQYMUNE® for 2 consecutive days at a dose of 2 g/kg was safe and efficacious. These results support the treatment of adult patients with chronic ITP with IQYMUNE®.

Keywords: Bleeding assessment; European Medicines Agency guidelines; High dose; Immune thrombocytopenia; Infusion rate; Intravenous immunoglobulin; Platelet count; Response.