A Post-Authorization Safety Surveillance Study to Report Clinical Experience with Purified Factor IX Concentrate in Pediatric Patients with Hemophilia B

Abstract

Zoran Igrutinović,1,2 Hélène Louise Hooimeijer,3 Karim Kentouche,4 Jaco Botha,5 Peter L Turecek,6 Marta Kokot-Kierepa,5 Hanna T Gazda7 1Clinic of Pediatrics, Department of Hemato-Oncology, University Clinical Center of Kragujevac, Kragujevac, Serbia; 2Faculty of Medical Sciences, Department of Pediatrics, University of Kragujevac, Kragujevac, Serbia; 3Pediatric Hematology, Beatrix Children’s Hospital, University Medical Center Groningen, Groningen, the Netherlands; 4Klinik für Kinder- und Jugendmedizin, Universitätsklinikum Jena, Jena, Deutschland; 5Takeda Pharmaceuticals International AG, Zürich, Switzerland; 6Baxalta Innovations GmbH, Part of Takeda, Vienna, Austria; 7Takeda Development Center Americas, Inc., Cambridge, MA, USACorrespondence: Hanna T Gazda, Takeda Development Center Americas, Inc., 650 East Kendall Street, Cambridge, MA, 02142, USA, Tel [phone], Email [email protected]: Purified factor IX (FIX) concentrate (IMMUNINE®, Takeda Manufacturing Austria AG, Vienna, Austria) is indicated for the treatment and prophylaxis of bleeding episodes in patients with congenital hemophilia B. Data on the use of purified FIX concentrate in patients ≤ 6 years old with congenital hemophilia B are limited.Aim: Document real-world clinical experience with purified FIX concentrate in routine practice for pediatric patients with hemophilia B.Methods: This prospective post-authorization safety surveillance study enrolled patients ≤ 6 years old with moderate or severe hemophilia B (baseline FIX ≤ 5%) who were prescribed purified FIX concentrate, as determined by the treating physician. The planned observation period for each patient was either 12 months or ≥ 50 exposure days, whichever occurred first. The primary endpoints were the occurrence of treatment-related adverse events (AEs) and serious AEs (SAEs), and inhibitor development.Results: Thirteen male patients (mean ± standard deviation age, 3.80 ± 1.76 years) enrolled and received ≥ 1 treatment with purified FIX concentrate. Thirty-two AEs were reported in 6 patients; 4 were SAEs. No AEs were considered related to purified FIX concentrate. No patients developed inhibitory antibodies. Inhibitor testing was not conducted in 2 patients. Eighteen bleeding episodes were treated with purified FIX concentrate in 6 patients. Hemostatic efficacy was rated as either “excellent” or “good” in all patients with an available rating.Conclusion: No treatment-related AEs were reported, and purified FIX concentrate was shown to be effective in treating and preventing bleeding episodes in pediatric patients ≤ 6 years old with hemophilia B.Keywords: factor IX, hemophilia B, pediatrics, post-marketing product surveillance, surgery

Keywords

• factor ix
• hemophilia b
• pediatrics
• post-marketing product surveillance
• surgery