PB0673 - A Post-authorization Safety Surveillance Study To Report Clinical Experience With Purified FIX Concentrate in Pediatric Patients With Hemophilia B

Monday, July 11, 2022

6:30 PM – 7:30 PM

Location: Exhibition

Presentation Format: In-Person

Co-Author(s)

ZI

Zoran Igrutinović

Professor
Clinic of Pediatrics, Department of Hemato-oncology, University Clinical Center of Kragujevac; Faculty of Medical Sciences, Department of Pediatrics, University of Kragujevac
Kragujevac, Kosovo, Serbia
LH

Louise Hooijmeijer

Hematologist
Division of Hematology/Oncology, Department of Pediatrics, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands
Groningen, Groningen, Netherlands
KK

Karim Kentouche

Senior Physician
Klinik für Kinder- und Jugendmedizin, Universitätsklinikum Jena
Jena, Thuringen, Germany
JB

Jaco Botha

Director, Global Medical Affairs Group Lead
Takeda Pharmaceuticals International AG
Zurich, Zurich, Switzerland
Peter L. Turecek, PhD, PharmD

Professor
Baxalta Innovations GmbH, a Takeda company
Vienna, Wien, Austria

Presenting Author(s)

Marta Kokot-Kierepa, MD, PhD

Global Medical Lead
Takeda Pharmaceuticals International AG
Glattpark, Zurich, Switzerland

Co-Author(s)

HG

Hanna Gazda

Medical Director
Takeda Pharmaceutical Company Limited
Lexington, Massachusetts, United States

Background: The safety and efficacy of purified factor IX (FIX) concentrate (Baxter Innovations GmbH, Vienna, Austria) have been demonstrated in clinical trials in adult and pediatric patients with hemophilia B (HB).

Aims: To document clinical experience with purified FIX concentrate in routine practice for pediatric patients with HB.

Methods: This was a prospective, uncontrolled, open-label, post-authorization safety surveillance study. Patients aged ≤6 years with moderate or severe HB (baseline FIX ≤5%) who were prescribed purified FIX concentrate were eligible for inclusion. The observation period was either 12 months or ≥50 exposure days, whichever occurred first. The treatment regimen was determined by the treating physician. The primary endpoint was the occurrence of treatment-related adverse events (AEs) and serious AEs (SAEs), and inhibitor development. Secondary endpoints included hemostatic efficacy and number of infusions required to achieve bleed resolution.

Results: In total, 13 patients enrolled and received ≥1 treatment with purified FIX concentrate. All patients were male and mean (SD) age at baseline was 3.80 (1.76) years. During the study, 11 patients received prophylaxis for mean (SD) 73.6 (37.0) days and 6 received on-demand treatment for mean (SD) 3.7 (1.0) days. Two patients received prophylaxis for surgery. In total, 28 non-serious AEs in 6 patients and 4 SAEs in 3 patients were reported (Table 1). No AEs were considered related to study treatment. No patients (n=10) developed inhibitory antibodies. Inhibitor testing was not done for 3 patients. Overall hemostatic efficacy ratings and annualized bleeding rates are shown in Table 2. In total, 18 bleeding episodes reported in 6 patients were treated with purified FIX concentrate. Mean (SD) number of infusions per bleeding episode was 1.18 (0.57).

Conclusion(s): Purified FIX concentrate was not associated with any treatment-related AEs and was shown to be effective in treating and preventing bleeding episodes in pediatric patients aged ≤6 years with HB.