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. 2024 Mar 5:15:113-122.
doi: 10.2147/JBM.S425617. eCollection 2024.

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

Zoran Igrutinović  1   2 , Hélène Louise Hooimeijer  3 , Karim Kentouche  4 , Jaco Botha  5 , Peter L Turecek  6 , Marta Kokot-Kierepa  5 , Hanna T Gazda  7
Affiliations

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

Zoran Igrutinović et al. J Blood Med. .

Abstract

Introduction: 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.

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Conflict of interest statement

Zoran Igrutinović reports being an investigator for this post-authorization safety surveillance study, which was funded by Baxalta Innovations GmbH, Vienna, Austria, a Takeda company. Hélène Louise Hooimeijer has no interests that might be perceived as posing a conflict or bias for this study. Karim Kentouche reports being an investigator for this post-authorization safety surveillance study, which was funded by Baxalta Innovations GmbH, Vienna, Austria, a Takeda company. Jaco Botha and Marta Kokot-Kierepa are employees of Takeda Pharmaceuticals International AG, and Takeda stockholders. Peter L. Turecek is an employee of Baxalta Innovations GmbH, a Takeda company, and a Takeda stockholder. Hanna T. Gazda is an employee of Takeda Development Center Americas, Inc., a Takeda company, and a Takeda shareholder. The authors report no other conflicts of interest in this work.

Figures

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Figure 1
Patient disposition.
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Grants and funding

This study was funded by Baxalta Innovations GmbH, Vienna, Austria, a Takeda company.

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