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A Post-Authorization Safety Surveillance Study to Report Clinical Experience with Purified Factor IX Concentrate in
Zoran Igrutinović1,2, Hélène Louise Hooimeijer3, Karim Kentouche4
1Clinic of Pediatrics, Department of Hemato-Oncology, University Clinical Center of Kragujevac, Kragujevac, Serbia.
Insights
Purified factor IX (FIX) concentrate is safe and effective for treating young children with hemophilia B. This study documented positive real-world clinical experience in pediatric patients, showing no treatment-related adverse events.
Area of Science:
- Hematology
- Pediatric Medicine
- Pharmacovigilance
Background:
- Congenital hemophilia B is a rare bleeding disorder.
- Purified factor IX (FIX) concentrate is a standard treatment.
- Limited data exist for its use in children under 6 years old.
Purpose of the Study:
- To document real-world clinical experience with purified FIX concentrate.
- To assess safety and efficacy in pediatric patients with hemophilia B.
- Focus on patients aged 6 years or younger.
Main Methods:
- Prospective post-authorization safety surveillance study.
- Enrolled pediatric patients (≤6 years) with hemophilia B (FIX ≤5%).
- Observed for 12 months or ≥50 exposure days, monitoring adverse events and inhibitors.
Main Results:
- Thirteen male patients (mean age 3.8 years) received treatment.
- No treatment-related adverse events or serious adverse events were reported.
- Excellent or good hemostatic efficacy observed in all evaluable patients; no inhibitors developed.
Conclusions:
- Purified FIX concentrate demonstrated a favorable safety profile in pediatric patients.
- The concentrate was effective in treating and preventing bleeding episodes.
- Supports the use of purified FIX concentrate in young children with hemophilia B.
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.
