Study Finds No Treatment-Related Adverse Events in Boys Treated With Purified FIX Concentrate

Purified factor IX (FIX) concentrate was effective in treating and preventing bleeding episodes in real-world pediatric patients 6 years and younger with hemophilia B, according to a study published in the Journal of Blood Medicine.

“None of the 32 adverse events, including four serious adverse events, reported in this study were considered related to purified FIX concentrate, and all events resolved during the surveillance period,” wrote corresponding author Hanna T. Gazda, of Takeda Development Center Americas Inc., Cambridge, Massachusetts, and study coauthors.

The findings stemmed from a post-authorization safety surveillance study of 13 male pediatric patients with moderate or severe hemophilia B prescribed purified FIX concentrate at nine study sites before enrollment. Nine patients had more than 50 exposure days to FIX concentrate prior to enrollment. The mean ± standard deviation age of the 13 patients was 3.80 ± 1.76 years.

According to the study, four serious adverse events occurred in three patients: accidental head injuries in two patients, and two cases of sepsis in one patient. No adverse events were considered to be associated with purified FIX concentrate.

Among 11 patients who underwent inhibitor testing, none developed inhibitory antibodies, researchers reported.

In all, 18 bleeding episodes in a total six patients were treated with purified FIX concentrate. Physicians rated the drug’s hemostatic efficacy “excellent” or “good” in all patients with available ratings.

“The findings from this study, therefore, provide further support to the continued use of plasma-derived FIX concentrates such as Immunine in the management of patients with hemophilia B,” researchers wrote.

Reference

Igrutinović Z, Hooimeijer HL, Kentouche K, et al. A post-authorization safety surveillance study to report clinical experience with purified factor IX Concentrate in pediatric patients with hemophilia B. J Blood Med. 2024;15:113-122. doi:10.2147/JBM.S425617