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Recombinant factor IX (BAX326) in previously treated paediatric patients with haemophilia B: a prospective clinical

T Urasinski1, O Stasyshyn2, T Andreeva3

  • 1Department of Paediatrics, Paediatric Haematology and Oncology, Pomeranian Medical University in Szczecin, Szczecin, Poland.

Haemophilia : the Official Journal of the World Federation of Hemophilia
|December 16, 2014
PubMed

Insights

A new recombinant factor IX (BAX326) is safe and effective for preventing and treating bleeds in children under 12 with haemophilia B. The treatment showed excellent or good efficacy for over 96% of bleeds in a clinical trial.

Area of Science:

  • Hematology
  • Pediatric Medicine
  • Pharmacology

Background:

  • Hemophilia B is a genetic bleeding disorder caused by deficiency in coagulation factor IX (FIX).
  • Current treatments for hemophilia B include factor IX replacement therapy, with prophylaxis being a key strategy for managing severe and moderately severe cases.
  • Paediatric patients require safe and effective treatment options for long-term management of their condition.

Purpose of the Study:

  • To evaluate the safety, haemostatic efficacy, and pharmacokinetic profile of a newly developed recombinant factor IX (BAX326) in previously treated paediatric patients with hemophilia B.
  • To assess the tolerability and immunogenicity of BAX326 in children aged less than 12 years.
  • To determine the effectiveness of BAX326 in preventing and treating bleeding episodes.

Main Methods:

  • A prospective clinical trial involving 23 previously treated paediatric patients (aged <12 years) with severe or moderately severe hemophilia B.
  • BAX326 was administered twice weekly for prophylaxis over 6 months and on-demand for bleeds.
  • Safety assessments included adverse events, thrombotic events, and immunologic testing. Efficacy was measured by annualized bleeding rate (ABR) and treatment response. Pharmacokinetics (PK) were assessed over 72 hours.

Main Results:

  • No treatment-related serious adverse events (SAEs) or adverse events (AEs) were reported in any of the 23 treated subjects.
  • No thrombotic events or inhibitory antibodies against FIX, rFurin, or CHO protein were observed.
  • Haemostatic efficacy was excellent or good for over 96% of bleeds, with 87% of subjects experiencing no spontaneous bleeds. Younger children (<6 years) showed longer half-life (T1/2) and lower inhibitor rates (IR) compared to older children (6-12 years).

Conclusions:

  • Recombinant factor IX (BAX326) is safe and efficacious for prophylactic use and on-demand treatment of bleeds in paediatric patients (<12 years) with hemophilia B.
  • The pharmacokinetic profile suggests age-dependent differences, with younger children potentially benefiting from longer protection.
  • BAX326 represents a viable and well-tolerated therapeutic option for managing hemophilia B in the paediatric population.

Keywords:
haemophilia Bpaediatricpreviously treated patientsrecombinant factor IX

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