IDEAL study: A real-world assessment of pattern of use and clinical outcomes with recombinant factor IX albumin fusion protein (rIX-FP) in patients with haemophilia B in Italy

G. Castaman¹, A. Tagliaferri², A. Molinari³, F. Peyvandi⁴, A. Coppola⁵, A. Finardi⁶, I. Schiavetti⁷, D. Vaccari⁸, A. Rocino⁹

¹“Careggi” University Hospital, Hemorrhagic and Coagulation Disease Unit, Department of Oncology., Florence, Toscana, Italy, ²Regional Reference Center for Inherited Bleeding Disorders, University Hospital of Parma, Parma, Emilia-Romagna, Italy, ³Regional Reference Center for Hemorrhagic Diseases, G. Gaslini Children's Hospital, Genova, Liguria, Italy, ⁴Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi, Hemophilia and Thrombosis Center, and Università degli Studi di Milano, Department of Pathophysiology and Transplantation, Milano, Lombardia, Italy, ⁵Regional Reference Centre for Inherited Bleeding Disorders, University Hospital of Parma, Parma, Emilia-Romagna, Italy, ⁶CSL Behring, Milano, Lombardia, Italy, ⁷Department of Health Sciences, University of Genoa, Genova, Liguria, Italy, ⁸Hippocrates Research, Genova, Liguria, Italy, ⁹Ospedale del Mare Hospital, Napoli, Campania, Italy

Abstract Number: VPB1165

Meeting: ISTH 2022 Congress

Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical

Background: Factor IX replacement therapy is used for treatment and prophylaxis of bleeding in hemophilia B. rIX-FP is a long-acting albumin-fusion protein, which, in clinical studies, has demonstrated prolonged dosing intervals up to 21 days for routine prophylaxis, providing therapeutic benefit.

Aims: To describe dosing frequency and consumption (primary endpoint), efficacy and safety of rIX-FP treatment during routine clinical practice in Italy.

Methods: Patients with moderate/severe hemophilia B on prophylaxis with rIX-FP for ≥6 months, providing written informed consent, were enrolled in this observational study from March 2017 to February 2019 and followed-up for two years. Descriptive analysis included prospective and retrospective data (12 months prior to switching to rIX-FP). The study was approved by competent Ethics Committees.

Results: Data were collected from 59 male patients (median age 30.1 yrs) enrolled by 23 Italian centers. Of them, 50 were on prophylaxis during the entire observation period and completed the study. The infusion frequency changed from every 2-3 times/week in 86.0% of patients with previous treatment, to less than once a week in 84.0% of patients treated with rIX-FP at the second-year follow-up. Annual number of infusions decreased of about 70%, however, mean FIX activity trough level increased from 3.8% to 14.4% (Table 1). Median Annualized Bleeding Rate of 0.00 was achieved across all prophylaxis regimens (Table 2). Subjects with zero bleedings increased with rIX-FP from 66.0 % to 78.0%. In the 2-year follow-up, the rate of patients with chronic joint pain decreased from 44.0% at baseline to 16.0%, while that of patients with target joints from 30.0% to 8.0%. No safety concerns were identified, no patient developed inhibitors.

Conclusion(s): Treatment with rIX-FP reduced infusion frequency, while providing higher FIX trough levels, reduction of target joints and chronic pain, with a good safety profile.

Table 1

Table 1. Factor IX activity level -%-: previous treatment vs rIX-FP 2Y Follow Up

SD – Standard Deviation

rIX-FP – recombinant factor IX albumin fusion protein

Table 2

Table 2. Annualized Bleeding Rate -ABR-: previous treatment vs rIX-FP 2Y Follow Up

SD – Standard Deviation

rIX-FP – recombinant factor IX albumin fusion protein

To cite this abstract in AMA style:

Castaman G, Tagliaferri A, Molinari A, Peyvandi F, Coppola A, Finardi A, Schiavetti I, Vaccari D, Rocino A. IDEAL study: A real-world assessment of pattern of use and clinical outcomes with recombinant factor IX albumin fusion protein (rIX-FP) in patients with haemophilia B in Italy [abstract]. https://abstracts.isth.org/abstract/ideal-study-a-real-world-assessment-of-pattern-of-use-and-clinical-outcomes-with-recombinant-factor-ix-albumin-fusion-protein-rix-fp-in-patients-with-haemophilia-b-in-italy/. Accessed September 21, 2023.

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