VPB1165 - 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

Tuesday, July 12, 2022

6:30 PM – 7:30 PM

Location: Exhibition

Presentation Format: Virtual

Presenting Author(s)

Giancarlo Castaman, MD

Medical Director
Center for Bleeding Disorders and Coagulation, Careggi University Hospital, Florence, Italy
Florence, Toscana, Italy

Co-Author(s)

AT

Annarita Tagliaferri

Dr
Regional Reference Center for Inherited Bleeding Disorders, University Hospital of Parma
Parma, Emilia-Romagna, Italy
AM

ANGELO C. MOLINARI

HEMOSTASIS AND THROMBOSIS HEAD
HEMOSTASIS AND THROMBOSIS UNIT AT GASLINI CHILDREN'S INSTITUTE
GENOVA, Liguria, Italy
FP

Flora Peyvandi

Director
Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia e Trhombosis Center, Milan, Italy
Milano, Lombardia, Italy
AC

Antonio Coppola

Dr
Regional Reference Centre for Inherited Bleeding Disorders, University Hospital of Parma
Parma, Emilia-Romagna, Italy
AF

Adele Finardi

Dr
CSL Behring
Milano, Lombardia, Italy
IS

Irene Schiavetti

Dr
Department of Health Sciences, University of Genoa
Genova, Liguria, Italy
DV

Daniella Vaccari

Dr
Hippocrates Research
Genova, Liguria, Italy
AR

Angiola Rocino

Responsabile UOC di Ematologia e Centro Emofilia e Trombosi
Ospedale del Mare Hospital
Napoli, Campania, Italy

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.