PB1148 - Real-world usage of extended half-life FIX in a Portuguese Haemophilia Centre – prophylaxis must be for moderate and severe hemophilia B patients

Tuesday, July 12, 2022

6:30 PM – 7:30 PM

Location: Exhibition

Presentation Format: In-Person

Presenting Author(s)

Carolina Peixoto, MD

Doctor
Centro Hospitalar Universitário Lisboa Norte
Lisboa, Lisboa, Portugal

Co-Author(s)

FR

Fátima Rodrigues

Doctor
Centro Hospitalar Universitário Lisboa Norte
Lisboa, Lisboa, Portugal
AP

Artur Pereira

Doctor
Centro Hospitalar Universitário Lisboa Norte
Lisboa, Lisboa, Portugal
SC

Soraia Campaniço

Medical Doctor
Centro Hospitalar Universitário Lisboa Norte - Hospital Santa Maria, Lisbon, Portugal
Lisboa, Lisboa, Portugal
CJ

Catarina J. Jacinto

Doctor
Centro Hospitalar Universitário Lisboa Norte
Lisboa, Lisboa, Portugal
PA

Patrícia Afonso

Pharmaceutic
Centro Hospitalar Universitário Lisboa Norte
Lisboa, Lisboa, Portugal
JP

Joana S. Pestana

Resident
Evora
Evora, Evora, Portugal
LM

Luís Moura

Doctor
Centro Hospitalar Tondela-Viseu
Porto, Porto, Portugal
LP

Liubov Parusnikova

Medical Doctor
Centro Hospitalar Universitário Lisboa Norte - Hospital Santa Maria, Lisbon, Portugal
Lisboa, Lisboa, Portugal
Cristina Catarino, Dr

Doctor
Centro Hospitalar Universitário Lisboa Norte
Lisboa, Lisboa, Portugal

Background: Extended half-life factor IX (EHL-FIX) products promise less frequent dosing while improving protection, compliance and outcomes. All our moderate and severe hemophilia B (HB) patients, were offered prophylaxis with recombinant FIX Fc fusion (rFIXFc).

Aims: To compare prophylaxis regimens and outcomes before and after switching from a standard recombinant half-life FIX product (SHL) to EHL rFIXFc.

Methods: Data on patients’ characteristics, previous and actual regimen, pharmacokinetics (PK), annual bleeding rate (ABR) and factor consumption (FC) were reviewed.

Results: Ten patients (9adults and 1child; aged 8-69 years) with HB (5 severe, 5 moderate), are now on prophylaxis with rFIX-Fc. Five severe HB patients were already on prophylaxis (4 on a twice and 1 on a weekly regimen). Five moderate patients, previously on demand (OD) started prophylaxis with rFIX-Fc. These patients have been treated with rFIX between 1 to 21 months.
After evaluation of PK, 9/10 patients were put on a fortnight dosing regimen, aiming FIX levels>3–5 IU/dL. 3 patients were later switched to a weekly regimen as higher through levels and better protection were needed. A 10-day regimen was considered safer for a previously untreated boy (PUP). Dosage per infusion (31-45 UI/Kg) did not change in the patients previously on prophylaxis. In these patients a marked decrease in the number of annual infusions (61%) and FC (58,2%) was observed. Mean ABR decreased from 15 to 5, while traumatic and spontaneous joint ABR decreased from 6 to 1 and from 4 to 0, respectively. More patients had zero joint bleeds after starting prophylaxis with the EHL FIX product (50% vs 10%). More important, in the last 12 months, no patient had a spontaneous joint bleed. No patient developed inhibitors to rFIXFc.

Conclusion(s): Prophylaxis with rFIX-Fc allowed a significant reduction in the frequency of infusions and a lower bleeding rate but also an improved protection and QoL.