PB0807 - Inhibitors in 3 patients with type 3 von Willebrand disease in a Portuguese Centre – management of bleeding and surgeries

Monday, July 11, 2022

6:30 PM – 7:30 PM

Location: Exhibition

Presentation Format: In-Person

Presenting Author(s)

Cristina Catarino, Dr

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
Carolina Peixoto, MD

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

Liubov Parusnikova

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

Joana S. Pestana

Resident
Evora
Evora, Evora, Portugal
SC

Soraia Campaniço

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

Joana Lucas

Medical Doctor
Hospital Nélio Mendonça
Funchal, Madeira, Portugal
PA

Patrícia Afonso

Pharmaceutic
Centro Hospitalar Universitário Lisboa Norte
Lisboa, Lisboa, Portugal

Background: The development of alloantibodies against von Willebrand factor (VWF) represents a rare but serious complication of the treatment of VW disease (VWD), being associated with the lack of hemostatic response to VWF concentrates but, also, with the risk of anaphylactic reactions.

Aims: Demonstrade the efficacy and safety of recombinant activated factor VII (rFVIIa) in this group of patients.

Methods: We, retrospectively, evaluated data on bleeding episodes and surgical procedures in 3 patients with type 3 VWD with inhibitors (VWDi) followed at our center.

Results: Between 2006 and 2021, 3 patients with VWD and inhibitors (VWDi): 2 females and 1 male were diagnosed and treated at our center (ages 4-20). Inhibitors were suspected due to the absence of bleeding control while on therapy with FVIII/FVW concentrates; to laboratory results that did not match the administered therapy with FVIII/FVW concentrates; and to an allergic reaction related to the infusion of FVIII/FVW concentrate.
During this period, more than 40 bleeding episodes were treated with rFVIIa. These episodes include mostly mucocutaneus bleeding (oral; gastrointestinal; gynecological; urological; epistaxis), large muscular hematomas; hemarthrosis and retroperitoneal hemorrhages. rFVIIa was also effective in several dental extractions and in 2 major surgeries (1 patient was submitted to an hysterectomy and another patient to an abdominal surgery due to a life-threatening bleeding).
Average dosage of rFVIIa was 100 micrograms and the schedule was decided according to the type and severity of the clinical situation. In severe bleeding, and in major surgeries rFVIIa was administered, in general, with 2 or 3 hours intervals.
No adverse effects were observed related to hemostatic therapy.

Conclusion(s): Even though, in our patients, rFVIIa was clearly effective, we think it would be important to have a way to compile information on this group of patients, in order to determine the most appropriate approach.