ISTH Congress Abstracts

Official abstracts site for the ISTH Congress

MENU 

Results from a Phase 4, Real-World Study Confirm the Safety and Effectiveness of rFXIII-A2 for the Treatment of Congenital FXIII A-Subunit Deficiency

1Aarhus University Hospital and Aarhus University, Haemophilia Center, Department of Hematology, Aarhus, Denmark, 2The Ohio State University College of Medicine, Nationwide Children's Hospital, Columbus, OH, United States, 3Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Firenze, Italy, 4Regional Reference Center for Hemorrhagic Diseases, Giannina Gaslini Children's Hospital, Genoa, Italy, 5Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Cà Granda, Ospedale Maggiore Policlinico, Milan, Italy, 6Novo Nordisk Service Centre India Private Ltd, Bangalore, India, 7Novo Nordisk A/S, Søborg, Denmark, 8Division of Haematology/Oncology, Department of Paediatrics and Child Health Evaluative Sciences, Research Institute, Hospital for Sick Children, University of Toronto, Toronto, ON, Canada

Abstract Number: PB1169

Meeting: ISTH 2020 Congress

Theme: Hemophilia and Rare Bleeding Disorders » Rare Bleeding Disorders

Background: Recombinant factor XIII-A2 (rFXIII-A2) is indicated for prophylaxis in patients with congenital FXIII A-subunit deficiency (FXIII-A CD). Real-world evidence on usage of rFXIII-A2 is lacking.

Aims: Investigate the long-term, real-world rFXIII-A2 use on effectiveness and adverse drug reactions from mentor™6 (a prospective, multinational, non-interventional, post-authorisation safety study), and effectiveness and safety outcomes from patients in the Prospective Rare Bleeding Disorders Database (PRO-RBDD) registry.

Methods: Both mentor™6 and PRO-RBDD prospectively assessed the following endpoints: annualised bleeding rate (ABR), efficacy for minor surgery and bleed treatment, serious adverse events (SAEs), medical events of special interest (MESIs), and specific adverse drug reactions (anti-FXIII antibodies, allergic reactions, embolic/thrombotic events and lack of therapeutic effect).

Results: mentor™6 enrolled 30 patients (Table 1, mean [range] age: 25.5 [2-68] years), total exposure time: 75.4 person-years.
No spontaneous treatment-requiring bleeds occurred during mentor™6. Six traumatic bleeds required treatment with a FXIII-containing product, thus the mean ABR was 0.066 bleeds/year; 5/6 treatment-requiring bleeds were treated with rFXIII-A2 and all had a successful outcome (Table 2). Of nine minor surgeries performed during rFXIII-A2 prophylaxis, eight reported successful haemostatic outcomes (one missing evaluation).
No neutralising anti-FXIII antibodies (inhibitors), allergic reactions, withdrawals due to AEs, or embolic/thrombotic events with causal relation to rFXIII-A2 were reported. Eighteen patients reported 44 AEs; 30 were mild, 13 moderate and one severe. Eleven AEs were possibly/probably related to rFXIII-A2. Ten AEs were considered SAEs, all were unlikely related to rFXIII-A2; of four MESIs, two were possibly/probably related to rFXIII-A2 (non-neutralising [binding] antibodies and suspected lack of efficacy). All patients with related AEs, MESIs or SAEs recovered.
PRO-RBDD included four patients exposed for ≈9.5 person-years. No AEs, or treatment-requiring or spontaneous bleeds were reported.

Conclusions: These real-world, long-term data confirm the safety and effectiveness of rFXIII-A2 for prophylaxis, treatment of bleeding episodes and minor surgeries in patients with FXIII-A CD.

[Table 1 AEs during mentor™6.]


[Table 2 Treatment-requiring bleeds (all were traumatic) during mentor™6.]

To cite this abstract in AMA style:

Hvitfeldt Poulsen L, Kerlin BA, Castaman G, Molinari AC, Menegatti M, Dey S, Garly M-, Carcao M. Results from a Phase 4, Real-World Study Confirm the Safety and Effectiveness of rFXIII-A2 for the Treatment of Congenital FXIII A-Subunit Deficiency [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/results-from-a-phase-4-real-world-study-confirm-the-safety-and-effectiveness-of-rfxiii-a2-for-the-treatment-of-congenital-fxiii-a-subunit-deficiency/. Accessed September 27, 2023.

« Back to ISTH 2020 Congress

ISTH Congress Abstracts - https://abstracts.isth.org/abstract/results-from-a-phase-4-real-world-study-confirm-the-safety-and-effectiveness-of-rfxiii-a2-for-the-treatment-of-congenital-fxiii-a-subunit-deficiency/