Abstract Number: PB1206
Meeting: ISTH 2020 Congress
Background: FXIII deficiency is a rare coagulation disorder. Bleeds are especially mucocutaneous, but life-threatening intracranial haemorrhages (ICHs) can occur in case of patients with severe disease. These subjects should therefore be treated in prophylaxis from childhood to prevent bleeding.
However, in the literature the data relating to the use of rFXIII in real life are insufficient.
Aims: To evaluate the characteristics of patients with FXIIII deficiency treated with rFXIII, the real dosage and frequency of the catridecacog administration used, and the clinical outcomes.
Methods: All patients with FXIII deficiency treated with catridecacog at nine Italian Haemophilia Centres were collected in this Survey. All data were subsequently statistically evaluated.
Results: Overall 17 patients with FXIII deficiency were collected, 75.0% presenting severe disorder. 8/15 were females. Mean age at diagnosis was 11 years (range birth-32 years). Two-thirds were a family disorder. In the 13/17 of cases the diagnosis was performed after major bleeding, only one of them was an ICH. These acute events were treated respectively with plasma (eight cases), pdFXIII (five cases), embolization (one case), rFXIII (one case), not reported (two cases). 76.5% of patients were subsequently put on prophylaxis with catridecacog at mean dosage of 33.1 IU/kg (range 25.0-80.0 IU/kg), on average every 4.0 weeks (range 3.0-8.0 weeks). Only two of them had muscular haematomas during a mean follow-up of 31 months, quickly resolved. Pharmacokinetics assessment was performed in the 88.2% of cases before starting prophylaxis. The other four patients, ¾ with severe FXIII, remained on-demand treatment. One of them experienced a subsequent severe ICH.
Conclusions: This is a first survey on real-world use of catridecacog performed in patients with FXIII deficiency. The efficacy and safety of rFXIII, used in most cases at the recommended dosage and frequency, was proven in all treated patients. The role of prophylaxis to prevent recurrences was also highlighted.
To cite this abstract in AMA style:Zanon E, Pasca S, Sottilotta G, Molinari AC, Banov L, Ferretti A, Di Gregorio P, Pollio B, Siboni SM, Palla R, Spiezia L, Pizzuti M, Notarangelo LD, Simioni P. An Italian Survey on the Real-world Use of rFXIII (Catridecacog) in Patients with Factor XIII Deficiency [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/an-italian-survey-on-the-real-world-use-of-rfxiii-catridecacog-in-patients-with-factor-xiii-deficiency/. Accessed January 23, 2022.
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