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Population Parmacokinetics of rFXIII (Catridecacog) and Clinical Outcomes of Prophylaxis in an Italian Population of Patients with Factor XIII Deficiency

1Padua University Hospital, Padua, Italy, 2Hospital of Reggio Calabria, Reggio Calabria, Italy, 3Gaslini Children Hospital, Genoa, Italy, 4Umberto I University Hospital, Rome, Italy, 5SS. Annunziata Hospital, Chieti, Italy, 6Regina Margherita Children Hospital, Turin, Italy, 7Milan University Hospital, Milan, Italy, 8S. Carlo Hospital, Potenza, Italy, 9ASST Spedali Civili of Brescia, Brescia, Italy, 10Udine University Hospital, Udine, Italy, 11Cesena Hospital, Cesena, Italy

Abstract Number: PB0692

Meeting: ISTH 2021 Congress

Theme: Hemophilia and Rare Bleeding Disorders » Rare Bleeding Disorders

Background: FXIII deficiency is a very rare coagulation disorder. Bleeds are usually mucocutaneous, but life-threatening intracranial hemorrhages (ICHs) can occur in cases of severe disease. Currently the best treatment consists in the use of FXIII replacement concentrates in prophylaxis since birth. Data concerning the use of catridecacog in the real-life is scarce.

Aims: The primary end-point is to compare the different PK-profiles of rFXIII using a population statistics model, secondary end-point is to evaluate the clinical outcomes of prophylaxis with rFXIIII.

Methods: This study enrolled all patients presenting FXIII deficiency treated with catridecacog at ten Italian Hemophilia Centers. PK-profiles were evaluated at the Pharmacology Department of Udine University Hospital. All clinical data and outcomes were collected and analyzed.

Results: Overall 20 patients with FXIII deficiency were collected, 75% presenting severe disorder. 11/20 were females. Mean age at diagnosis was 15 years (range birth-74 years). 60% had a known family disorder. Pharmacokinetics was assessed in 16/20 of cases before starting prophylaxis. Mean age at PK-assessment was 36 years (6-74 years), mean dose of rFXIII infused for PK evaluation was 31.5 IU/kg (25-50 IU/kg), Figure 1.
Summary of population pharmacokinetic parameters
Individual steady-state rFXIII concentration-time profiles of 16 evaluated patients is represented in Figure 2. Prophylaxis was subsequently started on 65% of patients at a 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). During a mean follow-up of 43 months, one ileo-psoas hematoma which quickly resolved, one muscular hematoma, and two minor surgeries were reported. One severe patient who remained on demand treatment experienced a severe ICH.
Individual steady-state rFXIII concentration-time profiles of 16 evaluated patients

Conclusions: The pharmacokinetics population  obtained was similar to that reported in the MENTOR studies but dose used and infusion timing are in some cases different. Efficacy and safety of rFXIII was proven in all patients. The role of prophylaxis to prevent severe hemorrhagic events 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, Peyvandi F, Pizzuti M, Notarangelo LD, Cojutti P, Biasoli C, Simioni P, Pea F. Population Parmacokinetics of rFXIII (Catridecacog) and Clinical Outcomes of Prophylaxis in an Italian Population of Patients with Factor XIII Deficiency [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/population-parmacokinetics-of-rfxiii-catridecacog-and-clinical-outcomes-of-prophylaxis-in-an-italian-population-of-patients-with-factor-xiii-deficiency/. Accessed October 1, 2023.

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ISTH Congress Abstracts - https://abstracts.isth.org/abstract/population-parmacokinetics-of-rfxiii-catridecacog-and-clinical-outcomes-of-prophylaxis-in-an-italian-population-of-patients-with-factor-xiii-deficiency/