Background: FXIII deficiency is a very rare coagulation disorder that can affect equally males and females with an estimated incidence of 1 in 2 million persons worldwide. Due to this rarity, there are only few clinical and pharmacokinetic (PK) data deriving from the real-world.
Aim: The aim of this report is to compare head-to-head the pharmacokinetic data of catridecacog derived from the MENTORTM2 trial with our real-world (RW) study.
Methods: The PK-profiles of all patients with FXIII deficiency treated with catridecacog at eleven Italian Hemophilia Centers were compared with PK data obtained by Kerlin et al. in the MENTORTM2.
Results: Overall 18 real-world PK were compared with 23 PK derived from the pivotal study. In the RW 55.6% of patients were females, 26.2% in the MENTORTM2 (p < 0.05). The mean dosage of drug used for the PK assessment was 35 IU/kg in the MENTORTM2, and 33.9 IU/kg in the RW study.
Keywords: FXIII deficiency; FXIII treatment; MENTOR program; Pharmacokinetics of rFXIII; Recombinant FXIII (rFXIII).
© 2022. The Author(s), under exclusive licence to Springer Science+Business Media, LLC, part of Springer Nature.