The pharmacokinetics of recombinant FXIII (catridecacog) from the MENTOR (TM) 2 trial to a real-world study: a head-to-head comparison

摘要

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 MENTOR (TM) 2 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 MENTOR (TM) 2. 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 MENTOR (TM) 2 (p < 0.05). The mean dosage of drug used for the PK assessment was 35 IU/kg in the MENTOR (TM) 2, and 33.9 IU/kg in the RW study.