The pharmacokinetics of recombinant FXIII (catridecacog) from the MENTORTM2 trial to a real-world study: a head-to-head comparison.

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.