Abstract
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.
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Change history
01 December 2022
A Correction to this paper has been published: https://doi.org/10.1007/s11239-022-02744-z
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Acknowledgements
All the authors meet the International Committee of Medical Journal Editors criteria for authorship for this manuscript, take responsibility for the integrity of the work as a whole, and have given final approval of the version to be published. S.P and E.Z. concepted and designed the study; P.C. and F.P. analyzed and interpreted the data; S.P. wrote the manuscript; all authors revised and approved the manuscript. Thanks to all the Italian FXIII Study Group : Laura Banov (Gaslini Children Hospital, Genoa), Chiara Biasioli (Hospital of Cesena), Patrizia Di Gregorio (SS. Annunziata Hospital of Chieti), Antonietta Ferretti (Umberto I University Hospital, Rome), Angelo Claudio Molinari (Gaslini Children Hospital, Genoa), Lucia Dora Notarangelo (ASST Spedali Civili, Brescia), Roberta Palla (Haemophilia and Thrombosis Center, Milan University Hospital), Flora Peyvandi (Haemophilia and Thrombosis Center, Milan University Hospital), Michele Pizzuti (San Carlo Hospital, Potenza), Berardino Pollio (Regina Margherita Children Hospital, Turin), Gianluca Sottilotta (Hospital of Reggio Calabria), and Simona Maria Siboni (Haemophilia and Thrombosis Center, Milan University Hospital).
Funding
This research did not receive any specific grant from funding agencies in the public, commercial, or not-for-profit sectors.
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Pasca, S., PierGiorgio, C., Pea, F. et al. The pharmacokinetics of recombinant FXIII (catridecacog) from the MENTORTM2 trial to a real-world study: a head-to-head comparison. J Thromb Thrombolysis 54, 593–596 (2022). https://doi.org/10.1007/s11239-022-02700-x
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DOI: https://doi.org/10.1007/s11239-022-02700-x