Safety and effectiveness of recombinant factor XIII-A₂ in congenital factor XIII deficiency: Real-world evidence.

ABSTRACT:

Background

Regular factor XIII (FXIII) prophylaxis is standard treatment for congenital FXIII A-subunit deficiency (FXIII-A CD). Recombinant factor XIII-A₂ (rFXIII-A₂) was extensively evaluated in the mentor trials.

Objective

To assess real-world safety and treatment effectiveness of rFXIII-A₂ prophylaxis from the mentor 6 trial.

Patients/methods

mentor 6 was a noninterventional, postauthorization safety study investigating rFXIII-A₂ prophylaxis in FXIII-A CD. rFXIII-A₂ treatment was observed for 2 to 6 years per patient. The primary end point was documentation of adverse drug reactions (including anti-FXIII antibody development). Secondary end points were serious adverse events (SAEs), medical events of special i

SUBMITTER: Poulsen LH 

PROVIDER: S-EPMC8882239 | biostudies-literature | 2022 Feb

REPOSITORIES: biostudies-literature