Safety and effectiveness of recombinant factor XIII-A2 in congenital factor XIII deficiency: Real-world evidence.
Res Pract Thromb Haemost
; 6(2): e12628, 2022 Feb.
Article
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| MEDLINE
| ID: mdl-35243202
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 interest (MESIs), and annualized bleeding rate (ABR).RESULTS:
Among 30 patients (mean age, 25.5 years), there were 44 adverse events (AEs) (30 mild, 13 moderate, 1 severe). Eleven AEs were possibly/probably related to rFXIII-A2. Of four MESIs, two were unlikely related to rFXIII-A2 (accidental overdose, deep vein thrombosis), and two were possibly/probably related (nonneutralizing anti-FXIII antibody, decreased therapeutic response). All 10 SAEs were unlikely related to rFXIII-A2. Over a follow-up of 75.4 patient-years, there were six treatment-requiring bleeds (all trauma-related with no spontaneous bleeds), giving a treatment-requiring ABR of 0.066; five bleeds were treated successfully with rFXIII-A2. Eight of nine minor surgeries performed during rFXIII-A2 prophylaxis reported successful hemostatic outcomes (one missing evaluation).CONCLUSIONS:
These data confirm that rFXIII-A2 prophylaxis is well tolerated as long-term care. There were no spontaneous bleeds, ABR was low, and rFXIII-A2 successfully treated bleeds in patients receiving rFXIII-A2 prophylaxis.
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Colección:
01-internacional
Banco de datos:
MEDLINE
Idioma:
En
Revista:
Res Pract Thromb Haemost
Año:
2022
Tipo del documento:
Article