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Recombinant FXIII (rFXIII-A2) Prophylaxis Prevents Bleeding and Allows for Surgery in Patients with Congenital FXIII A-Subunit Deficiency.

Recombinant FXIII (rFXIII-A2) Prophylaxis Prevents Bleeding and Allows for Surgery in Patients with Congenital FXIII A-Subunit Deficiency.
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Carcao M, Altisent C, Castaman G, Fukutake K, Kerlin BA, Kessler C, Lassila R, Nugent D, Oldenburg J, Garly ML, Rosholm A, Inbal A,


Carcao M, Altisent C, Castaman G, Fukutake K, Kerlin BA, Kessler C, Lassila R, Nugent D, Oldenburg J, Garly ML, Rosholm A, Inbal A, (click to view)

Carcao M, Altisent C, Castaman G, Fukutake K, Kerlin BA, Kessler C, Lassila R, Nugent D, Oldenburg J, Garly ML, Rosholm A, Inbal A,

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Thrombosis and haemostasis 2018 02 15() doi 10.1055/s-0038-1624581

Abstract

Recombinant factor XIII-A(rFXIII-A) was developed for prophylaxis and treatment of bleeds in patients with congenital FXIII A-subunit deficiency. mentor™2 (NCT00978380), a multinational, open-label, single-arm, multiple-dosing extension to the pivotal mentor™1 trial, assessed long-term safety and efficacy of rFXIII-Aprophylaxis in eligible patients (patients with severe [<0.05 IU/mL] congenital FXIII subunit A deficiency) aged ≥6 years. Patients received 35 IU/kg rFXIII-A(exact dosing) every 28 ± 2 days for ≥52 weeks. Primary endpoint was safety (adverse events including immunogenicity); secondary endpoints were rate of bleeds requiring FXIII treatment, haemostatic response after one 35 IU/kg rFXIII-Adose for breakthrough bleeds and withdrawals due to lack of rFXIII-Aefficacy. Steady-state pharmacokinetic variables were also summarized. Elective surgery was permitted during the treatment period. Sixty patients were exposed to rFXIII-A; their median age was 26.0 years (range: 7.0-77.0). rFXIII-Awas well tolerated without any safety concerns. No non-neutralizing or neutralizing antibodies (inhibitors) against FXIII were detected. Mean annualized bleeding rate (ABR) was 0.043/patient-year. Mean spontaneous ABR was 0.011/patient-year. No patients withdrew due to lack of efficacy. Geometric mean FXIII trough level was 0.17 IU/mL. Geometric terminal half-life was 13.7 days. rFXIII-Aprophylaxis provided sufficient haemostatic coverage for 12 minor surgeries without the need for additional FXIII therapy; eight procedures were performed within 7 days of the patient's last scheduled rFXIII-Adose, and four were performed 10 to 21 days after the last dose.

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