Recombinant FXIII (rFXIII-A(2)) Prophylaxis Prevents Bleeding and Allows for Surgery in Patients with Congenital FXIII A-Subunit Deficiency

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Pysyväisosoite

http://hdl.handle.net/10138/233934

Lähdeviite

Carcao , M , Altisent , C , Castaman , G , Fukutake , K , Kerlin , B A , Kessler , C , Lassila , R , Nugent , D , Oldenburg , J , Garly , M-L , Rosholm , A & Inbal , A 2018 , ' Recombinant FXIII (rFXIII-A(2)) Prophylaxis Prevents Bleeding and Allows for Surgery in Patients with Congenital FXIII A-Subunit Deficiency ' , Thrombosis and Haemostasis , vol. 118 , no. 3 , pp. 451-460 . https://doi.org/10.1055/s-0038-1624581

Julkaisun nimi: Recombinant FXIII (rFXIII-A(2)) Prophylaxis Prevents Bleeding and Allows for Surgery in Patients with Congenital FXIII A-Subunit Deficiency
Tekijä: Carcao, Manuel; Altisent, Carmen; Castaman, Giancarlo; Fukutake, Katsuyuki; Kerlin, Bryce A.; Kessler, Craig; Lassila, Riitta; Nugent, Diane; Oldenburg, Johannes; Garly, May-Lill; Rosholm, Anders; Inbal, Aida
Tekijän organisaatio: Clinicum
Hematologian yksikkö
Department of Medicine
Department of Oncology
HUS Comprehensive Cancer Center
Päiväys: 2018-03
Kieli: eng
Sivumäärä: 10
Kuuluu julkaisusarjaan: Thrombosis and Haemostasis
ISSN: 0340-6245
DOI-tunniste: https://doi.org/10.1055/s-0038-1624581
URI: http://hdl.handle.net/10138/233934
Tiivistelmä: Recombinant factor XIII-A(2) (rFXIII-A(2)) was developed for prophylaxis and treatment of bleeds in patients with congenital FXIII A-subunit deficiency.mentor (TM) 2 (NCT00978380), a multinational, open-label, single-arm, multiple-dosing extension to the pivotal mentor (TM) 1 trial, assessed long-term safety and efficacy of rFXIII-A(2) prophylaxis in eligible patients (patients with severe [<0.05 IU/mL] congenital FXIII subunit A deficiency) aged >= 6 years. Patients received 35IU/kgrFXIII-A(2) (exactdosing) 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-A(2) dose for breakthrough bleeds and withdrawals due to lack of rFXIII-A(2) efficacy. Steady-state pharmacokinetic variables were also summarized. Elective surgery was permitted during the treatment period. Sixty patients were exposed to rFXIII-A(2); their median age was 26.0 years (range: 7.0-77.0). rFXIII-A(2) was 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 levelwas 0.17 IU/mL. Geometric terminal half-life was 13.7 days. rFXIII-A(2) prophylaxis 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-A(2) dose, and four were performed 10 to 21 days after the last dose.
Avainsanat: recombinant FXIII-A(2)
safety
congenital FXIII deficiency
prophylaxis
surgery
FACTOR-XIII DEFICIENCY
BRITISH-COMMITTEE
YOUNG-CHILDREN
PHARMACOKINETICS
CONCENTRATE
MANAGEMENT
DISORDERS
DIAGNOSIS
PLASMA
SAFETY
3121 General medicine, internal medicine and other clinical medicine
Vertaisarvioitu: Kyllä
Tekijänoikeustiedot: cc_by_nc_nd
Pääsyrajoitteet: openAccess
Rinnakkaistallennettu versio: publishedVersion


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