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

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dc.contributor.author Carcao, Manuel
dc.contributor.author Altisent, Carmen
dc.contributor.author Castaman, Giancarlo
dc.contributor.author Fukutake, Katsuyuki
dc.contributor.author Kerlin, Bryce A.
dc.contributor.author Kessler, Craig
dc.contributor.author Lassila, Riitta
dc.contributor.author Nugent, Diane
dc.contributor.author Oldenburg, Johannes
dc.contributor.author Garly, May-Lill
dc.contributor.author Rosholm, Anders
dc.contributor.author Inbal, Aida
dc.date.accessioned 2018-04-05T07:30:01Z
dc.date.available 2018-04-05T07:30:01Z
dc.date.issued 2018-03
dc.identifier.citation 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
dc.identifier.other PURE: 103030592
dc.identifier.other PURE UUID: 74816a2a-da89-4474-9172-209d2649d78f
dc.identifier.other WOS: 000427285600003
dc.identifier.other Scopus: 85042182125
dc.identifier.uri http://hdl.handle.net/10138/233934
dc.description.abstract 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. en
dc.format.extent 10
dc.language.iso eng
dc.relation.ispartof Thrombosis and Haemostasis
dc.rights cc_by_nc_nd
dc.rights.uri info:eu-repo/semantics/openAccess
dc.subject recombinant FXIII-A(2)
dc.subject safety
dc.subject congenital FXIII deficiency
dc.subject prophylaxis
dc.subject surgery
dc.subject FACTOR-XIII DEFICIENCY
dc.subject BRITISH-COMMITTEE
dc.subject YOUNG-CHILDREN
dc.subject PHARMACOKINETICS
dc.subject CONCENTRATE
dc.subject MANAGEMENT
dc.subject DISORDERS
dc.subject DIAGNOSIS
dc.subject PLASMA
dc.subject SAFETY
dc.subject 3121 General medicine, internal medicine and other clinical medicine
dc.title Recombinant FXIII (rFXIII-A(2)) Prophylaxis Prevents Bleeding and Allows for Surgery in Patients with Congenital FXIII A-Subunit Deficiency en
dc.type Article
dc.contributor.organization Clinicum
dc.contributor.organization Hematologian yksikkö
dc.contributor.organization Department of Medicine
dc.contributor.organization Department of Oncology
dc.contributor.organization HUS Comprehensive Cancer Center
dc.description.reviewstatus Peer reviewed
dc.relation.doi https://doi.org/10.1055/s-0038-1624581
dc.relation.issn 0340-6245
dc.rights.accesslevel openAccess
dc.type.version publishedVersion

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