Last update 01 Nov 2024

Factor Xiii, a Subunit, Deficiency Of

Last update 01 Nov 2024

Basic Info

Synonyms

FACTOR XIII, A SUBUNIT, DEFICIENCY OF, Factor XIII subunit A deficiency, Factor Xiii, A Subunit, Deficiency Of

+ [6]

Introduction

Deficiency of factor XIII subunit A, leading to a reduced factor XIII activity. Activated Factor XIII cross-links fibrin polymers solidifying the clot. []

Drugs associated with Factor Xiii, a Subunit, Deficiency Of

Catridecacog

Target

fibrin

Mechanism

fibrin modulators [+1]

Active Org.

Novo Nordisk A/S [+5]

Originator Org.

Novo Nordisk A/S

Active Indication

Factor XIII Deficiency [+2]

Inactive Indication

Colitis, Ulcerative [+3]

Drug Highest PhaseApproved

First Approval Ctry. / Loc.

EU [+3]

First Approval Date03 Sep 2012

Clinical Trials associated with Factor Xiii, a Subunit, Deficiency Of

NCT01253811 / CompletedPhase 3

A Multi-Centre, Multinational, Open-Label, Single-Arm and Multiple Dosing Trial on Safety and Efficacy of Monthly Replacement Therapy With Recombinant Factor XIII (rFXIII) in Paediatric Subjects With Congenital Factor XIII A-subunit Deficiency. Safety Extension Trial to F13CD-3760

This trial will be conducted in Asia, Europe and the United States of America (USA).
The aim of this clinical trial is to investigate long-term safety of rFXIII when administered for prevention of bleeding episodes in children aged between 1 and 6 years with congenital FXIII A-subunit deficiency. This trial is an extension to trial F13CD-3760 (mentor™4, NCT01230021). If applicable the trial will be extended up to maximum 3 years dependent on when recombinant factor XIII will be commercially available in subject's respective country for use in children of 1-6 years of age.

Start Date01 Jan 2011

Sponsor / Collaborator

Novo Nordisk A/S

NCT01230021 / CompletedPhase 3

A Phase 3b Trial Investigating the Pharmacokinetics and Safety Profile of a Single Intravenous Dose of rFXIII in Paediatric (1 to Less Than 6 Years Old) Subjects With Congenital FXIII A-subunit Deficiency

This trial is conducted in Europe and United States of America (USA). The aim of this clinical trial is to investigate the pharmacokinetics (at which rate the substance is distributed and eliminated from the body) and the safety profile of catridecacog (recombinant factor XIII (rFXIII)) in children with congenital FXIII A-subunit deficiency. Young children (1 to less than 6 years old) with congenital FXIII deficiency are evaluated.

Start Date01 Nov 2010

Sponsor / Collaborator

Novo Nordisk A/S

100 Clinical Results associated with Factor Xiii, a Subunit, Deficiency Of

100 Translational Medicine associated with Factor Xiii, a Subunit, Deficiency Of

0 Patents (Medical) associated with Factor Xiii, a Subunit, Deficiency Of

Literatures (Medical) associated with Factor Xiii, a Subunit, Deficiency Of

01 Feb 2020·AtherosclerosisQ2 · MEDICINE

Transglutaminase 2 limits the extravasation and the resultant myocardial fibrosis associated with factor XIII-A deficiency

Q2 · MEDICINE

Article

Author: Newell, Laura M ; Jackson, Christopher L ; Pease, Richard J ; Standeven, Kristina F ; Drinkhill, Mark J ; Simpson, Kingsley R ; Beckers, Cora M L ; Grant, Peter J ; Cheah, Lih T ; Griffin, Kathryn J ; Iismaa, Siiri E

BACKGROUND AND AIMSTransglutaminase (TG) 2 and Factor (F) XIII-A have both been implicated in cardiovascular protection and repair. This study was designed to differentiate between two competing hypotheses: that TG2 and FXIII-A mediate these functions in mice by fulfilling separate roles, or that they act redundantly in this respect.METHODSAtherosclerosis was assessed in brachiocephalic artery plaques of fat-fed mixed strain apolipoprotein (Apo)e deficient mice that lacked either or both transglutaminases. Cardiac fibrosis was assessed both in the mixed strain mice and also in C57BL/6J Apoe expressing mice lacking either or both transglutaminases.RESULTSNo difference was found in the density of buried fibrous caps within brachiocephalic plaques from mice expressing or lacking these transglutaminases. Cardiac fibrosis developed in both Apoe/F13a1 double knockout and F13a1 single knockout mice, but not in Tgm2 knockout mice. However, concomitant Tgm2 knockout markedly increased fibrosis, as apparent in both Apoe/Tgm2/F13a1 knockout and Tgm2/F13a1 knockout mice. Amongst F13a1 knockout and Tgm2/F13a1 knockout mice, the extent of fibrosis correlated with hemosiderin deposition, suggesting that TG2 limits the extravasation of blood in the myocardium, which in turn reduces the pro-fibrotic stimulus. The resulting fibrosis was interstitial in nature and caused only minor changes in cardiac function.CONCLUSIONSThese studies confirm that FXIII-A and TG2 fulfil different roles in the mouse myocardium. FXIII-A protects against vascular leakage while TG2 contributes to the stability or repair of the vasculature. The protective function of TG2 must be considered when designing clinical anti-fibrotic therapies based upon FXIII-A or TG2 inhibition.

01 Jun 2018·Journal of Thrombosis and HaemostasisQ2 · MEDICINE

Recombinant factor XIII A‐subunit in a patient with factor XIII deficiency and recurrent pregnancy loss

Q2 · MEDICINE

Article

Author: Al-Khabori, M ; Pathare, A ; Menegatti, M ; Peyvandi, F

Essentials Inherited factor XIII deficiency is a very rare bleeding disorder. We used recombinant factor XIII-A in a pregnant patient with factor XIII-A subunit deficiency. The patient had a successful pregnancy outcome with no pregnancy related complications. The dose of recombinant factor XIII-A was minimized by using frequent trough level monitoring.SUMMARYInherited factor XIII deficiency is a very rare bleeding disorder, and is one of the causes of recurrent pregnancy loss. The use of plasma-derived FXIII to improve pregnancy outcomes has been reported. We report a 26-year-old woman with FXIII A-subunit (FXIII-A) deficiency who was treated with recombinant FXIII-A and had a successful pregnancy outcome with no pregnancy-related complications. Our case illustrates that the dose of recombinant FXIII-A can be minimized and adjusted on the basis of frequent trough level monitoring.

01 Mar 2016·HaemophiliaQ2 · MEDICINE

Alloantibody developed in a factor XIII A subunit deficient patient during substitution therapy; characterization of the antibody

Q2 · MEDICINE

Article

Author: Rivard, G. E. ; Pénzes, K. ; Katona, É. ; Bereczky, Z. ; Vezina, C. ; Kun, M. ; Muszbek, L.

IntroductionIn factor XIII A subunit (FXIIIA) deficiency, the development of alloantibodies is extremely rare. Only four reports have been published and the antibodies were not characterized.AimThe aim of this study was to describe the clinical course and the laboratory diagnosis of a FXIII‐A deficient patient who developed alloantibodies.MethodsFXIII activity was assessed with an ammonia release assay. FXIII‐A, FXIII B subunit (FXIII‐B) and the complex plasma FXIII (FXIII‐A2B2) antigens were determined by ELISA. The causative mutation was detected by fluorescent DNA sequencing. The binding of alloantibody to FXIII‐A2 and FXIII‐A2B2 was studied by surface plasmon resonance. The cleavage of FXIII‐A by thrombin and Ca2+‐induced activation of thrombin‐cleaved FXIII were followed by western blotting and activity measurement, respectively.ResultsFXIII activity, FXIII‐A2B2 and FXIII‐A antigens were below the limit of detection in the patient's plasma. The severe FXIII‐A deficiency was due to a novel homozygous mutation resulting in early stop codon (c.127C>T, p.Gln42STOP). The alloantibody bound to FXIII‐A2 and FXIII‐A2B2 with equally high affinity (Kd~10−8). It accelerated the elimination of administered FXIII concentrate from the circulation, interfered with thrombin and Ca2+‐induced activation and inhibited FXIII activity. Attempts to eliminate the alloantibody resulted only in transient improvement. Patient developed intracerebral haemorrhage after a minor trauma and died in spite of aggressive replacement therapy with FXIII concentrate.ConclusionThe anti‐FXIII‐A alloantibody caused an unmanageable bleeding complication. The antibody was of combined subtype which accelerated the elimination of FXIII and exerted a multiple inhibitory effect on FXIII activation/activity.

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Factor Xiii, a Subunit, Deficiency Of

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