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Last update 08 May 2025

Factor Xiii, a Subunit, Deficiency Of

Last update 08 May 2025

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.

European Union [+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 Mar 2025·Seminars in Thrombosis and Hemostasis

Factor XIII Deficiency: Laboratory, Molecular, and Clinical Aspects

Review

Author: Zadeh, Masoumeh Kiani ; Dorgalaleh, Akbar ; Jozdani, Sina

AbstractFactor XIII-A (FXIII-A) deficiency is an ultra-rare bleeding disorder characterized by high rates of morbidity and mortality, primarily resulting from intracranial hemorrhage, umbilical cord bleeding, and miscarriage, whereas patients with severe FXIII-B deficiency present with a milder phenotype. Although the estimated incidence of severe FXIII-A deficiency is one per 2 million, a high prevalence ranging from 0.8 to 3.5% has been observed for heterozygous FXIII-A deficiency. Unlike most bleeding disorders, individuals with heterozygous FXIII-A deficiency, particularly women, are more likely to experience hemorrhagic complications during hemostatic challenges. About 200 Mutations have been observed in F13A and F13B genes, with most being missense mutations, while large deletions are the rarest. There is no correlation between genotype and phenotype and a moderate to strong correlation between factor activity and clinical severity in FXIII-A deficiency, making it difficult to predict bleeding patterns based on genotype and FXIII activity levels. Primary prophylaxis is mandatory for all patients with severe FXIII-A deficiency, while those with heterozygous deficiency are generally asymptomatic and may require on-demand therapy during hemostatic challenges, most commonly in women. On the other hand, patients with severe FXIII-B deficiency may only require on-demand therapy, while heterozygotes are generally asymptomatic. Although there are general recommended therapeutic regimens for prophylaxis or on-demand therapy in different situations, personalized pharmacokinetic-based replacement therapy represents the optimal approach that can optimize intervention efficacy. In such an approach, several factors may affect the effectiveness of treatment and determine the dose and type of intervention, including the classification of FXIII deficiency, residual plasma levels of FXIII, clinical situation requiring intervention, age, weight, and also gender.

01 Sep 2021·BMJ Case Reports

Patient-centered approach to managing factor XIII deficiency

Article

Author: Iyengar, Varun ; Montcrieff, Caitlin ; Pels, Salley

Factor XIII (FXIII) is a thrombin-activated protransglutaminase that plays a key role in blood clot formation. Congenital FXIII A-subunit deficiency represents a rare bleeding disorder that affects one in 2–3 million individuals worldwide and is treated with recombinant FXIII (rFXIII). However, due to the rarity of the disease, clinicians are often left to weigh individual variation in FXIII activity and/or symptoms to optimally guide dosing. Cases often become further complicated when patients experience refractory bleeding, which can be difficult to treat. This report describes an approach to rFXIII dosing in two patients who required deviation from standard protocols to maintain therapeutic FXIII troughs. We highlight limitations in our understanding of FXIII deficiency management, while also providing an example of the application of pharmacokinetic data to individualise therapy for improved outcomes. Finally, the case reminds us of the importance of patient-centered, cost-conscious care and multidisplinary teamwork in complex cases.

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 ; Beckers, Cora M L ; Simpson, Kingsley R ; 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.

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