Orphan designation: Human coagulation factor X, Treatment of hereditary factor X deficiency

SummaryMonospecific antisera against the human coagulation factor X have been raised in rabbits by injections of purified antigen. Such antiserum was used to study the cross-reacting material without factor X activity which is present in the blood of warfarin-treated patients and animals as well as to study the changes in factor X during coagulation. One patient with congenital factor X deficiency was also studied.A complete identity was found between factor X in Macaca mulatta and human blood. During warfarin treatment antigenically cross-reacting material appeared in plasma. This was not adsorbed on BaSO4, and inhibited the coagulation activity of normal factor X.Both this material, normal factor X and the cross-reacting material in plasma from a patient congenitally deficient in factor X gave rise to split products during coagulation by the intrinsic pathway, i. e. all of them served as substrates for the intrinsic activator of factor X.

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Human medicines European public assessment report (EPAR): Coagadex, human coagulation factor X, Factor X Deficiency, Date of authorisation: 16/03/2016, Revision: 3, Status: Authorised

Case Medical Research ◽

10.31525/cmr-5871bc ◽

2019 ◽

Author(s):

Keyword(s):

Coagulation Factor ◽

Factor X ◽

Assessment Report ◽

Factor X Deficiency ◽

European Public ◽

European Public Assessment Report

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Experience of a new high-purity factor X concentrate in subjects with hereditary factor X deficiency undergoing surgery

Haemophilia ◽

10.1111/hae.12954 ◽

2016 ◽

Vol 22 (5) ◽

pp. 713-720 ◽

Cited By ~ 12

Author(s):

M. A. Escobar ◽

G. Auerswald ◽

S. Austin ◽

J. N. Huang ◽

M. Norton ◽

...

Keyword(s):

High Purity ◽

Factor X ◽

Hereditary Factor ◽

Factor X Deficiency

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Use of a High-Purity Factor X Concentrate in Turkish Subjects with Hereditary Factor X Deficiency: Post Hoc Cohort Subanalysis of a Phase 3 Study

Turkish Journal of Hematology ◽

10.4274/tjh.2017.0446 ◽

2018 ◽

Vol 35 (2) ◽

pp. 129-133

Author(s):

Ahmet F. Öner ◽

Tiraje Celkan ◽

Çetin Timur ◽

Miranda Norton ◽

Kaan Kavaklı

Keyword(s):

High Purity ◽

Factor X ◽

Phase 3 ◽

Hereditary Factor ◽

Factor X Deficiency ◽

Post Hoc

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A Phenome-Wide Association Study Uncovers a Pathological Role of Coagulation Factor X duringAcinetobacter baumanniiInfection

Infection and Immunity ◽

10.1128/iai.00031-19 ◽

2019 ◽

Vol 87 (5) ◽

Cited By ~ 3

Author(s):

Jacob E. Choby ◽

Andrew J. Monteith ◽

Lauren E. Himmel ◽

Paris Margaritis ◽

Jana K. Shirey-Rice ◽

...

Keyword(s):

Immune Response ◽

Association Study ◽

Immune Cell ◽

Coagulation Factor ◽

Factor X ◽

Human Pathogens ◽

Chemokine Production ◽

Content Type ◽

Factor X Deficiency ◽

Deficient Mice

ABSTRACTCoagulation and inflammation are interconnected, suggesting that coagulation plays a key role in the inflammatory response to pathogens. A phenome-wide association study (PheWAS) was used to identify clinical phenotypes of patients with a polymorphism in coagulation factor X. Patients with this single nucleotide polymorphism (SNP) were more likely to be hospitalized with hemostatic and infection-related disorders, suggesting that factor X contributes to the immune response to infection. To investigate this, we modeled infections by human pathogens in a mouse model of factor X deficiency. Factor X-deficient mice were protected from systemicAcinetobacter baumanniiinfection, suggesting that factor X plays a role in the immune response toA. baumannii. Factor X deficiency was associated with reduced cytokine and chemokine production and alterations in immune cell population during infection: factor X-deficient mice demonstrated increased abundance of neutrophils, macrophages, and effector T cells. Together, these results suggest that factor X activity is associated with an inefficient immune response and contributes to the pathology ofA. baumanniiinfection.

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Acquired Factor X Deficiency in a Negro Boy

PEDIATRICS ◽

10.1542/peds.44.6.1007 ◽

1969 ◽

Vol 44 (6) ◽

pp. 1007-1009

Author(s):

William L. Bayer ◽

Dario Curiel C. ◽

Isabel L. F. Szeto ◽

Jessica H. Lewis

Keyword(s):

Coagulation Factor ◽

Hemorrhagic Disease ◽

Vitamin K1 ◽

Factor X ◽

Factor X Deficiency

A Negro boy suffering from severe hemorrhagic disease associated with a single acquired defect in coagulation factor X is presented. No cause for this deficiency could be found. Vitamin K1 had no effect. He improved spontaneously and is completely healthy 6 months later.

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A family with hereditary factor X deficiency with a point mutation Gla32 to Gln in the Gla domain (factor X Tokyo)

British Journal of Haematology ◽

10.1046/j.1365-2141.1999.01614.x ◽

1999 ◽

Vol 106 (3) ◽

pp. 809-811 ◽

Cited By ~ 15

Author(s):

Takeru Zama ◽

Mitsuru Murata ◽

Reiko Watanabe ◽

Kenji Yokoyama ◽

Takanori Moriki ◽

...

Keyword(s):

Point Mutation ◽

Factor X ◽

Hereditary Factor ◽

Factor X Deficiency ◽

Gla Domain

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Blood Coagulation Factor X Deficiency Causes Partial Embryonic Lethality and Fatal Neonatal Bleeding in Mice

Thrombosis and Haemostasis ◽

10.1055/s-0037-1613783 ◽

2000 ◽

Vol 83 (02) ◽

pp. 185-190 ◽

Cited By ~ 120

Author(s):

Mieke Dewerchin ◽

Zhong Liang ◽

Lieve Moons ◽

Peter Carmeliet ◽

Francis Castellino ◽

...

Keyword(s):

Blood Coagulation ◽

Coagulation Factor ◽

Massive Bleeding ◽

Embryonic Lethality ◽

Genotype Distribution ◽

Bleeding Disorders ◽

Factor X ◽

Factor X Deficiency ◽

Effective Models ◽

The Brain

SummaryMice with a total deficiency in blood coagulation Factor X (FX) were generated by targeted replacement of an 18-kb fragment of the FX gene, comprising all exons encoding the mature FX protein, with a neor cassette. The genotype distribution among the offspring from heterozygous breeding pairs suggested that FX deficiency resulted in partial embryonic lethality, with approximately one-third of the FX −/− embryos dying around embryonic day (E) 11.5-12.5. Two of 44 non-resorbed FX −/− embryos analyzed at these stages showed signs of massive bleeding, one of which into the brain ventricles, but no histological defects in the vasculature of these embryos or their yolk sac were observed. The remainder of the FX −/− embryos appeared normal and survived to term, but the majority of neonates (90%) died within 5 days, most frequently from intraabdominal bleeding. The remaining FX −/− animals succumbed between postnatal day (P)5 and P20 with intraabdominal, subcutaneous, or intracranial bleeding or a combination thereof. The lethal phenotype of the FX −/− mice illustrates the importance of FX function in embryonic and postnatal survival and demonstrates that these mice serve as effective models of the bleeding disorders observed in severe FX deficiency in humans.

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