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Hereditary combined deficiency of vitamin K-dependent clotting factors

ORPHA:98434DiseaseAutosomal recessiveAdolescent, Adult, Infancy, Neonatal

Ассоциированные гены (2)

GGCX
gamma-glutamyl carboxylase
Disease-causing germline mutation(s) in
OMIM: 137167
VKORC1
vitamin K epoxide reductase complex subunit 1
Disease-causing germline mutation(s) in
OMIM: 608547

Фенотипы (31)

Частый (30–79%)10
HP:0000978Bruising susceptibility
HP:0001892Abnormal bleeding
HP:0003645Prolonged partial thromboplastin time
HP:0004855Reduced protein S activity
HP:0005543Reduced protein C activity
HP:0008151Prolonged prothrombin time
HP:0008169Reduced factor VII activity
HP:0008321Reduced factor X activity
HP:0011858Reduced factor IX activity
HP:0040226Decreased level of heparin co-factor II
Периодический (5–29%)16
HP:0002621Atherosclerosis
HP:0004646Hypoplasia of the nasal bone
HP:0005261Joint hemorrhage
HP:0006118Shortening of all distal phalanges of the fingers
HP:0010655Epiphyseal stippling
HP:0011890Prolonged bleeding following procedure
HP:0011891Post-partum hemorrhage
HP:0012541Cephalohematoma
HP:0030137Prolonged bleeding following circumcision
HP:0031364Ecchymosis
HP:0000132Menorrhagia
HP:0000421Epistaxis
HP:0000939Osteoporosis
HP:0000973Cutis laxa
HP:0001102Angioid streaks of the fundus
HP:0002170Intracranial hemorrhage
Очень редкий (1–4%)5
HP:0001629Ventricular septal defect
HP:0001631Atrial septal defect
HP:0002239Gastrointestinal hemorrhage
HP:0004415Pulmonary artery stenosis
HP:0011884Abnormal umbilical stump bleeding

Эпидемиология (2)

Cases/families
Worldwide
Point prevalence
<1 / 1 000 000
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы