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Combined deficiency of factor V and factor VIII

ORPHA:35909DiseaseAutosomal recessiveAll ages

Ассоциированные гены (2)

MCFD2
multiple coagulation factor deficiency 2, ER cargo receptor complex subunit
Disease-causing germline mutation(s) in
OMIM: 607788
LMAN1
lectin, mannose binding 1
Disease-causing germline mutation(s) (loss of function) in
OMIM: 601567

Фенотипы (18)

Очень частый (80–99%)4
HP:0003125Reduced factor VIII activity
HP:0003225Reduced coagulation factor V activity
HP:0003645Prolonged partial thromboplastin time
HP:0008151Prolonged prothrombin time
Частый (30–79%)6
HP:0000225Gingival bleeding
HP:0000421Epistaxis
HP:0000978Bruising susceptibility
HP:0006298Prolonged bleeding after dental extraction
HP:0011889Bleeding with minor or no trauma
HP:0030137Prolonged bleeding following circumcision
Периодический (5–29%)6
HP:0000132Menorrhagia
HP:0000790Hematuria
HP:0002170Intracranial hemorrhage
HP:0002239Gastrointestinal hemorrhage
HP:0004846Prolonged bleeding after surgery
HP:0005261Joint hemorrhage
Очень редкий (1–4%)2
HP:0002149Hyperuricemia
HP:0003077Hyperlipidemia

Эпидемиология (2)

Point prevalence
1-9 / 1 000 000
Europe
Point prevalence
1-9 / 100 000
Iran, Islamic Republic of

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы