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Severe hemophilia A

ORPHA:169802Clinical subtypeX-linked recessiveInfancy, Neonatal

Ассоциированные гены (1)

F8
coagulation factor VIII
Disease-causing germline mutation(s) in
OMIM: 300841

Фенотипы (27)

Очень частый (80–99%)2
HP:0003125Reduced factor VIII activity
HP:0003645Prolonged partial thromboplastin time
Частый (30–79%)8
HP:0000421Epistaxis
HP:0000978Bruising susceptibility
HP:0001058Poor wound healing
HP:0001386Joint swelling
HP:0001934Persistent bleeding after trauma
HP:0004846Prolonged bleeding after surgery
HP:0005261Joint hemorrhage
HP:0030140Oral cavity bleeding
Периодический (5–29%)17
HP:0000132Menorrhagia
HP:0001376Limitation of joint mobility
HP:0001903Anemia
HP:0002170Intracranial hemorrhage
HP:0002239Gastrointestinal hemorrhage
HP:0002315Headache
HP:0002829Arthralgia
HP:0003121Limb joint contracture
HP:0005187Progressive joint destruction
HP:0008330Reduced von Willebrand factor activity
HP:0012233Intramuscular hematoma
HP:0012541Cephalohematoma
HP:0012587Macroscopic hematuria
HP:0030137Prolonged bleeding following circumcision
HP:0100309Subdural hemorrhage
HP:0100310Epidural hemorrhage
HP:0100769Synovitis

Эпидемиология (1)

Point prevalence
1-9 / 100 000
Europe

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы