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Langerhans cell histiocytosis

ORPHA:389DiseaseUnknownAll ages

Ассоциированные гены (3)

MAP2K1
mitogen-activated protein kinase kinase 1
Disease-causing germline mutation(s) in
OMIM: 176872
NRAS
NRAS proto-oncogene, GTPase
Disease-causing somatic mutation(s) in
OMIM: 164790
BRAF
B-Raf proto-oncogene, serine/threonine kinase
Disease-causing somatic mutation(s) in
OMIM: 164757

Фенотипы (27)

Очень частый (80–99%)2
HP:0002653Bone pain
HP:0002797Osteolysis
Частый (30–79%)7
HP:0000964Eczematoid dermatitis
HP:0000988Skin rash
HP:0001744Splenomegaly
HP:0001945Fever
HP:0002240Hepatomegaly
HP:0002716Lymphadenopathy
HP:0012735Cough
Периодический (5–29%)15
HP:0000155Oral ulcer
HP:0000365Hearing impairment
HP:0000873Diabetes insipidus
HP:0001251Ataxia
HP:0001260Dysarthria
HP:0001268Mental deterioration
HP:0001510Growth delay
HP:0001824Weight loss
HP:0001873Thrombocytopenia
HP:0001882Leukopenia
HP:0002108Spontaneous pneumothorax
HP:0003073Hypoalbuminemia
HP:0003249Genital ulcers
HP:0034323Reduced circulating growth hormone concentration
HP:0034769Pulmonary Langerhans cell histiocytosis
Очень редкий (1–4%)3
HP:0001399Hepatic failure
HP:0002093Respiratory insufficiency
HP:0030991Sclerosing cholangitis

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