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X-linked agammaglobulinemia

ORPHA:47Clinical subtypeX-linked recessiveChildhood

Ассоциированные гены (1)

BTK
Bruton tyrosine kinase
Disease-causing germline mutation(s) in
OMIM: 300300

Фенотипы (35)

Очень частый (80–99%)17
HP:0000162Glossoptosis
HP:0000246Sinusitis
HP:0000389Chronic otitis media
HP:0000509Conjunctivitis
HP:0000988Skin rash
HP:0001508Failure to thrive
HP:0001945Fever
HP:0002028Chronic diarrhea
HP:0002721Immunodeficiency
HP:0004322Short stature
HP:0004432Agammaglobulinemia
HP:0006532Recurrent pneumonia
HP:0012378Fatigue
HP:0100763Abnormality of the lymphatic system
HP:0100765Abnormality of the tonsils
HP:0100838Recurrent cutaneous abscess formation
HP:0200042Skin ulcer
Частый (30–79%)8
HP:0000407Sensorineural hearing impairment
HP:0001287Meningitis
HP:0001369Arthritis
HP:0001875Decreased total neutrophil count
HP:0002088Abnormal lung morphology
HP:0002901Hypocalcemia
HP:0100658Cellulitis
HP:0100806Sepsis
Периодический (5–29%)10
HP:0001053Hypopigmented skin patches
HP:0001596Alopecia
HP:0001824Weight loss
HP:0001873Thrombocytopenia
HP:0001903Anemia
HP:0002024Malabsorption
HP:0002664Neoplasm
HP:0002754Osteomyelitis
HP:0002960Autoimmunity
HP:0012115Hepatitis

Эпидемиология (19)

Point prevalence
1-9 / 1 000 000
Europe
Point prevalence
1-9 / 1 000 000
Worldwide
Point prevalence
1-9 / 1 000 000
France
Point prevalence
1-9 / 1 000 000
Italy
Point prevalence
<1 / 1 000 000
United Kingdom
Point prevalence
<1 / 1 000 000
Spain
Point prevalence
<1 / 1 000 000
Germany
Point prevalence
<1 / 1 000 000
Poland
Point prevalence
1-9 / 1 000 000
Croatia
Point prevalence
1-9 / 1 000 000
Hungary
Point prevalence
1-9 / 1 000 000
North Macedonia
Point prevalence
1-9 / 1 000 000
Slovenia
Point prevalence
<1 / 1 000 000
Serbia
Point prevalence
<1 / 1 000 000
Ukraine
Point prevalence
<1 / 1 000 000
Belarus
Point prevalence
<1 / 1 000 000
Romania
Prevalence at birth
1-9 / 1 000 000
United States
Point prevalence
<1 / 1 000 000
United States
Point prevalence
1-9 / 1 000 000
Korea, Republic of

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы