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Hereditary pulmonary alveolar proteinosis

ORPHA:264675DiseaseAutosomal recessiveAdolescent, Adult, Childhood, Elderly, Infancy

Ассоциированные гены (2)

CSF2RB
colony stimulating factor 2 receptor subunit beta
Disease-causing germline mutation(s) in
OMIM: 138981
CSF2RA
colony stimulating factor 2 receptor subunit alpha
Disease-causing germline mutation(s) in
OMIM: 306250

Фенотипы (15)

Очень частый (80–99%)1
HP:0010876Abnormal circulating protein level
Частый (30–79%)6
HP:0001531Failure to thrive in infancy
HP:0002091Restrictive ventilatory defect
HP:0002098Respiratory distress
HP:0004887Respiratory failure requiring assisted ventilation
HP:0012418Hypoxemia
HP:0025391Crazy paving pattern on pulmonary HRCT
Периодический (5–29%)8
HP:0001649Tachycardia
HP:0002789Tachypnea
HP:0003651Foam cells
HP:0011949Acute infectious pneumonia
HP:0012735Cough
HP:0030057Autoimmune antibody positivity
HP:0030830Crackles
HP:0031029Elevated carcinoembryonic antigen level

Эпидемиология (1)

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