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WHIM syndrome

ORPHA:51636DiseaseAutosomal dominant, Autosomal recessive, Not applicableAdolescent, Adult, Childhood

Ассоциированные гены (1)

CXCR4
C-X-C motif chemokine receptor 4
Disease-causing germline mutation(s) (gain of function) in
OMIM: 162643

Фенотипы (34)

Очень частый (80–99%)5
HP:0001875Decreased total neutrophil count
HP:0001888Lymphopenia
HP:0011992Abnormality of neutrophil morphology
HP:0031020Bone marrow hypercellularity
HP:0031160Myelokathexis
Частый (30–79%)8
HP:0002090Pneumonia
HP:0002718Recurrent bacterial infections
HP:0002788Recurrent upper respiratory tract infections
HP:0004313Decreased circulating antibody level
HP:0006532Recurrent pneumonia
HP:0011947Respiratory tract infection
HP:0012740Papilloma
HP:0200043Verrucae
Периодический (5–29%)11
HP:0000246Sinusitis
HP:0000388Otitis media
HP:0001636Tetralogy of Fallot
HP:0002070Limb ataxia
HP:0002110Bronchiectasis
HP:0002167Abnormality of speech or vocalization
HP:0002172Postural instability
HP:0002244Abnormality of the small intestine
HP:0007010Poor fine motor coordination
HP:0025439Pharyngitis
HP:0030079Cervix cancer
Очень редкий (1–4%)10
HP:0000166Severe periodontitis
HP:0001045Vitiligo
HP:0001250Seizure
HP:0001287Meningitis
HP:0002840Lymphadenitis
HP:0011850Parotitis
HP:0012056Cutaneous melanoma
HP:0100658Cellulitis
HP:0100750Atelectasis
HP:0100806Sepsis

Эпидемиология (3)

Cases/families
Worldwide
Point prevalence
<1 / 1 000 000
Worldwide
Prevalence at birth
<1 / 1 000 000
France

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы