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Nephroblastoma

ORPHA:654DiseaseAutosomal dominant, Not applicableChildhood

Ассоциированные гены (12)

BRCA2
BRCA2 DNA repair associated
Candidate gene tested in
OMIM: 600185
GPC3
glypican 3
Biomarker tested in
OMIM: 300037
CTR9
CTR9 component of Paf1/RNA polymerase II complex
Disease-causing germline mutation(s) in
OMIM: 609366
TRIM28
tripartite motif containing 28
Disease-causing germline mutation(s) in
OMIM: 601742
TRIM28
tripartite motif containing 28
Disease-causing somatic mutation(s) in
OMIM: 601742
REST
RE1 silencing transcription factor
Major susceptibility factor in
OMIM: 600571
TRIP13
thyroid hormone receptor interactor 13
Disease-causing germline mutation(s) (loss of function) in
OMIM: 604507
POU6F2
POU class 6 homeobox 2
Major susceptibility factor in
OMIM: 609062
WT1
WT1 transcription factor
Disease-causing somatic mutation(s) in
OMIM: 607102
WT1
WT1 transcription factor
Major susceptibility factor in
OMIM: 607102
H19
H19 imprinted maternally expressed transcript
Role in the phenotype of
OMIM: 103280
DIS3L2
DIS3 like 3'-5' exoribonuclease 2
Major susceptibility factor in
OMIM: 614184

Фенотипы (27)

Очень частый (80–99%)3
HP:0002664Neoplasm
HP:0002667Nephroblastoma
HP:0031500Abdominal mass
Частый (30–79%)3
HP:0002027Abdominal pain
HP:0005580Duplication of renal pelvis
HP:0012587Macroscopic hematuria
Периодический (5–29%)20
HP:0000028Cryptorchidism
HP:0000047Hypospadias
HP:0000085Horseshoe kidney
HP:0000086Ectopic kidney
HP:0000526Aniridia
HP:0000822Hypertension
HP:0001528Hemihypertrophy
HP:0001824Weight loss
HP:0001901Polycythemia
HP:0001903Anemia
HP:0001945Fever
HP:0002716Lymphadenopathy
HP:0002896Neoplasm of the liver
HP:0002907Microscopic hematuria
HP:0003072Hypercalcemia
HP:0008330Reduced von Willebrand factor activity
HP:0012871Varicocele
HP:0031105Abnormal uterus morphology
HP:0033834Malaise
HP:0100526Neoplasm of the lung
Очень редкий (1–4%)1
HP:0002094Dyspnea

Эпидемиология (27)

Annual incidence
1-9 / 1 000 000
Europe
Point prevalence
1-9 / 100 000
Europe
Lifetime Prevalence
1-9 / 100 000
Europe
Annual incidence
1-9 / 1 000 000
United Kingdom
Prevalence at birth
1-5 / 10 000
Europe
Annual incidence
<1 / 1 000 000
Bulgaria
Annual incidence
<1 / 1 000 000
Czech Republic
Annual incidence
<1 / 1 000 000
Germany
Annual incidence
<1 / 1 000 000
Iceland
Annual incidence
<1 / 1 000 000
Latvia
Annual incidence
<1 / 1 000 000
Portugal
Annual incidence
1-9 / 1 000 000
Austria
Annual incidence
1-9 / 1 000 000
Belgium
Annual incidence
1-9 / 1 000 000
Croatia
Annual incidence
1-9 / 1 000 000
Estonia
Annual incidence
1-9 / 1 000 000
Finland
Annual incidence
1-9 / 1 000 000
Ireland
Annual incidence
1-9 / 1 000 000
Italy
Annual incidence
1-9 / 1 000 000
Lithuania
Annual incidence
1-9 / 1 000 000
Malta
Annual incidence
1-9 / 1 000 000
Norway
Annual incidence
1-9 / 1 000 000
Poland
Annual incidence
1-9 / 1 000 000
Slovakia
Annual incidence
1-9 / 1 000 000
Slovenia
Annual incidence
1-9 / 1 000 000
Spain
Annual incidence
1-9 / 1 000 000
Switzerland
Annual incidence
1-9 / 1 000 000
Netherlands

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы