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Li-Fraumeni syndrome

ORPHA:524DiseaseAutosomal dominantAll ages

Ассоциированные гены (4)

CDKN2A
cyclin dependent kinase inhibitor 2A
Candidate gene tested in
OMIM: 600160
MDM2
MDM2 proto-oncogene
Modifying germline mutation in
OMIM: 164785
TP53
tumor protein p53
Disease-causing germline mutation(s) in
OMIM: 191170
CHEK2
checkpoint kinase 2
Candidate gene tested in
OMIM: 604373

Фенотипы (35)

Очень частый (80–99%)1
HP:0002664Neoplasm
Частый (30–79%)1
HP:0003002Breast carcinoma
Периодический (5–29%)14
HP:0001909Leukemia
HP:0002665Lymphoma
HP:0002669Osteosarcoma
HP:0002859Rhabdomyosarcoma
HP:0002888Ependymoma
HP:0006744Adrenocortical carcinoma
HP:0007378Neoplasm of the gastrointestinal tract
HP:0009592Astrocytoma
HP:0012126Stomach cancer
HP:0012174Glioblastoma multiforme
HP:0030070Central primitive neuroectodermal tumor
HP:0030392Choroid plexus carcinoma
HP:0100006Neoplasm of the central nervous system
HP:0200063Colorectal polyposis
Очень редкий (1–4%)19
HP:0002861Melanoma
HP:0002863Myelodysplasia
HP:0002885Medulloblastoma
HP:0002890Thyroid carcinoma
HP:0002894Neoplasm of the pancreas
HP:0003003Colon cancer
HP:0004808Acute myeloid leukemia
HP:0006721Acute lymphoblastic leukemia
HP:0009726Renal neoplasm
HP:0010788Testicular neoplasm
HP:0012125Prostate cancer
HP:0012189Hodgkin lymphoma
HP:0012288Neoplasm of head and neck
HP:0012539Non-Hodgkin lymphoma
HP:0100526Neoplasm of the lung
HP:0100605Neoplasm of the larynx
HP:0100615Ovarian neoplasm
HP:0100743Neoplasm of the rectum
HP:0100768Choriocarcinoma

Эпидемиология (3)

Point prevalence
1-9 / 100 000
United Kingdom
Point prevalence
1-9 / 100 000
United States
Point prevalence
Unknown
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы