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Hereditary pheochromocytoma-paraganglioma

ORPHA:29072DiseaseAutosomal dominantChildhood

Ассоциированные гены (15)

SLC25A11
solute carrier family 25 member 11
Disease-causing germline mutation(s) in
OMIM: 604165
KIF1B
kinesin family member 1B
Disease-causing germline mutation(s) (loss of function) in
OMIM: 605995
MDH2
malate dehydrogenase 2
Disease-causing germline mutation(s) in
OMIM: 154100
DLST
dihydrolipoamide S-succinyltransferase
Disease-causing germline mutation(s) in
OMIM: 126063
RET
ret proto-oncogene
Candidate gene tested in
OMIM: 164761
SDHA
succinate dehydrogenase complex flavoprotein subunit A
Disease-causing germline mutation(s) in
OMIM: 600857
SDHC
succinate dehydrogenase complex subunit C
Disease-causing germline mutation(s) in
OMIM: 602413
SDHD
succinate dehydrogenase complex subunit D
Disease-causing germline mutation(s) in
OMIM: 602690
FH
fumarate hydratase
Disease-causing germline mutation(s) (loss of function) in
OMIM: 136850
SDHB
succinate dehydrogenase complex iron sulfur subunit B
Disease-causing germline mutation(s) in
OMIM: 185470
TMEM127
transmembrane protein 127
Candidate gene tested in
OMIM: 613403
MAX
MYC associated transcriptional regulator X
Disease-causing germline mutation(s) in
OMIM: 154950
NF1
neurofibromin 1
Disease-causing germline mutation(s) in
OMIM: 613113
VHL
von Hippel-Lindau tumor suppressor
Disease-causing germline mutation(s) in
OMIM: 608537
SDHAF2
succinate dehydrogenase complex assembly factor 2
Disease-causing germline mutation(s) in
OMIM: 613019

Фенотипы (41)

Очень частый (80–99%)3
HP:0002668Paraganglioma
HP:0006737Extraadrenal pheochromocytoma
HP:0006748Adrenal pheochromocytoma
Частый (30–79%)25
HP:0000093Proteinuria
HP:0000096Glomerulosclerosis
HP:0000740Episodic paroxysmal anxiety
HP:0001069Episodic hyperhidrosis
HP:0001095Hypertensive retinopathy
HP:0001342Cerebral hemorrhage
HP:0001618Dysphonia
HP:0001824Weight loss
HP:0001962Palpitations
HP:0002018Nausea
HP:0002331Recurrent paroxysmal headache
HP:0002574Episodic abdominal pain
HP:0002640Hypertension associated with pheochromocytoma
HP:0002864Paraganglioma of head and neck
HP:0003072Hypercalcemia
HP:0003345Elevated urinary norepinephrine
HP:0003574Positive regitine blocking test
HP:0003639Elevated urinary epinephrine
HP:0008629Pulsatile tinnitus
HP:0010532Paroxysmal vertigo
HP:0011703Sinus tachycardia
HP:0011979Elevated urinary dopamine
HP:0012378Fatigue
HP:0031284Flushing
HP:0100749Chest pain
Периодический (5–29%)12
HP:0000405Conductive hearing impairment
HP:0000790Hematuria
HP:0000980Pallor
HP:0001293Cranial nerve compression
HP:0001337Tremor
HP:0001605Vocal cord paralysis
HP:0001635Congestive heart failure
HP:0003528Elevated calcitonin
HP:0005584Renal cell carcinoma
HP:0009711Retinal capillary hemangioma
HP:0012222Arachnoid hemangiomatosis
HP:0025269Panic attack
Очень редкий (1–4%)1
HP:0000526Aniridia

Эпидемиология (2)

Annual incidence
1-9 / 1 000 000
Worldwide
Point prevalence
1-9 / 1 000 000
Europe

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы