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Combined pituitary hormone deficiencies, genetic forms

ORPHA:95494DiseaseAutosomal dominant, Autosomal recessive, X-linked recessiveInfancy, Neonatal

Ассоциированные гены (7)

FOXA2
forkhead box A2
Disease-causing germline mutation(s) in
OMIM: 600288
POU1F1
POU class 1 homeobox 1
Disease-causing germline mutation(s) in
OMIM: 173110
PROP1
PROP paired-like homeobox 1
Disease-causing germline mutation(s) in
OMIM: 601538
HESX1
HESX homeobox 1
Disease-causing germline mutation(s) in
OMIM: 601802
OTX2
orthodenticle homeobox 2
Disease-causing germline mutation(s) in
OMIM: 600037
LHX4
LIM homeobox 4
Disease-causing germline mutation(s) (loss of function) in
OMIM: 602146
GLI2
GLI family zinc finger 2
Disease-causing germline mutation(s) in
OMIM: 165230

Фенотипы (39)

Облигатный (100%)1
HP:0040075Hypopituitarism
Частый (30–79%)18
HP:0000044Hypogonadotropic hypogonadism
HP:0000141Amenorrhea
HP:0000457Depressed nasal ridge
HP:0000789Infertility
HP:0000824Decreased response to growth hormone stimulation test
HP:0000938Osteopenia
HP:0001510Growth delay
HP:0001943Hypoglycemia
HP:0002615Hypotension
HP:0002920Decreased circulating ACTH level
HP:0008245Pituitary hypothyroidism
HP:0008734Decreased testicular size
HP:0009888Abnormality of secondary sexual hair
HP:0010311Aplasia/Hypoplasia of the breasts
HP:0010626Anterior pituitary agenesis
HP:0010627Anterior pituitary hypoplasia
HP:0012378Fatigue
HP:0040086Abnormal prolactin level
Периодический (5–29%)6
HP:0000823Delayed puberty
HP:0000839Pituitary dwarfism
HP:0002019Constipation
HP:0002750Delayed skeletal maturation
HP:0005625Osteoporosis of vertebrae
HP:0008187Absence of secondary sex characteristics
Очень редкий (1–4%)14
HP:0009099Median cleft palate
HP:0000478Abnormality of the eye
HP:0000609Optic nerve hypoplasia
HP:0001250Seizure
HP:0001274Agenesis of corpus callosum
HP:0001331Absent septum pellucidum
HP:0001360Holoprosencephaly
HP:0004637Decreased cervical spine mobility
HP:0010442Polydactyly
HP:0011297Abnormal digit morphology
HP:0011344Severe global developmental delay
HP:0011755Ectopic posterior pituitary
HP:0012731Ectopic anterior pituitary gland
HP:0100842Septo-optic dysplasia

Эпидемиология (1)

Point prevalence
Unknown
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы