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Aggressive systemic mastocytosis

ORPHA:98850DiseaseNot applicableAdult, Antenatal, Elderly, Neonatal

Ассоциированные гены (5)

TET2
tet methylcytosine dioxygenase 2
Disease-causing somatic mutation(s) in
OMIM: 612839
SRSF2
serine and arginine rich splicing factor 2
Disease-causing somatic mutation(s) in
OMIM: 600813
ASXL1
ASXL transcriptional regulator 1
Disease-causing somatic mutation(s) in
OMIM: 612990
RUNX1
RUNX family transcription factor 1
Disease-causing somatic mutation(s) in
OMIM: 151385
CBL
Cbl proto-oncogene
Disease-causing somatic mutation(s) in
OMIM: 165360

Фенотипы (39)

Очень частый (80–99%)1
HP:0100494Abnormal mast cell morphology
Частый (30–79%)21
HP:0000939Osteoporosis
HP:0000989Pruritus
HP:0001433Hepatosplenomegaly
HP:0001824Weight loss
HP:0001974Leukocytosis
HP:0002014Diarrhea
HP:0002024Malabsorption
HP:0002027Abdominal pain
HP:0002039Anorexia
HP:0002615Hypotension
HP:0002653Bone pain
HP:0002716Lymphadenopathy
HP:0002829Arthralgia
HP:0003155Elevated circulating alkaline phosphatase concentration
HP:0012378Fatigue
HP:0025142Constitutional symptom
HP:0031284Flushing
HP:0031408Increased proportion of CD25+ mast cells
HP:0031901Increased serum mast cell beta-tryptase concentration
HP:0032155Abdominal cramps
HP:0100845Anaphylactic shock
Периодический (5–29%)17
HP:0001025Urticaria
HP:0001409Portal hypertension
HP:0001410Decreased liver function
HP:0001541Ascites
HP:0001873Thrombocytopenia
HP:0001875Decreased total neutrophil count
HP:0001876Pancytopenia
HP:0001903Anemia
HP:0001909Leukemia
HP:0001971Hypersplenism
HP:0002239Gastrointestinal hemorrhage
HP:0002756Pathologic fracture
HP:0002797Osteolysis
HP:0004377Hematological neoplasm
HP:0008066Abnormal blistering of the skin
HP:0011121Abnormal skin morphology
HP:0040186Maculopapular exanthema

Эпидемиология (1)

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