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Systemic mastocytosis with associated hematologic neoplasm

ORPHA:98849DiseaseNot applicableAdult, Elderly

Ассоциированные гены (4)

KIT
KIT proto-oncogene, receptor tyrosine kinase
Disease-causing somatic mutation(s) in
OMIM: 164920
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

Фенотипы (45)

Очень частый (80–99%)4
HP:0004377Hematological neoplasm
HP:0012324Myeloid leukemia
HP:0031901Increased serum mast cell beta-tryptase concentration
HP:0100494Abnormal mast cell morphology
Частый (30–79%)13
HP:0000980Pallor
HP:0000989Pruritus
HP:0001824Weight loss
HP:0001873Thrombocytopenia
HP:0001880Eosinophilia
HP:0001895Normochromic anemia
HP:0001897Normocytic anemia
HP:0001945Fever
HP:0001974Leukocytosis
HP:0002315Headache
HP:0005547Myeloproliferative disorder
HP:0012378Fatigue
HP:0031020Bone marrow hypercellularity
Периодический (5–29%)25
HP:0000939Osteoporosis
HP:0001025Urticaria
HP:0001279Syncope
HP:0001649Tachycardia
HP:0001744Splenomegaly
HP:0002014Diarrhea
HP:0002018Nausea
HP:0002027Abdominal pain
HP:0002086Abnormality of the respiratory system
HP:0002240Hepatomegaly
HP:0002615Hypotension
HP:0002653Bone pain
HP:0002659Increased susceptibility to fractures
HP:0002665Lymphoma
HP:0002716Lymphadenopathy
HP:0002829Arthralgia
HP:0002863Myelodysplasia
HP:0003326Myalgia
HP:0004398Peptic ulcer
HP:0004808Acute myeloid leukemia
HP:0011897Neutrophilia
HP:0012138Granulocytic hyperplasia
HP:0012325Chronic myelomonocytic leukemia
HP:0031284Flushing
HP:0031807Increased basophil count
Очень редкий (1–4%)3
HP:0005550Chronic lymphatic leukemia
HP:0006775Multiple myeloma
HP:0011034Amyloidosis

Эпидемиология (1)

Point prevalence
1-9 / 100 000
Europe

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы