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Primary myelofibrosis

ORPHA:824DiseaseNot applicableAdult

Ассоциированные гены (4)

JAK2
Janus kinase 2
Disease-causing somatic mutation(s) in
OMIM: 147796
MPL
MPL proto-oncogene, thrombopoietin receptor
Disease-causing somatic mutation(s) in
OMIM: 159530
TET2
tet methylcytosine dioxygenase 2
Disease-causing somatic mutation(s) in
OMIM: 612839
CALR
calreticulin
Disease-causing somatic mutation(s) in
OMIM: 109091

Фенотипы (36)

Очень частый (80–99%)1
HP:0005561Abnormality of bone marrow cell morphology
Частый (30–79%)10
HP:0000980Pallor
HP:0001433Hepatosplenomegaly
HP:0001744Splenomegaly
HP:0001871Abnormality of blood and blood-forming tissues
HP:0001873Thrombocytopenia
HP:0001903Anemia
HP:0002240Hepatomegaly
HP:0012143Abnormal megakaryocyte morphology
HP:0012378Fatigue
HP:0025142Constitutional symptom
Периодический (5–29%)20
HP:0000967Petechiae
HP:0000979Purpura
HP:0001409Portal hypertension
HP:0001876Pancytopenia
HP:0001892Abnormal bleeding
HP:0001894Thrombocytosis
HP:0001945Fever
HP:0001974Leukocytosis
HP:0001977Abnormal thrombosis
HP:0001978Extramedullary hematopoiesis
HP:0002039Anorexia
HP:0002716Lymphadenopathy
HP:0003388Easy fatigability
HP:0004420Arterial thrombosis
HP:0004447Poikilocytosis
HP:0004936Venous thrombosis
HP:0011134Low-grade fever
HP:0030157Flank pain
HP:0031020Bone marrow hypercellularity
HP:0031364Ecchymosis
Очень редкий (1–4%)4
HP:0001028Hemangioma
HP:0004326Cachexia
HP:0004377Hematological neoplasm
HP:0025435Increased circulating lactate dehydrogenase concentration
Исключён (0%)1
HP:0030057Autoimmune antibody positivity

Эпидемиология (2)

Annual incidence
1-9 / 100 000
Europe
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