DISEASE SCANNER

Global Incurable Diseases Tracker

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Hematologic Disorder

Myelofibrosis

HIGH SEVERITY

A rare myeloproliferative neoplasm where scar tissue forms in the bone marrow, disrupting normal blood cell production. Can be primary or secondary to polycythemia vera or essential thrombocythemia. Risk of progression to acute leukemia.

Global Affected

180.0K

Countries

15

Symptoms

Fatigue
Enlarged spleen (splenomegaly)
Weight loss
Night sweats
Fever
Bone/joint pain
Shortness of breath
Easy bruising
Abdominal fullness

Treatment Options

Transfusions
Iron supplementation
Vitamin supplementation
Erythropoietin
Immunosuppressants
Chemotherapy
Stem cell transplant
Gene therapy

Risk Factors

1Genetic mutations
2Family history
3Autoimmune disease
4Nutritional deficiencies
5Chronic disease
6Medications
7Environmental exposures

Diagnostic Methods

  • 1Complete blood count
  • 2Peripheral smear
  • 3Bone marrow biopsy
  • 4Coagulation studies
  • 5Hemoglobin electrophoresis
  • 6Flow cytometry
  • 7Genetic testing

Prognosis

Median survival 3.5-5.5 years for primary myelofibrosis. Dynamic International Prognostic Scoring System (DIPSS) predicts outcome. Low risk: median 15.4 years; high risk: median 1.3 years. JAK inhibitors (ruxolitinib) reduce symptoms and spleen size but modest survival benefit. Allogeneic stem cell transplant is only curative option with 30-50% survival at 5 years. Transformation to acute leukemia occurs in 10-20% with very poor prognosis. Anemia is common and challenging to manage.

Prevention

  • Genetic counseling
  • Nutritional adequacy
  • Avoidance of triggers
  • Vaccinations
  • Regular monitoring
  • Family screening

Research Status

JAK inhibitors (ruxolitinib, fedratinib) improve symptoms and survival. Allogeneic stem cell transplant only curative. Splenectomy for massive splenomegaly. Supportive care with transfusions.

Sources

  • https://www.hematology.org
  • https://medlineplus.gov/blooddiseases.html
  • https://www.hematology.org/education/patients
  • https://www.ncbi.nlm.nih.gov/books
  • https://rarediseases.org/rare-diseases

Medical Disclaimer

This information is for educational purposes only. Always consult healthcare professionals for medical advice, diagnosis, and treatment.