treatment

Last edited 12/2021 and last reviewed 12/2021

Treatment is palliative, and may include:

  • for anaemia
    • blood transfusions for anaemia
    • administration of recombinant erythropoietin may be beneficial
    • androgens - e.g. oxymetholone, nandrolone or danazol - to reduce transfusion requirement; but poorly tolerated by women
  • for splenomegaly
    • therapy should be considered only in patients who are truly symptomatic (1)
    • hydroxyurea, - most commonly
      • most commonly used initial medical therapy in splnomegaly
      • to reduce hypermetabolism, leucocytosis and thrombocytosis
      • adverse effects include myelosuppression which may exacerbate the underlying anaemia (1)
    • alkylating agents - busulphan, melphalan or chlorambucil
      • can be used in some patients with splenomegaly although there is a risk of potential myelosuppression and increased risk of eventual blastic transformation (1)
  • for both anaemia and splenomegaly
    • agents which improves splenomegaly do so by non specific myelosuppression effects
    • immunomodulatory drugs (IMiDs) have shown that it has  the ability to improve both the conditions e.g. -  thalidomide, lenalidomide and pomalidomide (investigational agent) (1)
  • splenectomy is indicated if:
    • the spleen becomes very large and painful
    • transfusion requirement are high
    • thrombocytopenia is severe
  • newer drugs
    • immunomodulatory drugs (IMiDs)
    • hypomethylating agents - azacitidine and decitabine
    • JAK2 Inhibitors  (1,2)
      • the identification of driver mutations in JAK2, CALR, and MPL has contributed to a better understanding of disease pathogenesis, implicating near-universal upregulation of JAK-STAT signaling, and has led to the development of the sole targeted therapy for MF, the JAK2 inhibitor ruxolitinib

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