Treatment

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

Reference:

• (1) Mesa RA. New drugs for the treatment of myelofibrosis. Curr Hematol Malig Rep. 2010;5(1):15-21.
• (2) Schieber M, Crispino JD, Stein B. Myelofibrosis in 2019: moving beyond JAK2 inhibition. Blood Cancer J. 2019;9(9):74. Published 2019 Sep 11. doi:10.1038/s41408-019-0236-2