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
Reference:
fedratinib for treating disease-related splenomegaly or symptoms in myelofibrosis