Ruxolitinib

  • An agent that regulates hemoglobin gene expression, used for the treatment of other haematological conditions, such as myelofibrosis and resistant to hydroxyurea polycythemia vera.
  • A Phase 2a trial (TRUTH) in 30 transfusion-dependent β-thalassemia with splenomegaly showed that ruxolitinib therapy for 30 weeks resulted in slight improvement in pre-transfusion haemoglobin levels with a noticeable reduction in spleen size, rendering the drug a promising alternative to splenectomy. The results were published as a research letter in Blood, in Jan. 11, 2018.