Rare Disease Report

Treating Polycythemia Vera Patients Refractory to Hydroxyurea

SEPTEMBER 13, 2016
Srdan Verstovsek, MD, PhD

Verstovsek: This patient is definitely a candidate for a new therapy and a new therapy is, in fact, ruxolitinib, which is a targeted therapy for what is abnormal in polycythemia vera. We know that in polycythemia vera we have hyperactive intracellular single pathway, called JAK-STAT pathway, which is active in most patients because of the JAK-2 mutation. Now ruxolitinib does not act on a mutation; it’s not a JAK 2 mutation driven drug. The consequence of mutations in a JAK 2 gene are increased activities of a JAK stat pathway in the bone marrow cells. And this is a JAK inhibitor. Jakafi or Ruxolitinib is a JAK inhibitor so it inhibits the hyperactive intercellular single pathway and that leads to a decrease in the cell growth, decrease in inflammation that comes with the disease. That leads to improvement in the quality of life, control of the counts, and obviously possible and we’ve at least heard hints that it’s possible, a decrease in thromboembolic events.
In a case study of a  45-year old man with polycythemia vera is refractory to hydroxyurea, Srdan Verstovsek, MD, PhD of the MD Anderson Cancer Center in Houston, Texas describes how ruxolitinib is a viable treatment option.

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