Jakafi and its uses in treating polycythemia vera
Polycythemia vera is a type of blood cancer that causes the bone marrow to make too many red blood cells (RBCs). This overproduction leads to thickening of the blood, which can increase the risk of clots over time. Itchiness, numbness in the hands or feet, bloating and pain due to an enlarged spleen, unusual bleeding, and joint swelling are common symptoms. Although rare and slow-progressing, the condition can be life-threatening without proper treatment.
Treatment for polycythemia vera
While there are no cures for the condition, doctors recommend different treatments to reduce the symptoms and the risk of complications. For example, regular blood withdrawals (or phlebotomy) may be advised to remove excess blood cells. If this does not help, the expert may recommend treatments like Jakafi (ruxolitinib) to reduce the number of red blood cells (RBCs) in the body.
Working mechanism of Jakafi
Jakafi (ruxolitinib) is the first FDA-approved treatment for adults with polycythemia vera who do not show improvement with other treatments. Often recommended for bone marrow disorders, Jakafi works by blocking the production of growth factors in the body, thus controlling hematocrit levels (the percentage of RBC volume).
With time, Jakafi helps reduce the severity and occurrence of symptoms like abdominal discomfort, pain under the ribs, early satiation from meals, night sweats, itching, and muscle or bone pain.
Jakafi trial results
To test its efficacy, Jakafi was pitched against other standard therapies for polycythemia vera. During the tests, it was found to be effective in controlling hematocrit levels while simultaneously reducing the spleen size by at least 35%. These results improved over time.
- After eight months of treatment, 23% of patients showed controlled hematocrit levels without a phlebotomy compared to just 1% in the other group.
- As the study reached its primary endpoint, these numbers had increased to 60% and 19%, respectively, showcasing the treatment’s effectiveness in controlling blood cell count. It was also found that only 1% of patients saw a reduction in spleen size with other treatments compared to 40% of those who used Jakafi.
- By the trial’s secondary endpoint, researchers noted that Jakafi more effectively achieved the combined goal of reducing hematocrit and controlling white blood cell (WBC) and platelet count than other therapies. At this point, an efficacy rate of 24% was noted among those prescribed Jakafi, compared to 8% among those undergoing other therapies.
Long-term effects of Jakafi
To study the long-term effects of Jakafi, researchers also conducted additional studies approximately at the 20-month mark. During this time, it was found that 76% of people who had achieved their primary goal (hematocrit control and spleen size reduction) at the eight-month mark maintained it even after 20 months.
It is important to note that every person with polycythemia vera has unique needs. One should talk to a healthcare professional to determine if Jakafi is right for their current situation and devise a treatment plan accordingly.