From the Guidelines
Ruxolitinib (Jakafi) is the most effective treatment option for patients with polycythemia vera who are resistant or intolerant to hydroxyurea, as evidenced by the RESPONSE trials 1. The use of ruxolitinib has been shown to improve hematocrit response and primary response in these patients, with a moderate level of evidence overall 1. Some key points to consider when using ruxolitinib include:
- Typical starting dose: 10 mg twice daily
- Dose adjustments based on response and tolerability
- Maximum dose: 25 mg twice daily
- Mechanism of action: inhibition of the JAK-STAT signaling pathway
- Common side effects: anemia, thrombocytopenia, bruising, dizziness, and headache
- Importance of regular blood count monitoring during treatment Other options, such as pegylated interferon alfa (Pegasys) and ropeginterferon alfa-2b (Besremi), may be considered for younger patients or those in need of cytoreductive therapy who cannot use hydroxyurea 1. However, ruxolitinib remains the preferred choice for patients who have had an inadequate response to or are intolerant of hydroxyurea, due to its proven efficacy and safety profile 1.
From the Research
New Treatments for Polycythemia Vera (PV)
Aside from Hydroxyurea (Hydrea), several new treatments have been developed for Polycythemia Vera (PV). Some of these treatments include:
- Ruxolitinib, a Janus kinase (JAK) 1 and 2 inhibitor, which has been shown to be effective in controlling hematocrit levels, reducing spleen volume, and improving symptoms in patients with PV who are resistant to or intolerant of hydroxyurea 2, 3, 4, 5, 6
- Interferon or pegylated interferon, which can be used as an alternative to hydroxyurea in some patients 2, 3
- Pipobroman, anagrelide, and approved immunomodulators, which can be used as part of best available therapy in patients with PV who are resistant to or intolerant of hydroxyurea 2, 3
Efficacy and Safety of Ruxolitinib
Ruxolitinib has been shown to be effective in achieving hematocrit control, reducing spleen volume, and improving symptoms in patients with PV. The efficacy and safety of ruxolitinib have been demonstrated in several clinical trials, including the RESPONSE and RESPONSE-2 trials 2, 3, 5, 6. Some of the key findings from these trials include:
- Ruxolitinib was superior to best available therapy in achieving hematocrit control and reducing spleen volume in patients with PV who were resistant to or intolerant of hydroxyurea 2, 3, 5, 6
- Ruxolitinib was effective in improving symptoms and reducing the risk of thromboembolic events in patients with PV 2, 3, 5, 6
- The most common adverse events associated with ruxolitinib were anemia, thrombocytopenia, and herpes zoster infection 2, 3, 5, 6
Long-Term Efficacy and Safety of Ruxolitinib
The long-term efficacy and safety of ruxolitinib have been evaluated in several studies, including the RESPONSE trial and a 10-year review of clinical efficacy and safety 2, 4, 5. Some of the key findings from these studies include:
- Ruxolitinib was effective in maintaining hematocrit control and reducing spleen volume over a period of 5 years in patients with PV who were resistant to or intolerant of hydroxyurea 2
- The probability of maintaining primary composite response, complete hematological remission, and overall clinicohaematological response was high in patients treated with ruxolitinib 2
- The long-term safety profile of ruxolitinib was consistent with the known safety profile of the drug, with the most common adverse events being anemia, thrombocytopenia, and herpes zoster infection 2, 4, 5