Cancer Treatment Today

This FDA indicated for polycythemia vera in patients who have had an inadequate response to or are intolerant of hydroxyurea. The National Comprehensive Cancer Network (NCCN) added myeloproliferative neoplasms as a whole to the guidelines in the fall of 2016. Treatment guidelines for polycythemia vera were added in the summer of 2017. The NCCN supports the first-line treatment with hydroxyurea, after phlebotomy.
n randomized studies of patients with polycythemia vera, ruxolitinib was superior to the best alternative ther­apy in controlling red cell counts, decreasing vascular events, improving symptoms, and reducing splenomegaly. The RESPONSE trial (Study of Efficacy and Safety in Polycythemia Vera Subjects Who Are Resistant to or Intolerant of Hydroxyurea: JAK Inhibitor INC424 [INCB018424] Tablets Versus Best Available Care) eval­uated ruxolitinib in patients with an inadequate res­ponse to hydroxyurea and who had splenomegaly and inadequately controlled disease. The primary endpoint was the proportion of patients with both control of hematocrit levels and a reduction in spleen volume of at least 35% from baseline to week 32. This endpoint was achieved in 21% of patients treated with ruxolitinib vs 1% of those treated with standard therapy (P<.001). Ruxolitinib was also superior to best available therapy in improving symptom burden and decreasing the need for phlebotomy.

The more recent RESPONSE-2 trial (Ruxolitinib Efficacy and Safety in Patients With HU Resistant or Intolerant Polycythemia Vera vs Best Available Therapy) evaluated ruxolitinib in patients with an inadequate response to hydroxyurea and inadequately controlled disease, but who did not have splenomegaly. Ruxolitinib was similarly effective in this population.isslinger H, Klade C, Georgiev P, et al. Final results from PROUD-PV a randomized controlled phase 3 trial comparing ropeginterferon alfa-2b to hydroxyurea in polycythemia vera patients. [ASH abstract 475]. Blood. 2016;128(suppl 22).

National Comprehensive Cancer Network. NCCN Clinical Practice Guidelines in Oncology: myelo­proliferative neoplasms. Version 2.2018. https://www.nccn.org/professionals/physician_gls/pdf/mpn.pdf. Updated September 7, 2017. Accessed September 26, 2017.

Passamonti F, Griesshammer M, Palandri F, et al. Ruxolitinib for the treat­ment of inadequately controlled polycythaemia vera without splenomegaly (RESPONSE-2): a randomised, open-label, phase 3b study. Lancet Oncol. 2017;18(1):88-99.

Vannucchi AM, Kiladjian JJ, Griesshammer M, et al. Ruxolitinib versus standard therapy for the treatment of polycythemia vera. N Engl J Med. 2015;372(5):
426-435.

Verstovsek S, Vannucchi AM, Griesshammer M, et al. Ruxolitinib versus best available therapy in patients with polycythemia v