Ruxolitinib Effective as Long-term Treatment Option for Polycythemia Vera

Share this content:
Ruxolitinib Effective as Long-term Treatment Option for Polycythemia Vera
Ruxolitinib Effective as Long-term Treatment Option for Polycythemia Vera

Ruxolitinib is an effective long-term treatment option for patients with polycythemia resistant or intolerant to hydroxyurea, according to a study presented at the 2016 European Hematology Association Congress.1

The phase 3 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; Identifier: NCT01243944) is evaluating the efficacy and safety of ruxolitinib vs best available care in hydroxyurea-resistant or intolerant patients with polcythemia vera.

After 80 weeks of follow-up, results showed that no patients remained on best available therapy and 87.5% had crossed over to ruxolitinib, most at or soon after week 32.

The investigators found that 22.7% of patients receiving ruxolitinib had achieved a primary response by week 32 compared with only 0.9% of those randomly assigned to best available therapy; hematocrit control was 60.0% vs 18.8%, respectively.

Results further demonstrated that 92% of ruxolitinib-treated patients maintained their primary response and 89% maintained their hematocrit response for 80 weeks or longer. Of the 44 who achieved a spleen response at week 32, 43 were maintained until week 80.

In addition, 23.6% of patients initially assigned to receive ruxolitinib achieved a complete hematologic remission at week 32, with 69% maintaining complete hematologic remission for 80 weeks or more.

In terms of safety, researchers observed new or worsening anemia, lymphopenia, and thrombocytopenia, which were primarily grade 1 to 2, in ruxolitnib-treated patients.


1. Vannucchi AM, Griesshammer M, Masszi T, et al. Ruxolitinib versus best available therapy in patients with polycythemia vera: 80-week follow-up from the RESPONSE trial. Poster presented at: European Hematology Association 21st Congress; June 9-12, 2016; Copenhagen, Denmark.

You must be a registered member of ONA to post a comment.

Sign Up for Free e-newsletters

Regimen and Drug Listings


Bone Cancer Regimens Drugs
Brain Cancer Regimens Drugs
Breast Cancer Regimens Drugs
Endocrine Cancer Regimens Drugs
Gastrointestinal Cancer Regimens Drugs
Genitourinary Cancer Regimens Drugs
Gynecologic Cancer Regimens Drugs
Head and Neck Cancer Regimens Drugs
Hematologic Cancer Regimens Drugs
Lung Cancer Regimens Drugs
Other Cancers Regimens
Rare Cancers Regimens
Skin Cancer Regimens Drugs