Dr. Jean Jacques Kiladjian at ASH 2017, 4 Year Follow up on RESPONSE Trial, a Phase 3 Study Comparing Ruxolitinib with Best Available Therapy for Treatment of Polycythemia Vera

Fifth in ASH 2017 Series

by David Wallace

This is part 2 of the interview with Dr. Kiladjian.  In this segment, we discuss “Results from 4-Year Follow-up of RESPONSE Trial, a Phase 3 Study Comparing Ruxolitinib (Rux) with Best Available Therapy (BAT) for the Treatment of Polycythemia Vera.”  Part 1 of the interview can be found here.

Summary of Key Points:

  • The purpose of the RESPONSE trial is to compare Ruxolitinib to best available treatment (BAT) in Polycythemia Vera (PV) patients that are resistant/intolerant of Hydroxyurea (HU). Findings presented at ASH were on the 4-year (208-week) follow-up.
  • At 32 weeks, there was shown to be an increase in risk of infection and non-melanoma skin cancer with the use of Ruxolitinib compared to BAT.
  • It is important to remember that all these patients had previously received long-term HU therapy, which causes skin toxicities.
  • In the RESPONSE trial at 4 years, the risk of infection decreased over time, and was lower than at the 80-week analysis.
  • After 4 years on Ruxolitinib, the incidence of non-melanoma skin cancer was stable, but there was a high correlation in patients that developed skin lesions with those who had a history of lesions before Ruxolitinib treatment.
  • It is important to monitor patients on long-term Ruxolitinib therapy for skin lesions, especially if they have a history of lesions, of HU treatment, or live in a southern climate.
  • A common adverse effect of Ruxolitinib is weight gain, which can be beneficial to some patients (especially with Myelofibrosis) who have lost too much weight because of an enlarged spleen.
  • In PV patients who are not underweight, this weight gain can be troubling, and doctors should be more aware of this adverse effect and try to mange it.
  • Clinicohematologic response (CLHM) is defined as a combination of complete hematological response on blood counts and a reduction of spleen size. Most patients maintained CLHM at 4 years, showing durable efficacy.
  • Overall survival rate at 4 years on Ruxolitinib is above 90%, which is encouraging, because PV patients who fail on HU can have a poor prognosis.


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