Dr. Raajit Rampal at ASH 2017, Single Arm Salvage Therapy with Pegasys for High Risk PV or ET Patients, Final Results from MPD-RC111 Study

Fourth in ASH 2017 Series

by David Wallace

This is part 2 of the interview with Dr. Rampal.  In this segment we discuss “Single Arm Salvage Therapy with Pegasys for High Risk PV or ET Patients, Final results from the MPD-RC111 Study.”

Summary of Key Points:

  • The purpose of the study was to take Polycythemia Vera (PV) or Essential Thrombocythemia (ET) patients who had taken Hydroxyurea (HU), but were intolerant or resistant to it, and see if they responded to treatment with pegylated interferon.
  • Results showed that these patients are responsive to treatment with interferon, which opens up treatment options to patients unable to benefit from the long-standard treatment of HU.
  • Interferon has been shown to reduce the allele burden of mutated JAK2 or CALR, the two main driver mutations of MPNs. Some patients in the study had a ten-fold or 100-fold reduction in mutated genes.
  • The meaning of this reduction is unclear— does it affect the length of remission or inhibit the progression of the disease.
  • Local hematologists can still be reluctant to prescribe interferon, possibly because of the difficulty in getting insurance approval, or discomfort with monitoring patients with regard to kidney, liver, and thyroid function, and vision, which can be adversely affected by interferon treatment.
  • MPN experts at major centers go around the country to smaller centers to speak to local hematologists and educate them about current research on the use of interferon therapy.
  • Most important findings at ASH 2017 include the study of progression in MPNs, the ongoing research into interferon treatment, and follow-up on the of the Ropeginterferon (ROPEG) treatment study in Europe.
  • ROPEG treatment looks very promising, but may not be available in the U.S. until more research is done here.


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