TRIAL COMPARING ROPEGINTERFERON VERSUS PHLEBOTOMY IN LOW-RISK PATIENTS WITH POLYCYTHEMIA VERA

[Manouche]

library.ehaweb.org/eha/2020...

Results

The primary composite endpoint was reached in 84% of cases on Ropeginterferon vs. 60% in the standard arm (Odds Ratio=3.5, 95% CI: 1.3-10.4, p=0.008) and included patients who maintained the HCT target (84% vs. control 66%, p=0.038) and those without disease progression, which occurred in standard arm only (8%) (Figure 1). This result allowed to stop the trial early for overwhelming efficacy, preserving the overall two-sided type I error rate for effectiveness at the 0.05 level. Thus, the steering committee and the DSMB decided to stop the enrolment of new patients yet continuing the 2-years follow-up, as for protocol.

Albeit secondary endpoints will be evaluated at the end of the trial, preliminary results showed that the total number of phlebotomies after 1 year was higher in the standard arm (57%) than in the Ropeginterferon arm (43%) and the difference was most evident starting 6 months after the drug exposure (p=0.024). Improvements in symptoms, as assessed by using the MPN symptom-assessment form, were seen in 7 out of 10 items (mean change -21%) in the Ropeginterferon arm whereas a worsening in half of items was noted in the standard arm (mean change +10%) (p=0.033). Among the other valuable secondary end-points, Ropeginterferon was associated with significant reduction of splenomegaly and leukocyte and platelet counts.

In regard to safety, there was no difference in grade >3 AEs, which accounted for 6% and 8% of patients treated with Ropeginterferon or standard therapy, respectively.

[hunter5582]

Interesting and encouraging results. Ropeg is under review by the FDA here in the USA. Hopefully will be available in the near future. I was unable to get into the article. Did the authors report out a comparison of the side effects of Ropeg vs Phlebotomy alone? If so, what did the risk/benefit analysis for each look like?

Thanks for posting this.

[Manouche in reply to hunter5582]

As far as I know they only evaluated the safety which was equivalent on both arms.

«.In regard to safety, there was no difference in grade >3 AEs, which accounted for 6% and 8% of patients treated with Ropeginterferon or standard therapy, respectively. »

[Chaz1]

Thank you for sharing, very interesting & does make you wonder if they might move to this in low risk patients. I expect it will come down to money but I'd like to see this move forward as it shows improvement in symptoms.