Treatment Patterns of Ropeginterferon Alfa-2b-Njft in the Real-World Setting

Introduction

Ropeginterferon alfa-2b-njft (ropeg) was approved for the treatment of Polycythemia Vera (PV) in US since November 2021. The National Comprehensive Cancer Network (NCCN) guidelines recently recommend ropeg as the preferred cytoreductive regimen in the first-line setting for patients with low-risk PV and a preferred interferon regimen in high-risk PV. The safety, efficacy and dosing of ropeg was established through Phase 1/2 and 3 clinical trials, including dose recommendations until hematologic parameters are stabilized (max dose 500 mcg). However, the treatment patterns, including dose escalation, dose frequency, and line of usage in the real-world setting has not been reported. This study investigates and describes the real-world utilization of ropeg in academic and community healthcare institutions.

Methods

The analysis was conducted using IntegraConnect electronic health record (EHR) data, which included 30,595 PV patients. We used ICD 10 diagnosis code and the NDC code to identify PV patients treated with ropeg. Line of therapy (LOT) was defined as the first identified drug utilized, and other drugs used within 60 days of follow-up. Treatments included hydroxyurea, ruxolitinib, peginterferon alfa-2a, ropeg, interferon alfa-2b, and phlebotomy. Treatment gaps of 400 days for oral drugs and 120 days for Intravenous (IV) injection or Subcutaneous (SC) injected treatments line of therapy (LOT) would advance. We used Student's t-test for hypothesis testing in numerical variables, and analysis was conducted using SAS 9.4.

Results

Of the 30,595 PV patients, 127 were prescribed with ropeg between Dec 2021 and Jan 2024. The average age at diagnosis was 59.0 years (SD=14.2; Median=62), 56 (43.9%) were male, 76 (59.8%) were high-risk patients.

The majority of ropeg patients had multiple LOT (n=103, 81.1%) and were treated in the second LOT (n=60, 47.2%), followed by third LOT(n=36, 28.3%), and first LOT (n=32, 25.2%). Hydroxyurea alone was the most used cytoreductive drug prior to ropeg (n=22, 36.7%) when ropeg was used in second and third LOT.

The average time on therapy for ropeg was 167.9 days (SD=175.4, Median=100). Patients use of ropeg in second LOT had the longest average time on therapy (Mean=195.8 days, Median=152 days) followed by first-line (Mean=156.3 days, Median=92.5 days) and third-line (Mean=155.1 days, Median=92 days). Patients on average were dosed every 17.9 days (SD=13.5, Median=14.0). Of the 127 patients, 36 had verified dosing information. Dose escalations varied and lacked a clear pattern. Of the 36 patients, only 2 (5.6%) reached the maximum dose of 500 mcg/ml.

Combination of treatments were observed among ropeg patients with 40.9% (n=52) receiving ropeg in addition to another cytoreductive agent. The most common combined treatment was with ruxolitinib (n=17), followed by hydroxyurea (n=16), then peginterferon alfa-2a (n=11). Patients in combination treatments stayed on treatment longer compared to those utilizing ropeg only. The average time in treatment for ropeg patients in combination of other drugs was 275.1 days (SD=192.2) compared to ropeg only 107.4 (SD=131.9; p<0.001). The average days in treatment for Ropeg patients with combination of other drugs were 275.1 days (SD=192.2), comparing to ropeg only-treated patients, that which was 107.4 (SD=131.9; p<0.001).

Discussion

Recent updates to NCCN guidelines recommend ropeg as a preferred first line cytoreductive treatment for low-risk and high-risk PV patients and translation into clinical practice is not being optimized in a real-world setting. In addition, a subset of patients with verified dosing noted variable dose titration and very few patients reached maximum per label dosing of ropeg. Enhanced patient management, physician support, and dose escalation might be the key to optimize the treatment of PV with ropeg.