Ropeginterferon Alfa-2B Demonstrates Efficacy Among High-Risk Patients With Philadelphia-Negative Myeloproliferative Neoplasms

Results from a Retrospective Observational Study

Findings from a retrospective observational study show that ropeginterferon alfa-2b demonstrated high response rates and tolerable safety among young high-risk patients with Philadelphia chromosome-negative myeloproliferative neoplasms (Ph-negative MPNs). 

Myeloproliferative neoplasms, including polycythemia vera (PV), essential thrombocytopenia (ET), and primary myelofibrosis (MF), “requires individualized approach depending on multiple factors,” stated Marija Popova-Labachevska, MD, Ss. Cyril and Methodius University, Skopje, RN Macedonia, and coauthors. This study aimed to measure the efficacy and safety of novel pegylated interferon (IFN) formulation ropeginterferon alpha-2b among young high-risk patients with Ph-negative MPN. 

In this study, 16 high-risk patients (identified using the International Prognostic Score of Thrombosis in Essential Thrombocythemia [IPSET] score) with Ph-negative MPN were enrolled and administered ropeginterferon alfa-2b twice monthly off-label. The average age at diagnosis was 36 years, ranging from 17 to 51 years. Among the patients enrolled, 75% (n = 12) had ET, 6.2% (n = 1) had PV, and 18.8% (n = 3) had primary MF in the hypercellular phase. 

A total of 7 patients received ropeginterferon alfa-2b in the first line, and 9 patients had been previously treated with hydroxyurea (HU) and/or standard IFN.  Response to treatment was measured using the International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) and European Leukemia Net (ELN) standardized criteria. 

Results indicated that the complete hematological response among the 7 initially ropeginterferon alfa-2b treated patients was 57.1% (n = 4), with partial response in 28.6% (n = 2) and suboptimal response in 14.3% (n = 1). Among the 9 patients previously treated with HU and/or IFN, 88.9% (n = 8) achieved a complete hematological response. The average time to blood count normalization was 8 weeks, using a dose ranging from 100 mcg to 300 mcg. Side effects were observed in 1 patient. 

“Our experience is in support of previous studies regarding ropeginterferon alfa-2b efficacy and safety profile in the treatment of young patients with Ph-[negative] MPNs,” the study authors concluded. 

Source: 

Popova-Labachevska M, Cvetanoski M, Ridova N, et al. Effectiveness of ropeginterferon alfa-2b in high-risk patients with Philadelphia chromosome negative myeloproliferative neoplasms- evaluation of clinicohaematologic response, and safety profile: single centre experience. Pril (Makedon Akad Nauk Umet Odd Med Nauki). Published online December 18, 2023. doi:10.2478/prilozi-2023-0047