Ropeginterferon alfa-2b-njft (ropeg), a mono-PEGylated interferon-α, showed efficacy and safety in patients with hydroxyurea-intolerant/resistant essential thrombocythemia (ET) in the phase 3 SURPASS-ET largely conducted in Asia. Ropeg showed efficacy and substantial molecular responses with good overall tolerability across a broad ET population. PharmaEssentia.

P1/2, N=30, Not yet recruiting, M.D. Anderson Cancer Center

P1, N=20, Completed, PharmaEssentia | Trial completion date: May 2025 --> Apr 2026 | Trial primary completion date: May 2025 --> Apr 2026

Phase III data from PROUD-PV (NCT01949805; EudraCT, 2012-005259-18) and its phase IIIb extension, CONTINUATION-PV (NCT02218047; EudraCT, 2014-001357-17), demonstrate that ropeg, compared with hydroxyurea, achieves hematologic response more slowly but provides greater, more durable responses after ∼18 months, underscoring the importance of long-term adherence. Early adverse events can challenge adherence, emphasizing the need for proactive symptom management. This review offers evidence- and experience-based perspectives on long-term ropeg treatment and adverse event management to support adherence and maximize therapeutic benefit in PV.

P2, N=35, Not yet recruiting, Jonsson Comprehensive Cancer Center | Trial completion date: Mar 2032 --> Sep 2032 | Initiation date: Mar 2026 --> Sep 2026 | Trial primary completion date: Mar 2031 --> Sep 2031

P3, N=1673, Active, not recruiting, National Cancer Institute (NCI) | Trial completion date: Mar 2026 --> Feb 2027

P2, N=88, Active, not recruiting, National Cancer Institute (NCI) | Trial completion date: Mar 2026 --> Mar 2027

P1, N=15, Not yet recruiting, University of Utah

The PROUD-CONTI study showed the superiority of rIFNα compared to hydroxyurea (HU), which led to the European Medicines Agency approval in 2019 of ropeginterferon alpha-2b ("ropegIFN") for ELN-defined high-risk PV patients. Moreover, as PV progresses, the development of myelofibrosis is the leading cause of morbidity, perhaps abetted by PHLEB-O. Here, we review recent progress in the treatment of PV with rIFNα and discuss our rationales and perspectives for, and the endorsement of the initial treatment with rIFNα of both low and high-risk PV patients, unless a contraindication exists to its use.

Our findings redefine epigenetic regulation in LUAD, demonstrating that regional methylation patterns-not global promoter status-orchestrate oncogene activation. We propose a novel framework for spatially resolved methylomics, advocating 1) dual-target assays monitoring both promoter and gene body methylation to improve diagnostic precision, and 2) therapeutic exploitation of SHOX2's intronic methylome as a druggable epigenetic switch.

NLR half reduction significantly predicted hematologic response (week 24 OR 6.42, p = 0.001) and molecular response consistently across all time points (week 24 OR 27.94, p < 0.001). NLR half reduction is a simple, cost-effective biomarker that may reflect molecular response and treatment efficacy in PV.

Clinical trials have demonstrated that ropeg-IFN reduces the JAK2 V617F allele burden, which is recognized as an independent risk factor for disease progression and thrombosis, apart from traditional factors such as age and prior cardiovascular events. To improve the prognosis of PV, it is essential to accumulate clinical evidence on trends in JAK2 V617F allele burden and the conditions required to maintain hematologic control after discontinuation of ropeg-IFN therapy in real-world settings.