Besremi (ropeginterferon alfa-2b-njft)

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.

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.

The patient maintained CHR and MR for 2 years following treatment discontinuation. We also discuss the potential benefit of ropeg-IFN in low-risk PV patients who were not previously considered candidates for aggressive cytoreductive therapy.

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.

The standard treatment is low-dose aspirin and phlebotomy, with cytoreductive therapy added for high-risk PV. All 3 patients achieved and maintained complete hematologic response with reduced JAK2 V617F allele burden. Ropeg-IFN is an effective and safe therapy for elderly patients with PV that also improves quality of life.

Ongoing treatment with hydroxyurea was substituted with ropeg (week 0: 250 mcg; week 2: 350 mcg; week 4 onwards: 500 mcg every 2 weeks). In conclusion, ropeg was safe and induced CHCR associated with significant molecular responses in patients with early MF. ClinicalTrials.gov Identifier: NCT04988815.

P2, N=35, Not yet recruiting, Jonsson Comprehensive Cancer Center

This is the first study to provide pharmacokinetic evidence of RopegIFN exposure during pregnancy and lactation. These findings support the safety of RopegIFN use in pregnant ET and PV patients and highlight the need for a collaborative registry to collect real-world data and guide management for this high-risk population.

P=N/A, N=319, Active, not recruiting, FROM- Fondazione per la Ricerca Ospedale di Bergamo- ETS | Recruiting --> Active, not recruiting

P=N/A, N=150, Not yet recruiting, Federico II University

P2, N=40, Not yet recruiting, M.D. Anderson Cancer Center