Efficacy and safety of pegylated interferon alfa-2b in myeloproliferative neoplasms: A multicenter retrospective study of 210 patients
Year: 2025
Type: conference-abstract
Abstract: Background： Pegylated interferon alfa-2b (Peg-IFN-α-2b) may induce molecular remission in myeloproliferative neoplasms (MPN) through JAK-STAT pathway modulation, but real-world evidence regarding its long-term efficacy and safety profile requires further validation. Objective： To evaluate hematologic overall response rate (ORR), molecular response rate (MRR), and treatment-emergent adverse events (AEs) incidence of Peg-IFN-α-2b in Chinese MPN patients. Methods： This retrospective study enrolled 210 MPN patients (183 essential thrombocythemia [ET], 20 polycythemia vera [PV], 7 primary myelofibrosis [PMF]) treated with Peg-IFN-α-2b across 10 centers (2016-2025). Primary endpoints included ORR (complete response [CR] and partial response [PR]) at 6 and 12 months, and MRR (complete molecular response [CMR] and partial molecular response [PMR]) at 12 months. Secondary endpoints covered MPN-SAF TSS improvement and AEs incidence (CTCAE v5.0). Results： Median age was 55 years (range 18-80), with 78.1% harboring JAK2 V617F mutation. At 6 months (n=177), ORR was 97.74% (CR rate 75.14%); at 12 months (n=113), ORR increased to 99.12% (CR rate 83.04%). Among 41 molecularly-evaluated patients, 97.6% showed variant allele frequency (VAF) reduction, with MRR of 63.41% (all PMR). MPN-SAF TSS assessment (n=166) demonstrated symptom improvement in 74.1% (65.85% with ≥10-point reduction), with median scores decreasing from 11.0/18.5 to 1.0/2.0 in ET/PV patients. AEs occurred in 53.33% of patients, including hepatic dysfunction (15.24%), hematologic toxicity (14.29%), and neurologic symptoms (6.67%). Grade ≥3 AEs incidence was 3.8%, with AE-related discontinuation rate of 3.8%. Conclusion： Peg-IFN-α-2b demonstrates sustained high hematologic and molecular response rates, significant symptom improvement, and manageable safety in Chinese MPN patients, particularly those with ET/PV. Keywords： Myeloproliferative neoplasms; Pegylated interferon alfa-2b; Molecular response; Real-world evidence
Source: Blood
Institutions: Anshan Hospital, Shandong University, Qilu Hospital of Shandong University, Yuhuangding Hospital, Liaocheng People's Hospital, Weihai Municipal Hospital, Affiliated Hospital of Qingdao University, Shandong Provincial Hospital, Shandong First Medical University, Jining First People's Hospital, Central Hospital of Zibo
Language: English
Subfield: Genetics
Field: Medicine
Domain: Health Sciences
Open Access status: bronze
OpenAlex ID: W4417012857
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