• Cih-En Huang, Yu-Ying Wu, Chia-Chen Hsu, Ying-Ju Chen, Hsing-Yi Tsou, Chian-Pei Li, Yi-Hua Lai, Chang-Hsien Lu, Ping-Tsung Chen, and Chih-Cheng Chen.
• Division of Hematology and Oncology, Department of Medicine, Chang Gung Memorial Hospital, Chiayi, Taiwan; College of Medicine, Chang Gung University, Tao-Yuan, Taiwan.
• J Formos Med Assoc. 2021 Feb 1; 120 (2): 863-873.

Background/PurposeRopeginterferon alpha-2b (Ropeg) is a novel pegylated interferon-alpha recently approved for the treatment of polycythemia vera (PV) in Europe. However, other than data from clinical trials, little is known about this agent in real world practice.MethodsA compassionate use program employing Ropeg for treating patients with unmet medical need was initiated in Taiwan in 2017. Herein, we collected clinical data and assessed the safety as well as efficacy of Ropeg in nine patients treated in this program.ResultsCollectively, among evaluable patients, both the molecular response and complete blood count remission rates were 62.5%. Most therapy-related side effects were mild, and there was no treatment discontinuation attributable to intolerable adverse events. The agent also showed efficacy in symptom amelioration and spleen size reduction. Although no specific patterns of cytokine level alteration could be identified, significantly attenuated plasma levels of inflammation markers were observed in one particular patient who happened to have normalized spleen size and most remarkable reduction in JAK2 mutant allele burden, indicating all-around improvement in every aspect of this case. Furthermore, plasma hepcidin levels increased in two-thirds of PV patients, illustrating the potential of Ropeg to restore normal regulation of erythropoiesis. Using RNA sequencing on pre- and post-treatment samples from one patient, we demonstrated altered expression of genes participating in IFN response, inflammation, apoptosis, and cellular differentiation.ConclusionConclusively, observed signs of efficacy and safety in our real-world experience prove Ropeg as a promising option for the treatment of MPN.Copyright © 2020 Formosan Medical Association. Published by Elsevier B.V. All rights reserved.

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