PMID- 37344895 OWN - NLM STAT- PubMed-not-MEDLINE LR - 20230701 IS - 2162-3619 (Print) IS - 2162-3619 (Electronic) IS - 2162-3619 (Linking) VI - 12 IP - 1 DP - 2023 Jun 21 TI - A new dosing regimen of ropeginterferon alfa-2b is highly effective and tolerable: findings from a phase 2 study in Chinese patients with polycythemia vera. PG - 55 LID - 10.1186/s40164-023-00415-0 [doi] LID - 55 AB - Ropeginterferon alfa-2b represents a new-generation pegylated interferon-based therapy and is administered every 2-4 weeks. It is approved for polycythemia vera (PV) treatment in the United States and Europe with a starting dose of 100 microg (50 microg for patients receiving hydoxyurea) and intra-patient dose titrations up to 500 microg at 50 microg increments, which took approximately 20 or more weeks to reach a plateau dose level. This study aimed to assess ropeginterferon alfa-2b at an alternative dosing regimen with a higher starting dose and quicker intra-patient dose titrations, i.e., the 250-350-500 mug schema, in 49 Chinese patients with PV with resistance or intolerance to hydroxyurea. The primary endpoint of the complete hematologic response rate at treatment weak 24 was 61.2%, which was notably higher than 43.1% at 12 months with the approved dosing schema. The JAK2(V617F) allele burden decreased from baseline to week 24 (17.8% +/- 18.0%), with one patient achieving a complete molecular response. Ropeginterferon alfa-2b was well-tolerated and most adverse events (AEs) were mild or moderate. Common AEs included alanine aminotransferase and aspartate aminotransferase increases mostly at grade 1 or 2 levels. Patients did not present with jaundice or significant bilirubin level increase. No grade 4 or 5 AEs occurred. Seven patients (14.3%) experienced reversible, drug-related grade 3 AEs. No AEs led to treatment discontinuation. Ropeginterferon alfa-2b at the 250-350-500 mug regimen is highly effective and well-tolerated and can help patients achieve greater and rapid complete hematologic and molecular responses.Clinical Trial Registration: This trial is registered at ClinicalTrials.gov (Identifier: NCT05485948) and in China (China National Medical Products Administration Registration Number: CTR20211664). CI - (c) 2023. The Author(s). FAU - Jin, Jie AU - Jin J AD - Department of Hematology, The First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, Zhejiang, China. jiej0503@zju.edu.cn. FAU - Zhang, Lei AU - Zhang L AD - State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, 300020, China. zhanglei1@ihcams.ac.cn. FAU - Qin, Albert AU - Qin A AD - Medical Research & Clinical Operations, PharmaEssentia Corporation, Taipei, Taiwan, Republic of China. albert_qin@pharmaessentia.com. FAU - Wu, Daoxiang AU - Wu D AD - PharmaEssentia Biotech (Beijing) Limited, Beijing, China. FAU - Shao, Zonghong AU - Shao Z AD - The Second Hospital of Tianjin Medical University, Tianjin, China. FAU - Bai, Jie AU - Bai J AD - The Second Hospital of Tianjin Medical University, Tianjin, China. FAU - Chen, Suning AU - Chen S AD - The First Affiliated Hospital of Soochow University, Suzhou, Jiangsu, China. FAU - Duan, Minghui AU - Duan M AD - Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China. FAU - Zhou, Hu AU - Zhou H AD - Affiliated Cancer Hospital of Zhengzhou University and Henan Cancer Hospital, Zhengzhou, Henan, China. FAU - Xu, Na AU - Xu N AD - Nanfang Hospital of Southern Medical University, Guangzhou, Guangdong, China. FAU - Zhang, Sujiang AU - Zhang S AD - Ruijin Hospital, Shanghai Jiaotong University School of Medicine, Shanghai, China. FAU - Zuo, Xuelan AU - Zuo X AD - Zhongnan Hospital, Wuhan University, Wuhan, Hubei, China. FAU - Du, Xin AU - Du X AD - Shenzhen Second People's Hospital, Shenzhen, Guangdong, China. FAU - Wang, Li AU - Wang L AD - The First Affiliated Hospital of Chongqing Medical University, Chongqing, China. FAU - Li, Pei AU - Li P AD - Huashan Hospital of Fudan University, Shanghai, China. FAU - Zhang, Xuhan AU - Zhang X AD - Division of Life Sciences and Medicine, The First Affiliated Hospital of USTC, University of Science and Technology of China, Hefei, China. FAU - Li, Yaning AU - Li Y AD - PharmaEssentia Biotech (Beijing) Limited, Beijing, China. FAU - Zhang, Jingjing AU - Zhang J AD - PharmaEssentia Biotech (Beijing) Limited, Beijing, China. FAU - Wang, Wei AU - Wang W AD - PharmaEssentia Biotech (Beijing) Limited, Beijing, China. FAU - Shen, Weihong AU - Shen W AD - PharmaEssentia Biotech (Beijing) Limited, Beijing, China. FAU - Zagrijtschuk, Oleh AU - Zagrijtschuk O AD - PharmaEssentia USA Corporation, Burlington, MA, USA. FAU - Urbanski, Raymond AU - Urbanski R AD - PharmaEssentia USA Corporation, Burlington, MA, USA. FAU - Sato, Toshiaki AU - Sato T AD - PharmaEssentia Japan K. K, Motoakasaka, Minato-Ku, Tokyo, Japan. FAU - Xiao, Zhijian AU - Xiao Z AD - Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China. LA - eng SI - ClinicalTrials.gov/NCT05485948 PT - Letter DEP - 20230621 PL - England TA - Exp Hematol Oncol JT - Experimental hematology & oncology JID - 101590676 PMC - PMC10286412 OTO - NOTNLM OT - Complete hematologic response OT - Molecular response, JAK2 V617F allele burden OT - New dosing regimen, Chinese patients with polycythemia vera OT - Ropeginterferon alfa-2b COIS- JJ, LZ, ZS, JBi, SC, MD, HZ, NX, SZ, XZ, XD, LW, PL, XZ, and ZX have no competing of interests to disclose. AQ, OZ, RU, TS are employees of PharmaEssentia Corporation. DW, WS, WW, JZ, and Yi are employees of PharmaEssentia Biotech (Beijing) Ltd. EDAT- 2023/06/22 01:07 MHDA- 2023/06/22 01:08 PMCR- 2023/06/21 CRDT- 2023/06/21 23:41 PHST- 2023/03/29 00:00 [received] PHST- 2023/05/16 00:00 [accepted] PHST- 2023/06/22 01:08 [medline] PHST- 2023/06/22 01:07 [pubmed] PHST- 2023/06/21 23:41 [entrez] PHST- 2023/06/21 00:00 [pmc-release] AID - 10.1186/s40164-023-00415-0 [pii] AID - 415 [pii] AID - 10.1186/s40164-023-00415-0 [doi] PST - epublish SO - Exp Hematol Oncol. 2023 Jun 21;12(1):55. doi: 10.1186/s40164-023-00415-0.