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

作者全名："Jin, Jie; Zhang, Lei; Qin, Albert; Wu, Daoxiang; Shao, Zonghong; Bai, Jie; Chen, Suning; Duan, Minghui; Zhou, Hu; Xu, Na; Zhang, Sujiang; Zuo, Xuelan; Du, Xin; Wang, Li; Li, Pei; Zhang, Xuhan; Li, Yaning; Zhang, Jingjing; Wang, Wei; Shen, Weihong; Zagrijtschuk, Oleh; Urbanski, Raymond; Sato, Toshiaki; Xiao, Zhijian"

作者地址："[Jin, Jie] Zhejiang Univ, Affiliated Hosp 1, Dept Hematol, Sch Med, Hangzhou, Zhejiang, Peoples R China; [Zhang, Lei] Chinese Acad Med Sci & Peking Union Med Coll, Inst Hematol, State Key Lab Expt Hematol, Natl Clin Res Ctr Blood Dis, Tianjin 300020, Peoples R China; [Zhang, Lei] Chinese Acad emy Med Sci & Peking Union Med Coll, Blood Dis Hosp, Tianjin 300020, Peoples R China; [Qin, Albert] PharmaEssentia Corp, Med Res & Clin Operat, Taipei, Taiwan; [Wu, Daoxiang; Li, Yaning; Zhang, Jingjing; Wang, Wei; Shen, Weihong] PharmaEssentia Biotech Beijing Ltd, Beijing, Peoples R China; [Shao, Zonghong; Bai, Jie] Tianjin Med Univ, Hosp 2, Tianjin, Peoples R China; [Chen, Suning] Soochow Univ, Affiliated Hosp 1, Suzhou, Jiangsu, Peoples R China; [Duan, Minghui] Chinese Acad Med Sci & Peking Union Med Coll, Peking Union Med Coll Hosp, Beijing, Peoples R China; [Zhou, Hu] Zhengzhou Univ, Affiliated Canc Hosp, Zhengzhou, Henan, Peoples R China; [Zhou, Hu] Henan Canc Hosp, Zhengzhou, Henan, Peoples R China; [Xu, Na] Southern Med Univ, Nanfang Hosp, Guangzhou, Guangdong, Peoples R China; [Zhang, Sujiang] Shanghai Jiao Tong Univ, Ruijin Hosp, Sch Med, Shanghai, Peoples R China; [Zuo, Xuelan] Wuhan Univ, Zhongnan Hosp, Wuhan, Hubei, Peoples R China; [Du, Xin] Shenzhen Second Peoples Hosp, Shenzhen, Guangdong, Peoples R China; [Wang, Li] Chongqing Med Univ, Affiliated Hosp 1, Chongqing, Peoples R China; [Li, Pei] Fudan Univ, Huashan Hosp, Shanghai, Peoples R China; [Zhang, Xuhan] Univ Sci & Technol China, Affiliated Hosp USTC 1, Div Life Sci & Med, Hefei, Peoples R China; [Zagrijtschuk, Oleh; Urbanski, Raymond] PharmaEssentia USA Corp, Burlington, MA USA; [Sato, Toshiaki] PharmaEssentia Japan K K, Minato Ku, Tokyo, Japan; [Xiao, Zhijian] Chinese Acad Med Sci & Peking Union Med Coll, Inst Hematol, Tianjin, Peoples R China; [Xiao, Zhijian] Chinese Acad Med Sci & Peking Union Med Coll, Blood Dis Hosp, Tianjin, Peoples R China"

通信作者："Jin, J (通讯作者)，Zhejiang Univ, Affiliated Hosp 1, Dept Hematol, Sch Med, Hangzhou, Zhejiang, Peoples R China.; Zhang, L (通讯作者)，Chinese Acad Med Sci & Peking Union Med Coll, Inst Hematol, State Key Lab Expt Hematol, Natl Clin Res Ctr Blood Dis, Tianjin 300020, Peoples R China.; Zhang, L (通讯作者)，Chinese Acad emy Med Sci & Peking Union Med Coll, Blood Dis Hosp, Tianjin 300020, Peoples R China.; Qin, AL (通讯作者)，PharmaEssentia Corp, Med Res & Clin Operat, Taipei, Taiwan."

来源：EXPERIMENTAL HEMATOLOGY & ONCOLOGY

ESI学科分类：CLINICAL MEDICINE

WOS号：WOS:001017246400001

JCR分区：Q1

影响因子：9.4

年份：2023

卷号：12

期号：1

开始页：　

结束页：　

文献类型：Letter

关键词：Ropeginterferon alfa-2b; New dosing regimen; Chinese patients with polycythemia vera; Complete hematologic response; Molecular response; JAK2(V617F) allele burden

摘要："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 mu g (50 mu g for patients receiving hydoxyurea) and intra-patient dose titrations up to 500 mu g at 50 mu g 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 mu g 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 JAK2V617F 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 mu g regimen is highly effective and well-tolerated and can help patients achieve greater and rapid complete hematologic and molecular responses."

基金机构：PharmaEssentia Corporation

基金资助正文：The study was supported by PharmaEssentia Corporation.