PB0265 - Reduced doses of emicizumab achieve good efficacy: results from a national-wide multicenter real-world study in China

Sunday, June 23, 2024

13:45 – 14:45 ICT

Room: Exhibition Hall

Presenting Author(s)

Yuan Xu, MD

MD
State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases
Tianjin, Tianjin, China (People's Republic)

Co-Author(s)

YW

Ying Wang

professor
Shenzhen Children's Hospital
Shenzhen, Guangdong, China (People's Republic)
Runhui Wu, PhD, MD

Professor
Department of Hematology, Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health, Beijing, 100045, China
Beijing, Beijing, China (People's Republic)
CZ

Changcheng Zheng

Professor
The First Affiliated Hospital of University of Science and Technology of China,Anhui, P.R. China
Hefei, Anhui, China (People's Republic)
LZ

Li Zhang

professor
Children's Hospital of Chongqing Medical University
Chongqing, Chongqing, China (People's Republic)
WX

Weiqun Xu

professor
Children's Hospital, Zhejiang University School of Medicine
Hangzhou, Zhejiang, China (People's Republic)
XF

Xiaoqin Feng

No. 1838 North Guangzhou Avenue, Guangzhou, China
Department of Pediatrics, Nanfang Hospital, Southern Medical University
Guangzhou, Guangdong, China (People's Republic)
HW

Hua Wang

professor
Xi'an Children's Hospital
Xi'an, Shaanxi, China (People's Republic)
XC

Xiangshan Cao

professor
The Affiliated BenQ Hospital of Nanjing Medical University
Suzhou, Jiangsu, China (People's Republic)
LH

Liya He

professor
Guangzhou Women and Children's Medical Center
Guangzhou, Guangdong, China (People's Republic)
TX

Tianyang Xue

professor
Xuzhou Star Hospital
Xuzhou, Jiangsu, China (People's Republic)
MJ

Mingwei Jin

professor
Xuzhou Star Hospital
Xuzhou, Jiangsu, China (People's Republic)
BX

Bingshou Xie

professor
Wenzhou People's Hospital
Wenzhou, Zhejiang, China (People's Republic)
JL

Jing Ling

professor
Children's Hospital of Soochow University
Suzhou, Jiangsu, China (People's Republic)
LS

Lirong Sun

professor
Affiliated Hospital of Qingdao University
Qingdao, Shandong, China (People's Republic)
MP

Man-Chiu Poon

Professor
University of Calgary
Calgary, Alberta, Canada
WL

Wei Liu

professor
State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases
tianjin, Tianjin, China (People's Republic)
LZ

Lei Zhang

professor
State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Tianjin 300020, China
Tianjin, Tianjin, China (People's Republic)
Feng Xue, MD

Chief physician
State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Tianjin 300020, China
Tianjin, Tianjin, China (People's Republic)
Renchi Yang, MD

Head of Thrombosis and Hemostasis Center
Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College
Tianjin, Hebei, China (People's Republic)

Background: Real-world experiences of hemophilia A (HA) patients receiving emicizumab prophylaxis in China are insufficient due to the high cost. Patients chose variant regimens of emicizumab in the real world based on their affordability.

Aims: This study aimed to investigate the reasons for switching to emicizumab, safety, and efficacy especially in those who were on reduced dose regimens in a real-world setting.

Methods: We carried out a multicenter study on 127 HA patients from 28 hospitals receiving emicizumab prophylaxis between June 2019 and June 2023 in China and retrospectively analyzed the characteristics including demographics, diagnosis, treatment, bleeding episodes, and surgical procedures.

Results: While on emicizumab, 67 (52.8%) patients had no bleeds, and 26 (20.5%) patients had treated bleeds. Compared to previous treatments, patients on emicizumab had significantly decreased annualized bleeding rate (ABR), annualized joint bleeding rate (AJBR), number of target joints and intracerebral hemorrhage. Their inhibitor titers significantly decreased, and they had significantly less days away from schools or employment (table 1). Different dosages of emicizumab in our patients had similar efficacy except the proportion of patients with treated spontaneous bleeds, treated epistaxis, and target joints (table 2). Adverse events were reported in 12 (9.4%) patients. Postoperative excessive bleeding occurred following 2 of 9 procedures.

Conclusion(s): This is the largest study describing patients with HA receiving emicizumab prophylaxis on variant dose regimens in China. We confirmed that non-standard dose of emicizumab is efficacious and can be considered where full-dose emicizumab is ill affordable.