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Reduced doses of emicizumab achieve good efficacy: Results from a national-wide multicentre real-world study in China.
Xu, Yuan; Wang, Ying; Wu, Runhui; Zheng, Changcheng; Zhang, Li; Xu, Weiqun; Feng, Xiaoqin; Wang, Hua; Cao, Xiangshan; He, Liya; Xue, Tianyang; Jin, Mingwei; Xie, Bingshou; Ling, Jing; Sun, Lirong; Su, Rui; Cheng, Hongbo; Fang, Yongjun; Poon, Man-Chiu; Liu, Wei; Zhang, Lei; Xue, Feng; Yang, Renchi.
Afiliação
  • Xu Y; 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 The
  • Wang Y; Tianjin Institutes of Health Science, Tianjin, China.
  • Wu R; Department of Hematology and Oncology, Shenzhen Children's Hospital, Shenzhen, China.
  • Zheng C; Hematology Oncology Center, National Center for Children's Health, Beijing Children's Hospital, Capital Medical University, Beijing, China.
  • Zhang L; Department of Hematology, The First Affiliated Hospital of USTC, Division of Life Sciences and Medicine, University of Science and Technology of China, Hefei, China.
  • Xu W; Department of Hematology, Children's Hospital of Chongqing Medical University, Chongqing, China.
  • Feng X; Division/Center of Pediatric Hematology-Oncology, the Children's Hospital of Zhejiang University School of Medicine, Hangzhou, China.
  • Wang H; Pediatrics, Nanfang Hospital, Southern Medical University, Guangzhou, China.
  • Cao X; Department of Hematology and Oncology, Xi'an Children's Hospital, Xi'an, China.
  • He L; Department of Hematology, Suzhou BenQ Medical Center, The Affiliated BenQ Hospital of Nanjing Medical University, Suzhou, China.
  • Xue T; Department of Pediatric, Guangzhou Women and Children's Medical Center, Guangzhou Medical University, Guangzhou, China.
  • Jin M; Pediatrics, Xuzhou Star Hospital, Xuzhou, China.
  • Xie B; Pediatrics, Xuzhou Star Hospital, Xuzhou, China.
  • Ling J; Department of Hematology, Wenzhou People's Hospital, Wenzhou, China.
  • Sun L; Department of Hematology, Children's Hospital of Soochow University, Suzhou, China.
  • Su R; Department of Pediatrics, Affiliated Hospital of Qingdao University, Qingdao, China.
  • Cheng H; Department of Hematology, The First Affiliated Hospital of Xiamen University, Xiamen, China.
  • Fang Y; Department of Hematology, Jiangxi Provincial People's Hospital Affiliated to Nanchang University, Nanchang, China.
  • Poon MC; Department of Hematology/Oncology, Children's Hospital of Nanjing Medical University, Nanjing, China.
  • Liu W; Departments of Medicine, Paediatrics and Oncology, Cumming School of Medicine, University of Calgary, Calgary, Alberta, Canada.
  • Zhang L; 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 The
  • Xue F; Tianjin Institutes of Health Science, Tianjin, China.
  • Yang R; 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 The
Haemophilia ; 30(4): 959-969, 2024 Jul.
Article em En | MEDLINE | ID: mdl-38853005
ABSTRACT

INTRODUCTION:

Reduced doses of emicizumab improve the affordability among patients in developing countries. However, the relationship between variant dose selection and efficacy in the real world of China is still unclear.

AIM:

This study aimed to investigate the efficacy and safety of emicizumab especially in those on reduced dose regimens in a real-world setting.

METHODS:

We carried out a multicentre study from 28 hospitals between June 2019 and June 2023 in China and retrospectively analysed the characteristics including demographics, diagnosis, treatment, bleeding episodes, and surgical procedures.

RESULTS:

In total, 127 patients with haemophilia A, including 42 with inhibitors, were followed for a median duration of 16.0 (IQR 9.0-30.0) months. Median age at emicizumab initiation was 2.0 (IQR 1.0-4.0) years. Median (IQR) consumption for loading and maintenance was 12.0 (8.0-12.0) and 4.2 (3.0-6.0) mg/kg/4 weeks, respectively. While on emicizumab, 67 (52.8%) patients had no bleeds, whereas 60 (47.2%) patients had any bleeds, including 26 with treated bleeds. Compared to previous treatments, patients on emicizumab had significantly decreased annualized bleeding rate, annualized joint bleeding rate, target joints and intracerebral haemorrhage. Different dosages had similar efficacy except the proportion of patients with treated spontaneous bleeds and target joints. Adverse events were reported in 12 (9.4%) patients. Postoperative excessive bleeding occurred following two of nine procedures.

CONCLUSION:

This is the largest study describing patients with HA receiving emicizumab prophylaxis on variant dose regimens in China. We confirmed that nonstandard dose is efficacious and can be considered where full-dose emicizumab is ill affordable.
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Anticorpos Biespecíficos / Anticorpos Monoclonais Humanizados / Hemofilia A Limite: Child / Child, preschool / Female / Humans / Infant / Male País como assunto: Asia Idioma: En Ano de publicação: 2024 Tipo de documento: Article

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Anticorpos Biespecíficos / Anticorpos Monoclonais Humanizados / Hemofilia A Limite: Child / Child, preschool / Female / Humans / Infant / Male País como assunto: Asia Idioma: En Ano de publicação: 2024 Tipo de documento: Article