中国当代儿科杂志2026，Vol.28Issue(5)：580-585,6.DOI:10.7499/j.issn.1008-8830.2506113
减低剂量艾美赛珠单抗在婴儿重型血友病A患者中的有效性与安全性评估:单中心真实世界研究
Evaluation of the efficacy and safety of reduced-dose emicizumab in infants with severe hemophilia A:a single-center real-world study
摘要
Abstract
Objective To investigate the efficacy and safety of reduced-dose emicizumab(EMI)prophylaxis in infants under one year of age with severe hemophilia A(HA).Methods Medical records of 11 infants aged≤12 months with severe HA who received reduced-dose EMI in the Department of Hematology and Oncology,Shenzhen Children's Hospital,from July 2022 to June 2025 were retrospectively reviewed.Efficacy and safety were analyzed statistically.Results The median age at EMI initiation was 7.9 months(range 4.2-11.2 months).The median loading dose(first 4 weeks)and median maintenance dose were 6.2 mg/kg per 4 weeks(range 2.8-12.6 mg/kg per 4 weeks)and 3.1 mg/kg per 4 weeks(range 2.0-4.8 mg/kg per 4 weeks),respectively.The median duration of EMI prophylaxis was 15.8 months(range 1.7-36.0 months).After treatment,the median(range)annualized bleeding rate,spontaneous annualized bleeding rate,and annualized treated bleeding rate were 0.5(0-6.2),0(0-0.5),and 0(0-3.0),respectively.All decreased compared with pre-treatment,and the reduction in spontaneous annualized bleeding rate was statistically significant(P<0.05).Overall,64%(7/11)of patients experienced no bleeding events requiring treatment.No EMI-related adverse events were observed.Conclusions Reduced-dose EMI prophylaxis effectively controls bleeding in infants with hemophilia A and shows good safety.关键词
血友病A/艾美赛珠单抗/有效性/安全性/婴儿Key words
Hemophilia A/Emicizumab/Efficacy/Safety/Infant引用本文复制引用
周桂池,朱理想,刘四喜,王缨,李倩,彭冬,付笑迎,邓雅爽,王春艳,何学智,周春明,宋萍..减低剂量艾美赛珠单抗在婴儿重型血友病A患者中的有效性与安全性评估:单中心真实世界研究[J].中国当代儿科杂志,2026,28(5):580-585,6.基金项目
深圳市医疗卫生"三名工程"(SZSM202211033) （SZSM202211033）
广东省高水平临床重点专科(SZGSP012) （SZGSP012）
重大疾病多中心临床研究项目(LCYJ2022067). （LCYJ2022067）
