ArticleZhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics2026
[Evaluation of the efficacy and safety of reduced-dose emicizumab in infants with severe hemophilia A: a single-center real-world study].
Article in Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
objectivesTo investigate the efficacy and safety of reduced-dose emicizumab (EMI) prophylaxis in infants under one year of age with severe hemophilia A (HA).
methodsMedical 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.
resultsThe 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 (
conclusionsReduced-dose EMI prophylaxis effectively controls bleeding in infants with hemophilia A and shows good safety.
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