Blood Res.  2024;59:34. 10.1007/s44313-024-00039-1.

Real‑world experience of emicizumab prophylaxis in Korean children with severe hemophilia A without inhibitors

• Kim S
• Kim J
• Park J
• Lyu C
• Hahn S
• Han J
• Park Y

• KMID: 2562538
• DOI: http://doi.org/10.1007/s44313-024-00039-1

Abstract

Purpose
Hemophilia A is a genetic disorder characterized by a lack of factor VIII (FVIII). Emicizumab, a recombinant humanized bispecific monoclonal antibody, mimics the function of FVIII. In this article, we present data on an initial real-world evaluation of emicizumab use in Korean children with severe hemophilia A without inhibitors.
Methods
This study was conducted from June 2020 to March 2024 at 4 centers in Korea. The participants were pediatric patients with severe hemophilia A without inhibitors who had received emicizumab treatment for over 6 months. The mean and median annualized bleeding rates (ABRs) and mean and median annual joint bleeding rates (AJBRs) were compared.
Results
Each of the 21 patients in the study received an emicizumab loading regimen of 3 mg/kg weekly for 4 weeks, followed by a modified maintenance regimen of which 2 patients (9.5%) received a 1.5 mg/kg weekly dose, 3 patients (14.3%) received a 6 mg/kg dose every 4 weeks, and the remaining 16 patients (76.2%) received a 3 mg/kg dose every 2 weeks. Before emicizumab prophylaxis initiation, the mean and median ABRs for all patients were 7.04 (SD ± 5.83) and 6.52 (range 0–21.74), respectively. After receiving emicizumab treatment, the mean and mediam ABRs decreased to 0.41 and zero, respectively. Additionally, 85.7% of the patients achieved no bleeding events within 6 months of starting the treatment.
Conclusion
These first real-world data in Korea indicate that emicizumab is effective and safe for pediatric patients with severe hemophilia A without inhibitors.

Keyword

Emicizumab; Hemophilia A; Prophylaxis; Pediatric; Real-world data

Figure

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  Fig. 1 Comparison of pre-emicizumab and post-emicizumab annualized bleeding rates (P < 0.0001)

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  Fig. 2 Emicizumab concentrations after treatment

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