RESEARCH
Split Viewer
Blood Res (2024) 59:34
Published online October 18, 2024
https://doi.org/10.1007/s44313-024-00039-1
© The Korean Society of Hematology
Real-world experience of emicizumab prophylaxis in Korean children with severe hemophilia A without inhibitors
Correspondence to : Young Shil Park
pysped@khu.ac.kr
© The Author(s) 2024. Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/.
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.
Keywords Emicizumab, Hemophilia A, Prophylaxis, Pediatric, Real-world data
PDF
Standard view
Export citation
Share Article
RESEARCH
Blood Res 2024; 59():
Published online October 18, 2024 https://doi.org/10.1007/s44313-024-00039-1
Copyright © The Korean Society of Hematology.
Real-world experience of emicizumab prophylaxis in Korean children with severe hemophilia A without inhibitors
Sung Eun Kim1, Ji Yoon Kim1,2, Jeong A Park3, Chuhl Joo Lyu4, Seung Min Hahn4, Jung Woo Han4 and Young Shil Park5*
1 Department of Pediatrics, Kyungpook National University Hospital, Kyungpook National University Chilgok Hospital, 807 Hoguk‑Ro, Buk‑Gu, Daegu 41404, Republic of Korea
2 Department of Pediatrics, School of Medicine, Kyungpook National University, 680 Gukchaebosang‑Ro, Jung‑Gu, Daegu 41944, Republic of Korea
3 Department of Pediatrics, Inha University Hospital, 27 Inhang‑Ro, Jung‑Gu, Incheon 22332, Republic of Korea
4 Department of Pediatrics, Yonsei University Severance Children’s Hospital, 50‑1 Yonseiro, Seodaemun‑Gu, Seoul 03722, Republic of Korea
5 Department of Pediatrics, Kyung Hee University Hospital at Gangdong, 892 Dongnam‑Ro, Gangdong‑Gu, Seoul 05278, Republic of Korea
Correspondence to:Young Shil Park
pysped@khu.ac.kr
© The Author(s) 2024. Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/.
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.
Keywords: Emicizumab, Hemophilia A, Prophylaxis, Pediatric, Real-world data
Fig 1.
Download
Fig 2.
Download
-
Table 1 . Characteristics of the patients.
Pediatric Patients Without Inhibitors Total number, N 21 Median age at the start of emicizumab (range) 4.00 (0–11) Prior treatment regimen, N (%) Prophylaxis 20 (95.2) On demand 1 (4.8) Previous drug usage, N (%) pdFVIII 3 (14.3) rFVIII 18 (85.7) Presence of target joint, N (%) 3 (14.3)
-
Table 2 . Details of individual patients.
Patient Number Agea (Years) Previous Drug Usage Previous Treatment Method Emicizumab Regimen Pre-Emicizumab ABR Post-Emicizumab ABR 1 0b pdFVIII On demand Every 4 weeks 4.35 0 2 2 rFVIII Prophylaxis Every 4 weeks 8.7 0 3 1 rFVIII Prophylaxis Every 4 weeks 2.17 0 4 8 rFVIII Prophylaxis Every 2 weeks 2.17 2.17 5 9 rFVIII Prophylaxis Every 2 weeks 8.7 0 6 4 rFVIII Prophylaxis Every 2 weeks 2.17 0 7 2 rFVIII Prophylaxis Weekly 4.35 0 8 5 rFVIII Prophylaxis Every 2 weeks 2.17 0 9 10 rFVIII Prophylaxis Every 2 weeks 4.35 0 10 7 rFVIII Prophylaxis Every 2 weeks 6.52 4.35 11 11 rFVIII Prophylaxis Every 2 weeks 6.52 0 12 4 rFVIII Prophylaxis Every 2 weeks 6.52 0 13 10 pdFVIII Prophylaxis Weekly 6.52 0 14 4 rFVIII Prophylaxis Every 2 weeks 21.74 0 15 11 rFVIII Prophylaxis Every 2 weeks 0 0 16 0c pdFVIII Prophylaxis Every 2 weeks 2.17 0 17 3 rFVIII Prophylaxis Every 2 weeks 2.17 0 18 4 rFVIII Prophylaxis Every 2 weeks 13.04 0 19 2 rFVIII Prophylaxis Every 2 weeks 19.57 0 20 5 rFVIII Prophylaxis Every 2 weeks 13.04 0 21 9 rFVIII Prophylaxis Every 2 weeks 10.87 2.17 a Age at the start of emicizumab.
b Patient no. 1 age: 4 months.
c Patient no. 16 age: 5 months.
pdFVIII: plasma-derived factor VIII; rFVIII: recombinant factor VIII.
-
Table 3 . Bleeding data.
Pre-Emicizumab Post-Emicizumab P-value Mean ABR (± SD) 7.04 (± 5.83) 0.41 (± 1.11) < 0.0001* Median ABR (range) 6.52 (0–21.74) 0 (0–4.35) Mean AJBR (± SD) 2.28 (± 2.52) 0.21 (± 0.65) 0.0010* Median AJBR (range) 2.17 (0–6.52) 0 (0–2.17) Zero bleeding, N (%) 1 (4.8) 18 (85.7) < 0.0001$ * Wilcoxon signed-rank test.
$ McNemar’s test.
ABR Estimated annualized bleeding rate, SD Standard deviation, AJBR Estimated annualized joint bleeding rate.
Article Tools
Stats or Metrics
Share this article on
Related articles in BR
-
Evaluation of FVIII pharmacokinetic profiles in Korean hemophilia A patients assessed with myPKFiT: a retrospective chart review
Young-Shil Park, Ki-Young Yoo, Sang Kyu Park, Taiju Hwang, Aeran Jung and Eun Jin Choi
Blood Res 2024; 59(): -
Real-world insights into the management of hemophilia A in Italy: treatment patterns and healthcare resource utilization
Valentina Perrone, Melania Leogrande, Maria Cappuccilli and Luca Degli Esposti
Blood Res 2024; 59(): -
Clinical data on treatment regimen and use of medication among patients with hemophilia B in Korea
Young Shil Park, Ji Kyoung Park, Jeong A Park, Hee Jo Baek, Jae Hee Lee, Chur Woo You, Chuhl Joo Lyu and Eun Jin Choi
Blood Res 2024; 59():
