Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2023 Jun;45(3):622-629.
doi: 10.1007/s11096-022-01514-4. Epub 2023 Feb 27.

Data mining and analysis for emicizumab adverse event signals based on the Food and Drug Administration Adverse Event Reporting System database

Lianhui Wei #  1 , Ye Tian #  2 , Xiao Chen #  1 , Xiaojing Guo  1 , Chenxin Chen  1 , Yi Zheng  1 , Jinfang Xu  1 , Xiaofei Ye  3
Affiliations

Data mining and analysis for emicizumab adverse event signals based on the Food and Drug Administration Adverse Event Reporting System database

Lianhui Wei et al. Int J Clin Pharm. 2023 Jun.

Abstract

Background: Emicizumab is the latest treatment for patients with hemophilia A. Its safety in real-world data is limited, and regulatory agencies and clinical researchers have raised concerns about the risk of adverse events.

Aim: This study aimed to detect potential adverse event signals of emicizumab using the FDA Adverse Event Reporting System (FAERS) database.

Method: Data in FAERS from the fourth quarter of 2017 to the second quarter of 2021 were searched. Cases of adverse events were extracted using the Preferred Term in the Medical Dictionary for Regulatory Activities (version 24.0). Disproportionality analysis was performed using the reporting odds ratio (ROR) and information component (IC) methods based on statistical shrinkage transformation.

Results: A total of 5,598,717 patients were included, of which 1,244 took emicizumab. A total of 703 emicizumab-related adverse event signals were mined, and 101 positive signals were detected. Haemarthrosis (ROR/ROR975/ROR025 = 155.62/184.34/131.38, IC/IC975/IC025 = 7.28/7.48/7.01), haemorrhage (ROR/ROR975/ROR025 = 71.01/81.18/62.12, IC/IC975/IC025 = 6.15/6.31/5.94), muscle haemorrhage (ROR/ROR975/ROR025 = 53.38/75.83/37.58, IC/IC975/IC025 = 5.74/6.16/5.15), traumatic haemorrhage (ROR/ROR975/ROR025 = 27.78/46.29/16.67, IC/IC975/IC025 = 4.80/5.40/3.92), haematoma (ROR/ROR975/ROR025 = 18.15/26.35/12.51, IC/IC975/IC025 = 4.18/4.63/3.55), device-related thrombosis (ROR/ROR975/ROR025 = 21.27/37.57/12.04, IC/IC975/IC025 = 4.41/5.08/3.43), and activated partial thromboplastin time prolonged (ROR/ROR975/ROR025 = 20.68/36.51/11.71, IC/IC975/IC025 = 4.37/5.04/3.39) had the strongest signal intensities. Haemorrhage, haemarthrosis, arthralgia, fall, and injection site pain were reported more frequently.

Conclusion: This study found that mild arthralgia and injection site reaction were associated with emicizumab. Attention should also be paid to other serious adverse events related to emicizumab, such as acute myocardial infarction and sepsis, to ensure patient safety.

Keywords: Disproportionate analysis; Emicizumab; FAERS database; Hemophilia A.

PubMed Disclaimer

References

    1. Thrombosis and Hemostasis Group, Hematology Society of Chinese Medical Association; Hemophilia Treatment Center Collaborative Network of China. [Consensus of Chinese expert on the diagnosis and treatment of hemophilia (version 2017)]. Zhonghua Xue Ye Xue Za Zhi. 2017 May 14;38(5):364 – 70.
    1. Srivastava A, Brewer AK, Mauser-Bunschoten EP, et al. Treatment Guidelines Working Group on Behalf of The World Federation Of Hemophilia. Guidelines for the management of hemophilia. Haemophilia. 2013;19(1):e1–47. - DOI - PubMed
    1. Fischer K, Lewandowski D, Marijke van den Berg H, et al. Validity of assessing inhibitor development in haemophilia PUPs using registry data: the EUHASS project. Haemophilia. 2012;18(3):e241-6. - DOI - PubMed
    1. Yoneyama K, Schmitt C, Kotani N, et al. Pharmacometric approach to substitute for a conventional dose-finding study in rare diseases: example of phase iii dose selection for emicizumab in hemophilia A. Clin Pharmacokinet. 2018;57(9):1123–34. - DOI - PubMed
    1. Lenting PJ, Denis CV, Christophe OD. Emicizumab, a bispecific antibody recognizing coagulation factors IX and X: how does it actually compare to factor VIII? Blood. 2017;130(23):2463–8. - DOI - PubMed

LinkOut - more resources