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
. 2024 Jan-Dec:30:10760296231224357.
doi: 10.1177/10760296231224357.

Emicizumab Prophylaxis in Patients with Severe Hemophilia A: Insights from A Resource Limited Country

Munira Borhany  1 , Aisha Arshad  1 , Heeba Qureshi  1 , Rukhshanda Nadeem  1 , Arif Jamal  1 , Raheel Ahmed Khan  1
Affiliations

Emicizumab Prophylaxis in Patients with Severe Hemophilia A: Insights from A Resource Limited Country

Munira Borhany et al. Clin Appl Thromb Hemost. 2024 Jan-Dec.

Abstract

Methods: In this prospective study, severe HA patients were recruited from January 2022 to June 2023. Inhibitor positive and inhibitor negative patients with annual bleeding rate (ABR) 8 or greater and past histories of bleeding like intra-cranial, intra-abdominal, and pseudo-tumors were included. Emicizumab loading dose was 3 mg/kg in the first 4 weeks, and the maintenance dose was started at week 5 at 6 mg/kg/month. Patients' detailed bleeding history and demographics were recorded. The five-level EuroQol five-dimensional questionnaire (EQ-5D-5L) was used to evaluate patients' HRQoL. Furthermore, Hemophilia Joint Health Score (HJHS) and Functional Independence score in Hemophilia (FISH) were applied for the assessment of joints at different time points. Results were analyzed by SPSS version 21.

Results: A total of 36 HA male patients with the mean age of 19.7 ± 14.42 years were recruited in the study; among them, 19 patients were inhibitor positive, while 17 were negative. Patients clinically presented with bleeding symptoms which included: hemarthrosis 95%, GI bleeding 13.8%, and bruises and gums bleeding 13.8%. Significant reduction was observed in the bleeding episodes after the therapeutic intervention, and joints assessment and Euro-Quality-of-life Visual Analog Scale showed a significant improvement in health after treatment. Similarly, there was a remarkable reduction in bleeding episodes and improved quality of life among HA patients. The ABR decreased from 53.6% episodes per year prior to treatment to 2.4% during Emicizumab therapy. Prior to initiating Emicizumab therapy, participants exhibited an average FISH score of 16 and HJHS score of 10, indicating moderate limitations due to joint-related issues. After treatment, the mean FISH score improved to 9 and HJHS score to 4 reflecting a substantial enhancement in participants' ability to perform daily activities (P < 0.057).

Conclusion: Our results showed that HA patients on prophylactic treatment with Emicizumab were less restricted and had improved quality of life due to marked decrease in bleeding episodes which resulted in improved health and social lives. In addition, it was well tolerated, and no participant discontinued treatment because of adverse events.

Keywords: EQ-5D-5L; Functional Independence Score in Hemophilia; Hemophilia Joint Health Score; bleeding; health-related quality of life; hemophilia A; joint score.

PubMed Disclaimer

Conflict of interest statement

Declaration of Conflicting InterestsThe author(s) declared no potential conflicts of interest with respect to the research, authorship, and/or publication of this article.

Figures

Figure 1.
Figure 1.
Evaluation of HJHS and FISH scoring before and after treatment of Emicizumab. FISH, Functional Independence score in Hemophilia, HJHS, Hemophilia Joint Health Score.
Figure 2.
Figure 2.
HRQoL Pre-treatment with Emicizumab. MO: mobility SC: self-care UA: usual activities PD: pain/discomfort AD: anxiety/depression.
Figure 3.
Figure 3.
HRQoL Post-treatment with Emicizumab. MO: mobility SC: self-care UA: usual activities PD: pain/discomfort AD: anxiety/depression.
See this image and copyright information in PMC

Similar articles

See all similar articles

References

    1. Srivastava A, Santagostino E, Dougall A, et al. WFH guidelines for the management of hemophilia, 3rd edition. Haemophilia. 2020;27(4). doi:10.1111/hae.14046 - DOI - PubMed
    1. Blanchette V, Key N, Ljung L, Manco-Johnson M, Van Den Berg H, Srivastava A. Definitions in hemophilia: Communication from the SSC of the ISTH. J Thromb Haemostasis. 2014;12(11):1935-1939. - PubMed
    1. Kizilocak H, Young G. Diagnosis and treatment of hemophilia. Clin Adv Hematol Oncol. 2019;17(6):344-351. - PubMed
    1. Witmer C, Presley R, Kulkarni R, Michael Soucie J, Manno CS, Raffini L. Associations between intracranial haemorrhage and prescribed prophylaxis in a large cohort of haemophilia patients in the United States. Br J Haematol. 2011;152(2):211-216. - PubMed
    1. Bauer KA. Current challenges in the management of hemophilia. Am J Manag Care. 2015;21(6 Suppl):S112–-S122. - PubMed