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All Journal Medical Journal of Indonesia
Wiharta, Fitri Primacakti
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Medicine & Pharmacology

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Journal : Medical Journal of Indonesia

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Emicizumab prophylaxis in severe hemophilia A with inhibitor: impact on quality of life and musculoskeletal outcomes Wiharta, Fitri Primacakti; Saraswati, Made Citra; Abigail, Dina Clarisa Rumora; Chozie, Novie Amelia
Medical Journal of Indonesia Vol. 34 No. 3 (2025): September
Publisher : Faculty of Medicine Universitas Indonesia

Show Abstract | Download Original | Original Source | Check in Google Scholar | DOI: 10.13181/mji.cr.257819

Abstract

Emicizumab has been acknowledged for its effectiveness in preventing bleeding in patients with hemophilia A developing inhibitors. This report examined the impact of emicizumab prophylaxis on health-related quality of life (HRQoL) and musculoskeletal outcomes in a 10-year-old male with severe hemophilia A and a high titer of factor VIII (FVIII) inhibitor, presenting with hemophilic arthropathy on the right knee and left elbow. He had been unable to walk and was previously treated on-demand with a plasma-derived FVIII concentrate. He received emicizumab prophylaxis for 16 months. HRQoL was evaluated using a hemophilia-specific questionnaire, while musculoskeletal outcomes were assessed using hemophilia early arthropathy detection with ultrasound (HEAD-US) and the hemophilia joint health score (HJHS). During treatment, no breakthrough bleeding occurred, and the patient regained the ability to walk. Emicizumab prophylaxis significantly improved in HRQoL, especially in physical health and sports and and school domains, aligning with bleeding rates, HEAD-US, and HJHS assessments.
Co-Authors Abigail, Dina Clarisa Rumora Novie Amelia Chozie Saraswati, Made Citra