Non-factor replacement therapy - a new chapter in hemophilia A prophylaxis

Mimetik FVIII - novo poglavlje u profilaksi hemofilije A (skraćen naslov rada; naš rad nema podnaslov)

  • Božidar Lilić Hematology, Allergology and Clinical Immunology Clinic, UCC Niš
  • Miodrag Vučić Hematology, Allergology and Clinical Immunology Clinic, UCC Niš, University of Niš, Faculty of Medicine
  • Asist. dr Danijela Jovančić-Petković Pediatric Clinic, UCC Niš, Medical faculty, University of Niš
  • Asist. dr Jelena Vučić Pediatric Clinic, UCC Niš, Medical faculty, University of Niš
DOI: https://doi.org/10.5937/afmnai41-49466
Keywords: hemophilia, antihemophilic factor, emicizumab, inhibitors, prophylaxis

Abstract


Aim: The aim of this analysis is to present the effects of prophylactic non-factor replacement therapy in ten patients with hemophilia A. Patients and methods: This retrospective analysis was conducted on ten male patients (4 children, 1 adolescent, and 5 adults) with severe hemophilia A and a history of antihemophilic factor replacement prophylaxis, prior to the initiation of emicizumab prophylaxis. A single adult patient developed inhibitors during the course of factor replacement prophylaxis. Four adult patients had already developed hemophilic arthropathy before the initiation of non-factor replacement prophylaxis. Two adult patients received emicizumab prophylaxis every 4 weeks, while others received emicizumab every 2 weeks. After a 14 month period (average) of non-factor replacement prophylaxis, we analyzed the number of breakthrough bleeding episodes, annualized bleeding rate, involvement of target joints, adverse drug reactions, and interviewed the patients regarding their satisfaction with the non-factor replacement treatment. Results:  None of the patients on emicizumab prophylaxis experienced breakthrough bleeding, nor clinical worsening of the affected target joints during the period of emicizumab prophylaxis. Annualized bleeding rate was zero in all patients on emicizumab prophylaxis. No adverse drug reactions had occurred in our patients during emicizumab prophylaxis. All patients reported greater treatment satisfaction compared to the replacement prophylaxis. Conclusion: By providing safety from bleeding events and potentially the stability of affected joints, emicizumab prophylaxis enables greater activity and increases the quality of life of treated patients.

 

Author Biographies

Božidar Lilić, Hematology, Allergology and Clinical Immunology Clinic, UCC Niš

The second author of the article "Non-factor replacement therapy - a new chapter in hemophilia A prophylaxis" and the corresponding author.

Miodrag Vučić, Hematology, Allergology and Clinical Immunology Clinic, UCC Niš, University of Niš, Faculty of Medicine

The first author of the article "Non-factor replacement therapy - a new chapter in hemophilia A prophylaxis"

Asist. dr Danijela Jovančić-Petković, Pediatric Clinic, UCC Niš, Medical faculty, University of Niš

The third autor of the article "Non-factor replacement therapy - a new chapter in hemophilia A prophylaxis"

Asist. dr Jelena Vučić, Pediatric Clinic, UCC Niš, Medical faculty, University of Niš

The fourth author of the article "Non-factor replacement therapy - a new chapter in hemophilia A prophylaxis"

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https://doi.org/10.1056/NEJMoa1703068
Published
2025/11/20
Issue
Vol. 41 No. 2 (2024): Acta facultatis medicae Naissensis
Section
Profesionalni rad / Professional article