RESULTS OF THE USE OF EMICIZUMAB IN PATIENTS WITH HEMOPHILIA A – A PILOT SINGLE-CENTER STUDY
Keywords:
emicizumab, hemophilia A, coagulation factor VIII, coagulation factor VIII inhibitor, one-step clotting method, chromogenic methodAbstract
Ibragimova Sapura Zakhidovna, Almedova Nargiza Nigmatjonovna, Botirov Mirzokhid Mansurzhon Ugli, Shadibekova Oksana Borisovna, Aripova Nazokat Bahodirovna, Erimbetova Indira Oralbaevna. Results of the use of emicizumab in patients with hemophilia A – a pilot single-center study // Journal of Biomedicine and Practice. 2026, vol. 11, issue 1.
Resume. It is known that patients with severe hemophilia A, a hereditary bleeding disorder caused by a deficiency of coagulation factor VIII, often experience uncontrolled bleeding, which occurs despite the administration of prophylactic replacement therapy with coagulation factors. Such conditions are characteristic of the inhibitor form of hemophilia A [1,2]. An inhibitor is a polyclonal high-affinity IgG antibody that specifically neutralizes the procoagulant activity of coagulation factors, making bleeding management difficult. The emergence of inhibitors is associated with a worsening prognosis: a higher incidence of bleeding, accelerated development of arthropathy, and a decrease in quality of life. In more severe cases, this could lead to the death of children [3,6]. In order to prevent the above-mentioned conditions, improve the quality of life of patients and prevent complications of the disease, the Scientific and Practical Medical Center for Pediatric Hematology, Oncology and Immunology used the "Hemlibra" (emicizumab) in 15 children with hereditary bleeding disorder caused by the inhibitory form of coagulation factor VIII deficiency.
References
Румянцев А.Г., Масчан А.А., Вдовин В.В., Свирин П.В. Федеральные клинические рекомендации по диагностике и лечению детей с гемофилией А, гемофилией В и болезнью Виллебранда. М.; 2015. [Rumyantsev A.G., Maschan A.A., Vdovin V.V., Svirin P.V. Federal clinical guidelines for the diagnosis and treatment of children with hemophilia A, hemophilia B and von Willebrand disease. Moscow; 2015. (In Russ.)].
Srivastava A., Santagostino E., Dougall A., Kitchen S., Sutherland M., Pipe S.W., et al. WFH Guidelines for the Management of Hemophilia, 3rd edition. Haemophilia 2020; 26 Suppl 6: 1–158.
Barg A.A., Budnik I., Avishai E., Brutman‐Barazani T., Bashari D., Misgav M., et al. Emicizumab prophylaxis: Prospective longitudinal real‐ world follow‐up and monitoring. Haemophilia 2021; 27 (3): 383–91.
Долгов В.В., Свирин П.В., Вавилова Т.В. Лабораторная диагностика нарушений гемостаза. Учебно-методическое пособие. М; 2019. [Dolgov V.V., Svirin P.V., Vavilova T.V. Laboratory diagnosis of hemostasis disorders. Educational guide. Moscow; 2019. (In Russ.)].
Takeyama M., Nogami K., Shima M. A new parameter in the thrombin generation assay, mean velocity to peak thrombin, reflects factor VIII activity in patients with haemophilia A. Haemophilia 2016; 22 (5): e474–7.
Ketteler C., Hoffmann I., Davidson S., Tiede A., Richter N. Monitoring of different factor VIII replacement products using a factor VIII one‐stage clotting assay on cobas t 511/711 analysers. Haemophilia 2021; 27 (6): e704–12.
Gray E., Kitchen S., Bowyer A., Chowdary P., Jenkins P.V., Murphy P., et al. Laboratory measurement of factor replacement therapies in the treatment of congenital haemophilia: A United Kingdom Haemophilia Centre Doctors’ Organisation guideline. Haemophilia 2020; 26 (1): 6–16.
Жаркова П.А. Есть ли место заместительной терапии гемофилии А у детей в настоящем и будущем? Российский журнал детской гематологии и онкологии 2022; 9 (3): 56–64. DOI: 10.21682/2311-1267- 2022-9-3-56-64
Bowyer A.E., Lowe A.E., Tiefenbacher S. Laboratory issues in gene therapy and emicizumab. Haemophilia 2021; 27 (S3): 142–7.
Kizilocak H., Marquez‐Casas E., Malvar J., Carmona R., Young G. Determining the approximate factor VIII level of patients with severe haemophilia A on emicizumab using in vivo global haemostasis assays. Haemophilia 2021; 27 (5): 730–5
Jonsson F., Mercier F., Prins N.H., Schmitt C., Retout S. Exposure-response modeling of emicizumab for the prophylaxis of bleed counts in hemophilia A patients. 27th Meeting of the Population Approach Group in Europe (PAGE). 2018.
Adamkewicz J.I., Chen D.C., PazPriel I. Effects and Interferences of Emicizumab, a Humanised Bispecific Antibody Mimicking Activated Factor VIII Cofactor Function, on Coagulation Assays. Thromb Haemost 2019; 119 (7): 1084–93
