Emicizumab as a Promising Form of Therapy for Type A Hemophilia - A Review of Current Knowledge from Clinical Trials


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Аннотация

:Hemophilia is a plasma bleeding disorder characterized by a deficiency of certain blood clotting factors. The most common forms of this disease, i.e., type A and type B, affect approximately 400,000 people worldwide. Without appropriate treatment ensuring the proper coagulation cascade, this disease may lead to serious disability. Minimizing patient discomfort is possible via replacement therapy, consisting of the substitution of a missing coagulation factor via intravenous administration. Frequent medication and the risk related to factor inhibitors are significant disadvantages, necessitating the improvement of current therapies or the development of novel ones. This review examines the humanized bispecific antibody Emicizumab which ensures hemostasis by mimicking the action of the coagulation factor VIII, a deficiency of which causes type A hemophilia. The paper outlines the topic and then summarizes available clinical trials on Emicizumab in type A hemophilia. Several interventional clinical trials have found Emicizumab to be effective in decreasing bleeding episodes and raising patient satisfaction among various hemophilia A populations. Current Emicizumab-related trials are forecast to be completed between 2024 and 2030, and in addition to congenital hemophilia A, the trials cover acquired hemophilia A and patients playing sports. Providing a more comprehensive understanding of Emicizumab may revolutionize the management of hemophilia type A and improve quality of life. Conclusively, Emicizumab is a gentler therapy owing to subcutaneous delivery and fewer injections, which reduces injection-site reactions and makes therapy less burdensome, ultimately decreasing hospital visits and indirect costs.

Об авторах

Katarzyna Grabowska

Department of Functional Genomics, Medical University of Lodz

Email: info@benthamscience.net

Michalina Grzelak

Department of Functional Genomics, Medical University of Lodz

Email: info@benthamscience.net

Lin-Yong Zhao

Department of General Surgery & Laboratory of Gastric Cancer, State Key Laboratory of Biotherapy/Collaborative Innovation Center of Biotherapy and Cancer Center, West China Hospital, Sichuan University

Email: info@benthamscience.net

Elżbieta Płuciennik

Department of Functional Genomics, Medical University of Lodz

Email: info@benthamscience.net

Zbigniew Pasieka

Department of Biomedicine and Experimental Surgery, Medical University of Lodz

Email: info@benthamscience.net

Mateusz Kciuk

Department of Molecular Biotechnology and Genetics, University of Lodz

Email: info@benthamscience.net

Adrianna Gielecińska

Department of Molecular Biotechnology and Genetics, University of Lodz

Email: info@benthamscience.net

Aleksander Smakosz

Department of Pharmaceutical Biology and Biotechnology, Wroclaw Medical University

Email: info@benthamscience.net

Żaneta Kałuzińska-Kołat

Department of Functional Genomics, Medical University of Lodz

Email: info@benthamscience.net

Damian Kołat

Department of Functional Genomics, Medical University of Lodz

Автор, ответственный за переписку.
Email: info@benthamscience.net

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