в текстеNormalized hemostasis as a new goal in the treatment of hemophilia A in children: the role of efanesoctocog alfa

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Abstract

Hemophilia A remains one of the major challenges in pediatric hematology, because it is during childhood that long-term disease outcomes develop, including the degree of joint damage, the level of physical activity, social adaptation, and quality of life in adulthood. Over the past decades, hemophilia A management has undergone a remarkable transformation, from treating individual bleeding episodes to early prophylaxis, with the aim of preserving joint health and achieving the so-called normalized hemostasis. This review summarizes current knowledge on hemophilic arthropathy, the evolution of the goals of prophylaxis, and the clinical and pharmacokinetic characteristics of efanesoctocog alfa, an ultra-long-acting factor VIII that functions independently of endogenous von Willebrand factor. Special attention is given to pivotal results from the phase III XTEND-Kids and XTEND-1 studies, indirect comparative analyses of standard and extended-half-life products, and non-factor therapy with emicizumab.

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About the authors

Pavel A. Zharkov

The Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation

Author for correspondence.
Email: pavel.zharkov@fccho-moscow.ru
ORCID iD: 0000-0003-4384-6754

Dr. Med. Sci., Professor, a pediatrician, a hematologist, Head of the Department of Non-Malignant Hematology 

Russian Federation, Moscow

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2. Figure 1 The structure of efanesoctocog alfa molecule with three prolongation technologies (by [29, 30], with changes) vWF – von Willebrand factor

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3. Figure 2 A comparison of the pharmacokinetic parameters of recombinant FVIII products after a single injection [31, 32] SD – standard deviation

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