Management of patients with hemophilia A on emicizumab prophylactic treatment: Recommendation from Russian Experts

Author:

Andreeva T. A.1ORCID,Zharkov P. A.2ORCID,Zozulya N. I.3ORCID,Zorenko V. Yu.3ORCID,Konstantinova V. N.1ORCID,Lebedev V. V.4ORCID,Mamaev A. N.5ORCID,Markova I. V.6ORCID,Petrov V. Yu.7ORCID,Polyanskaya T. Yu.3ORCID,Schiller E. E.8ORCID

Affiliation:

1. City Polyclinic No 37, City Center for the Treatment of Patients with Hemophilia of St. Petersburg

2. Dmitriy Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology

3. National Medical Research Center for Hematology

4. Children's Regional Clinical Hospital

5. Altai Division of the National Research Center for Hematology

6. Raisa Gorbacheva Memorial Research Institute for Pediatric Oncology, Hematology and Transplantation

7. Morozovskaya Children's City Clinical Hospital of the Moscow Health Department

8. Odintsovo Region Hospital

Abstract

Introduction. In 2018 emicizumab was approved in Russia for prophylactic treatment in patients with hemophilia A (HA) with inhibitors and in 2019 for patients with severe HA without inhibitors. A significant amount of data has been accumulated from clinical trials and real-world data, which allow us to resolve most of the questions that hematologists may have when to prescribe emicizumab.Aim — to provide information on the management of patients on emicizumab.Results. The recommendations accumulated the currently available information and world experience in the management of patients receiving emicizumab in order to facilitate decision-making when prescribing and using emicizumab. Information on the use of emicizumab in patients with HA with FVIII inhibitors and severe HA without FVIII inhibitors is presented. Possible complications and measures for their prevention and treatment are presented.

Publisher

National Medical Research Center of Hematology of the Ministry of Health of the Russian Federation

Subject

Hematology

Reference33 articles.

1. MASAC Document 258 — Recommendation on the Use and Management of Emicizumab-kxwh (Hemlibra®) for Hemophilia A with and without Inhibitors. URL: https://www.hemophilia.org/healthcare-professionals/guidelines-on-care/masac-documents/masac-document-258-recommendation-on-the-use-and-management-of-emicizumab-kxwh-hemlibrar-for-hemophilia-a-with-and-without-inhibitors

2. Instruction for use of Emicizumab. URL: https://www.rlsnet.ru/tn_index_id_96727.htm#farmakokinetika (In Russian).

3. Yoneyama K., Schmitt C., Kotani N., et al. A pharmacometric approach to substitute for a conventional dose-finding study in rare diseases: Example of phase III dose selection for emicizumab in hemophilia A. Clin Pharmacokinet. 2018; 57(9): 1123–34. DOI: 10.1007/s40262-017-0616-3.

4. Ito S. Pharmacokinetics 101. Paediatr Child Health. 2011;16(9):535-6. DOI: 10.1093/pch/16.9.535.

5. Klinge J., Auberger K., Auerswald G., et al. Prevalence and outcome of intracranial haemorrhage in haemophiliacs — a survey of the paediatric group of the German Society of Thrombosis and Haemostasis (GTH). Eur J Pediatr. 1999; 158(Suppl 3): S162–5. DOI: 10.1007/pl00014346.

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