Clinical features and treatment of hemophilia B-Reference-Cited by-同舟云学术

Clinical features and treatment of hemophilia B

Published:2024-04-18 Issue:1 Volume:23 Page:192-199
ISSN:2414-9314
Container-title:Pediatric Hematology/Oncology and Immunopathology
language:
Short-container-title:Voprosy gematologii/onkologii i immunopatologii v pediatrii

Author:

Zozulya N. I.¹^ORCID,Andreeva T. A.²^ORCID,Zharkov P. A.³^ORCID,Vdovin V. V.⁴^ORCID

Affiliation:

1. National Medical Research Center for Hematology of of Ministry of Healthcare of the Russian Federation

2. City Center for the Treatment of Hemophilia Patients, City Outpatient Clinic No. 37

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

4. The Morozov Children's City Clinical Hospital of the Department of Health of Moscow

Abstract

Hemophilia B – a deficiency of blood coagulation factor IX (FIX) – is one of the most common hereditary coagulopathies along with hemophilia A and von Willebrand disease. As in hemophilia A, patients with hemophilia B require prophylactic treatment to prevent the development of bleeding and arthropathy, and there is a number of unsolved problems in their treatment. At the same time, the arsenal of drugs for the treatment of hemophilia B is significantly smaller compared to hemophilia A, and therefore the emergence of new drugs for the treatment of FIX deficiency is of great practical importance for doctors and patients. The article provides information about the pathogenesis and clinical course of hemophilia B, discusses the most promising areas in the treatment of this disease, such as innovative recombinant FIX molecules, rebalancing and gene therapy. In addition, we outlined clinical and laboratory criteria indicating the necessity to change treatment in patients with hemophilia B as well as presented clinical cases of patients who were switched to long-acting FIX products. The patients' parents gave their consent to the use of their children's data for research purposes and in publications.

Publisher

Fund Doctors, Innovations, Science for Children

Reference31 articles.

1. Clinical guidelines. Hemophilia. ID: 127. 2023. Clinical guidelines of Ministry of Healthcare of the Russian Federation. [Electronic resource] URL: https://cr.minzdrav.gov.ru/recomend/127_2 (access date 02. 02. 2024) (In Russ.)

2. The World Federation of Hemophilia website. [Electronic resource] URL: https://wfh.org/article/report-on-the-annual-global-survey-2021-now-available/ (access date 02. 02. 2024) (In Russ.)

3. Yafoshkina T.Yu., Florinskiy D.B., Pshonkin A.V., Fedorova D.V., Poletaev A.V., Seregina E.A., et al. Evaluation of the sensitivity and specificity of the Pediatric bleeding questionnaire in various hemorrhagic disorders. Pediatrics. Journal named after G.N. Speransky 2021; 100 (3): 35–41. (In Russ.)

4. Castaman G., Matino D. Hemophilia A and B: molecular and clinical similarities and differences. Haematologica 2019; 104 (9): 1702–9.

5. van den Berg H., Alvarez Roman M., Carcao M., Chambost H., Fischer K., Grentenkort-Andersson N., et al. The type and frequency of bleeds in 870 children with severe hemophilia A and B on primary prophylaxis: Data from the PedNet study group [abstract]. [Electronic resource] URL: https://abstracts.isth.org/abstract/the-type-and-frequency-of-bleeds-in-870-children-with-severe-hemophilia-a-and-b-on-primary-prophylaxis-data-from-the-pednet-study-group (accessed 02. 02. 2024).