Тромбоз, гемостаз и реология

Tromboz, Gemostaz I Reologiya
scientific and practical journal

ISSN 2078–1008 (Print); ISSN 2687-1483 (online)
2024, Original papers
2024

Changes in clinical and laboratory parameters in adolescents and young adults with hemophilia A after switching from standard half-life factor VIII to efmoroctocog alfa: 616.151.5

Original papers
Published 2024-10-11
Nadezhda I. Zozulya
National Medical Research Center for Hematology, Ministry of Health of the Russian Federation; 4 Novy Zykovsky Рroezd, Moscow 125167, Russia;
ФГБУ «Национальный медицинский исследовательский центр гематологии» Министерства здравоохранения Российской Федерации; Россия, 125167 Москва, Новый Зыковский проезд, 4;
https://orcid.org/0000-0001-7074-0926 (unauthenticated)
Tatiana A. Andreeva
Municipal Polyclinic No. 37, City Centre for Treatment of Haemophilia Patients; 6 Gorokhovaya Str., Saint Petersburg 191186, Russia;
СПб ГБУЗ «Городская поликлиника No 37», Городской центр по лечению гемофилии; Россия, 191186 Санкт-Петербург, Гороховая ул., 6;
https://orcid.org/0000-0002-5649-325X (unauthenticated)
Viсtor Yu. Petrov
Morozovskaya Children’s City Clinical Hospital, Moscow Healthcare Department; 1/9 4th Dobryninskiy Pereulok, Moscow 119049, Russia;
ГБУЗ «Морозовская детская городская клиническая больница Департамента здравоохранения города Москвы», Отделение гематологии; Россия, 119049 Москва, 4-й Добрынинский переулок, 1/9;
https://orcid.org/0000-0002-4583-1137 (unauthenticated)
Valeriia N. Konstantinova
Municipal Polyclinic No. 37, City Centre for Treatment of Haemophilia Patients; 6 Gorokhovaya Str., Saint Petersburg 191186, Russia;
СПб ГБУЗ «Городская поликлиника No 37», Городской центр по лечению гемофилии; Россия, 191186 Санкт-Петербург, Гороховая ул., 6;
https://orcid.org/0000-0003-0452-0145 (unauthenticated)
Olga A. Krasheninnikova
Municipal Polyclinic No. 37, City Centre for Treatment of Haemophilia Patients; 6 Gorokhovaya Str., Saint Petersburg 191186, Russia;
СПб ГБУЗ «Городская поликлиника No 37», Городской центр по лечению гемофилии; Россия, 191186 Санкт-Петербург, Гороховая ул., 6;
https://orcid.org/0000-0002-7431-9546 (unauthenticated)
Inna N. Lavrentyeva
Morozovskaya Children’s City Clinical Hospital, Moscow Healthcare Department; 1/9 4th Dobryninskiy Pereulok, Moscow 119049, Russia;
ГБУЗ «Морозовская детская городская клиническая больница Департамента здравоохранения города Москвы», Отделение гематологии; Россия, 119049 Москва, 4-й Добрынинский переулок, 1/9;
https://orcid.org/0000-0003-2747-695X (unauthenticated)
Pavel V. Svirin
Morozovskaya Children’s City Clinical Hospital, Moscow Healthcare Department; 1/9 4th Dobryninskiy Pereulok, Moscow 119049, Russia;
ГБУЗ «Морозовская детская городская клиническая больница Департамента здравоохранения города Москвы», Отделение гематологии; Россия, 119049 Москва, 4-й Добрынинский переулок, 1/9;
Oleg G. Melikhov
League of Clinical Research; 1 office 1008, Ulofa Palme Str., Moscow 119285, Russia
РОО «Лига содействия клиническим исследованиям»; Россия, 119285 Москва, ул. Улофа Пальме, 1, офис 1008
https://orcid.org/0000-0001-9442-7707 (unauthenticated)

Keywords

efmoroctocog alfa
hemophilia A
blood clotting factor VIII
FVIII
bleedings
  Full Text

Abstract

Summary. Introduction. Hemophilia is a hereditary genetic disease associated with a deficiency of blood clotting factors, which results in bleeding in various locations. The fundamental tenet of hemophilia treatment is specific replacement therapy with deficient blood clotting factors. Аim: to study changes in clinical and laboratory parameters in patients with hemophilia A after switching from standard half-life factor VIII (FVIII) preparations to efmoroctocog alfa for 12 months. Materials and Methods. An open, multicenter, non-comparative, observational single-arm study was conducted in a routine clinical setting. The study included 30 patients with hemophilia A of any severity aged 12 to 35 years with an average annual bleeding frequency of ≥ 2 episodes and prophylactic therapy with FVIII for at least one year. The patients were divided into two groups: 12–16 years (n=8) and 17–35 years (n=22). For each patient, data were collected during 5 visits over 12 months. Results. Treatment with efmoroctocog alfa, a prolonged-half-life agent, for 12 months resulted in improvements in clinical and laboratory parameters in patients with hemophilia A switched from a standard-half-life FVIII. In the 12–16-year age group, three patients experienced at least one bleeding episode, with a total of 5 bleeds occurring during the first 6 months. The mean annualized bleeding rate was 0.6. In the 17–35-year age group, 10 patients experienced at least one bleeding episode, with a total of 31 bleeds during the study period. The mean annualized bleeding rate was 1.44. No serious adverse events were reported. Conclusion. Positive changes were observed already in the first 6 months of the study in both age groups and included changes in bleedings frequency, the number of invasive procedures and surgeries, as well as improvements in joints condition. The results obtained confirm the high efficacy and safety of efmoroctocog alfa in the prophylactic treatment of patients with hemophilia A.

For citation: Zozulya N.I., Andreeva T.A., Petrov V.Yu., Konstantinova V.N., Krasheninnikova O.A., Lavrentyeva I.N., Svirin P.V., Melikhov O.G. Changes in clinical and laboratory parameters in adolescents and young adults with hemophilia A after switching from standard half-life factor VIII to efmoroctocog alfa. Tromboz, gemostaz i reologiya. 2024;(3):97–106. (In Russ.).

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