The use of emicizumab in children with hemophilia A without inhibitors in the real-world clinical setting

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Abstract

In our country, the use of emicizumab in children with hemophilia A without inhibitors (HA) in the real-world clinical setting is limited and is available only as few individual case reports. Our aim was to evaluate the effectiveness and safety of the prophylactic use of emicizumab in children with severe HA in the real-world clinical setting. We conducted a retrospective analysis of medical records of children with HA who had received emicizumab at 9 centers based in the Russian Federation. We assessed the annualized bleeding rate (ABR), annualized spontaneous bleeding rate (ASBR), annualized joint bleeding rate (AJBR), annualized bleeding rate for bleeding episodes that required additional treatment with FVIII concentrate (ABRRT) and the number of hospital admissions for bleeding both before and after the treatment with emicizumab, as well as the occurrence and severity of adverse events during the therapy. Ethics committee approval was not required for this study because it involved the use of aggregated retrospective data from routine clinical practice that were fully anonymized. Two emicizumab administration regimens were compared with regard to their effectiveness. Before the treatment with emicizumab, ABR was 5.38 (95% confidence interval (CI) 3.90–7.64), ASBR – 4.16 (95% CI 2.99–5.94), AJBR – 2.7 (95% CI 1.87–4.03), and ABRRT – 4.8 (95% CI 3.37–7.08). After the initiation of the treatment with emicizumab, the bleeding rate plummeted: ABR decreased by 93.9% (95% CI 88.8–96.7), ASBR – by 96.9% (95% CI 93.1–98.6), AJBR – by 96.1% (95% CI 90.4–98.4%) and ABRRT – by 95.1% (95% CI 90.0–97.6). During the treatment with emicizumab, the rate of bleeding episodes that required hospital admission decreased from 1.58 (95% CI 0.98–2.68) to 0.04 (0.01–0.10), which amounted to 97.6% (95% CI 91.1–99.4). The median follow-up time for the patients treated with emicizumab was 15.5 months (range 9–29 months). When comparing the annualized bleeding rates in the groups of the patients who were preventively treated with emicizumab at doses of 3 mg/kg (administered once every 2 weeks) and 1.5 mg/kg (once per week), we didn't find any statistically significant differences. In the real-world clinical setting, the use of emicizumab in the children with HA led to a significant reduction in all bleeding episodes (by more than 90%), regardless of the administration regimen.

About the authors

P. 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

Pavel A. Zharkov, Dr. Med. Sci., a pediatrician, a  hematologist at the Outpatient Department, Head of the Laboratory of Hemostasis Disorder Research, Professor at the Department of Hematology and Cell Technologies

1 Samory Mashela St., Moscow 117997

Russian Federation

K. A. Voronin

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

ORCID iD: 0000-0001-7578-9657

Moscow

Russian Federation

D. B. Florinskiy

The Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation;
Odintsovo Regional Hospital of the Department of Health of the Moscow Region

Moscow;

Odintsovo

Russian Federation

T. A. Andreeva

Hemophilia Treatment Center at City Outpatient Clinic No. 37

ORCID iD: 0000-0002-5649-325X

Saint Petersburg

Russian Federation

D. B. Batdiyeva

Karachay-Cherkess Republican Clinical Hospital

ORCID iD: 0009-0006-9036-4669

Cherkessk

Russian Federation

M. Yu. Goroshkova

The Yu.E. Malakhovsky Kuzbass Children's Clinical Hospital

ORCID iD: 0000-0002-4593-7556

Novokuznetsk

Russian Federation

S. V. Ignatyev

Kirov Research Institute of Hematology and Blood Transfusion of Federal Medical-Biological Agency

ORCID iD: 0000-0002-8457-2967

Kirov 

Russian Federation

V. V. Kreining

Regional Children’s Clinical Hospital

ORCID iD: 0009-0006-7788-896X

Yekaterinburg

Russian Federation

Kh. Ya. Medova

The A.O. Akhushkov Ingush Republican Clinical Hospital

ORCID iD: 0009-0002-2635-1645

Nazran

Russian Federation

N. S. Osmulskaya

Regional Children’s Clinical Hospital

ORCID iD: 0000-0002-5224-0677

Omsk

Russian Federation

V. Yu. Petrov

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

ORCID iD: 0000-0002-4583-1137

Moscow

Russian Federation

O. I. Plaksina

Nizhny Novgorod Regional Children's Hospital

ORCID iD: 0000-0001-8374-6358

Nizhny Novgorod

Russian Federation

E. E. Shiller

Odintsovo Regional Hospital of the Department of Health of the Moscow Region

ORCID iD: 0000-0003-0434-6474

Odintsovo

Russian Federation

Kh. M. Elgakaeva

The E.P. Glinka Republican Children’s Clinical Hospital

ORCID iD: 0000-0002-9292-7684

Grozny

Russian Federation

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Copyright (c) 2023 Zharkov P.A., Voronin K.A., Florinskiy D.B., Andreeva T.A., Batdiyeva D.B., Goroshkova M.Y., Ignatyev S.V., Kreining V.V., Medova K.Y., Osmulskaya N.S., Petrov V.Y., Plaksina O.I., Shiller E.E., Elgakaeva K.M.

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