The use of emicizumab in children with hemophilia A without inhibitors in the real-world clinical setting
- Authors: Zharkov P.A.1, Voronin K.A.1, Florinskiy D.B.1,2, Andreeva T.A.3, Batdiyeva D.B.4, Goroshkova M.Y.5, Ignatyev S.V.6, Kreining V.V.7, Medova K.Y.8, Osmulskaya N.S.7, Petrov V.Y.9, Plaksina O.I.10, Shiller E.E.2, Elgakaeva K.M.11
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Affiliations:
- 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
- Hemophilia Treatment Center at City Outpatient Clinic No. 37
- Karachay-Cherkess Republican Clinical Hospital
- The Yu.E. Malakhovsky Kuzbass Children's Clinical Hospital
- Kirov Research Institute of Hematology and Blood Transfusion of Federal Medical-Biological Agency
- Regional Children’s Clinical Hospital
- The A.O. Akhushkov Ingush Republican Clinical Hospital
- The Morozov Children's City Clinical Hospital of the Department of Health of Moscow
- Nizhny Novgorod Regional Children's Hospital
- The E.P. Glinka Republican Children’s Clinical Hospital
- Issue: Vol 22, No 4 (2023)
- Pages: 96-101
- Section: ORIGINAL ARTICLES
- Submitted: 06.01.2024
- Accepted: 06.01.2024
- Published: 20.12.2023
- URL: https://hemoncim.com/jour/article/view/807
- DOI: https://doi.org/10.24287/1726-1708-2023-22-4-96-101
- ID: 807
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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.
Keywords
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 FederationK. 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 FederationD. 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 FederationT. A. Andreeva
Hemophilia Treatment Center at City Outpatient Clinic No. 37
ORCID iD: 0000-0002-5649-325X
Saint Petersburg
Russian FederationD. B. Batdiyeva
Karachay-Cherkess Republican Clinical Hospital
ORCID iD: 0009-0006-9036-4669
Cherkessk
Russian FederationM. Yu. Goroshkova
The Yu.E. Malakhovsky Kuzbass Children's Clinical Hospital
ORCID iD: 0000-0002-4593-7556
Novokuznetsk
Russian FederationS. V. Ignatyev
Kirov Research Institute of Hematology and Blood Transfusion of Federal Medical-Biological Agency
ORCID iD: 0000-0002-8457-2967
Kirov
Russian FederationV. V. Kreining
Regional Children’s Clinical Hospital
ORCID iD: 0009-0006-7788-896X
Yekaterinburg
Russian FederationKh. Ya. Medova
The A.O. Akhushkov Ingush Republican Clinical Hospital
ORCID iD: 0009-0002-2635-1645
Nazran
Russian FederationN. S. Osmulskaya
Regional Children’s Clinical Hospital
ORCID iD: 0000-0002-5224-0677
Omsk
Russian FederationV. Yu. Petrov
The Morozov Children's City Clinical Hospital of the Department of Health of Moscow
ORCID iD: 0000-0002-4583-1137
Moscow
Russian FederationO. I. Plaksina
Nizhny Novgorod Regional Children's Hospital
ORCID iD: 0000-0001-8374-6358
Nizhny Novgorod
Russian FederationE. E. Shiller
Odintsovo Regional Hospital of the Department of Health of the Moscow Region
ORCID iD: 0000-0003-0434-6474
Odintsovo
Russian FederationKh. M. Elgakaeva
The E.P. Glinka Republican Children’s Clinical Hospital
ORCID iD: 0000-0002-9292-7684
Grozny
Russian FederationReferences
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