Bleeding rate reduction in children with hemophilia A and inhibitors treated with emicizumab in the real-world clinical setting

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  • Authors: Zharkov P.А.1, Voronin K.A.1, Andreeva T.A.2, Asekretova T.V.3, Belkina Y.E.4, Demikhov V.G.5, Zinina E.E.6, Kolyasina T.A.7, Lebedev V.V.3, Markova I.V.8, Osmulskaya N.S.9, Petrov V.Y.10, Skobin V.B.5, Spichak O.V.11, Shelekhova T.V.12, Sherstnev D.G.12
  • Affiliations:
    1. Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation
    2. Hemophilia Treatment Center at City Outpatient Clinic No. 37
    3. Regional Children’s Clinical Hospital of the Ministry of Healthcare of the Krasnodar Region
    4. Simferopol City Children’s Clinical Hospital
    5. Scientific and Clinical Center of Hematology, Oncology, and Immunology of Ryazan State Medical University named after Academician I.P. Pavlov, Ministry of Healthcare of the Russian Federation
    6. Surgut District Clinical Hospital
    7. Regional Children’s Hospital
    8. R.M. Gorbacheva Research Institute for Pediatric Oncology, Hematology and Transplantation of the I.P. Pavlov First Saint-Petersburg State Medical University of Ministry of Healthcare of the Russian Federation
    9. Regional Children’s Clinical Hospital
    10. Morozov Children’s City Clinical Hospital of the Department of Health of Moscow
    11. Nizhnevartovsk District Children’s Clinical Hospital
    12. V.I. Razumovsky Saratov State Medical University of Ministry of Healthcare of the Russian Federation
  • Issue: Vol 21, No 1 (2022)
  • Pages: 66-71
  • Section: ORIGINAL ARTICLES
  • Submitted: 28.03.2022
  • Accepted: 28.03.2022
  • Published: 28.03.2022
  • URL: https://hemoncim.com/jour/article/view/590
  • DOI: https://doi.org/10.24287/1726-1708-2022-21-1-66-71
  • ID: 590

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Abstract

There are only limited data coming from isolated case reports regarding the real-world use of emicizumab for the treatment of children with hemophilia A and inhibitors (HAI) in Russia. The aim of the study was to evaluate the efficacy and safety of emicizumab prophylaxis in children with severe HAI. Ethical approval was not required since the study only involved the use of anonymized and generalized retrospective data obtained during routine clinical practice. We retrospectively analyzed medical records of children with HAI who had been treated with emicizumab at 11 institutions located in Russia, taking into consideration such parameters as annualized bleeding rates (ABR), annualized spontaneous bleeding rates (ASBR), annualized joint bleeding rates (AJBR) and annualized bleeding rates for bleeds requiring additional therapy (ABRRT), as well as the presence and severity of adverse events during the treatment. The median age of patients at the time of initiation of emicizumab prophylaxis was 65 (11–170) months. Before the treatment, ABR was 19.9 (95% confidence interval (CI), 15.4–26.1), ASBR – 13.6 (95% CI, 10.6–17.8), AJBR – 6.6 (95% CI, 4.7–9.7), ABRRT – 16.6 (95% CI, 12.4–22.7). After the initiation of the treatment, bleeding rates changed dramatically: ABR decreased by 98.6% (95% CI, 96.7–99.4), AJBR – by 99.4% (95% CI, 95.3–99.9), ABRRT – by 98.8% (95% CI, 96.8–99.6); and there were no signs of spontaneous bleeding during 10 (1–32) months of treatment. No adverse events leading to the interruption or discontinuation of the treatment with emicizumab were reported. The use of emicizumab in children with HAI in the real-world clinical setting results in a significant (> 98%) and safe reduction in bleeding episodes without any signs of spontaneous bleeding.

About the authors

P. А. Zharkov

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 hematologist at the Outpatient Department, Head of the Department of Hemostasis Disorder Research

1 Samory Mashela St., Moscow 117997 

Russian Federation

K. A. Voronin

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

T. A. Andreeva

Hemophilia Treatment Center at City Outpatient Clinic No. 37

Saint Petersburg

Russian Federation

T. V. Asekretova

Regional Children’s Clinical Hospital of the Ministry of Healthcare of the Krasnodar Region

Krasnodar

Russian Federation

Yu. E. Belkina

Simferopol City Children’s Clinical Hospital

Simferopol

Russian Federation

V. G. Demikhov

Scientific and Clinical Center of Hematology, Oncology, and Immunology of Ryazan State Medical University named after Academician I.P. Pavlov, Ministry of Healthcare of the Russian Federation

ORCID iD: 0000-0002-6120-4678

Ryazan

Russian Federation

E. E. Zinina

Surgut District Clinical Hospital

Surgut

Russian Federation

T. A. Kolyasina

Regional Children’s Hospital

Yuzhno-Sakhalinsk

Russian Federation

V. V. Lebedev

Regional Children’s Clinical Hospital of the Ministry of Healthcare of the Krasnodar Region

Krasnodar

Russian Federation

I. V. Markova

R.M. Gorbacheva Research Institute for Pediatric Oncology, Hematology and Transplantation of the I.P. Pavlov First Saint-Petersburg State Medical University of Ministry of Healthcare of the Russian Federation

ORCID iD: 0000-0001-5861-7319

Saint Petersburg

Russian Federation

N. S. Osmulskaya

Regional Children’s Clinical Hospital

Omsk

Russian Federation

V. Yu. Petrov

Morozov Children’s City Clinical Hospital of the Department of Health of Moscow

Moscow

Russian Federation

V. B. Skobin

Scientific and Clinical Center of Hematology, Oncology, and Immunology of Ryazan State Medical University named after Academician I.P. Pavlov, Ministry of Healthcare of the Russian Federation

Ryazan

Russian Federation

O. V. Spichak

Nizhnevartovsk District Children’s Clinical Hospital

ORCID iD: 0000-0003-3918-6781

Nizhnevartovsk

Russian Federation

T. V. Shelekhova

V.I. Razumovsky Saratov State Medical University of Ministry of Healthcare of the Russian Federation

Saratov

Russian Federation

D. G. Sherstnev

V.I. Razumovsky Saratov State Medical University of Ministry of Healthcare of the Russian Federation

Saratov

Russian Federation

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Copyright (c) 2022 Zharkov P.А., Voronin K.A., Andreeva T.A., Asekretova T.V., Belkina Y.E., Demikhov V.G., Zinina E.E., Kolyasina T.A., Lebedev V.V., Markova I.V., Osmulskaya N.S., Petrov V.Y., Skobin V.B., Spichak O.V., Shelekhova T.V., Sherstnev D.G.

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