Intensity of factor VIII treatment and inhibitor development in children with severe hemophilia A: the RODIN study

Author:

Gouw Samantha C.1,van den Berg H. Marijke2,Fischer Kathelijn23,Auerswald Günter4,Carcao Manuel5,Chalmers Elizabeth6,Chambost Hervé7,Kurnik Karin8,Liesner Ri9,Petrini Pia10,Platokouki Helen11,Altisent Carmen12,Oldenburg Johannes13,Nolan Beatrice14,Garrido Rosario Pérez15,Mancuso M. Elisa16,Rafowicz Anne17,Williams Mike18,Clausen Niels19,Middelburg Rutger A.20,Ljung Rolf21,van der Bom Johanna G.2022

Affiliation:

1. Department of Paediatrics, Wilhelmina Children’s Hospital, University Medical Center Utrecht, Utrecht, The Netherlands;

2. Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Utrecht, The Netherlands;

3. Van Creveldkliniek, University Medical Center Utrecht, Utrecht, The Netherlands;

4. Gesundheit Nord, Klinikum Bremen Mitte, Prof.-Hess-Kinderklinik, Bremen, Germany;

5. Division of Haematology/Oncology, Hospital for Sick Children, Toronto, Canada;

6. Department of Haematology, Royal Hospital for Sick Children, Yorkhill, Glasgow, UK;

7. Service d’hématologie pédiatrique, Hôpital La Timone & Aix-Marseille Univ, Marseille, France;

8. Dr. v. Haunersches Kinderspital, University of Munich, Munich, Germany;

9. Hemophilia Center, Department of Haematology, Great Ormond Street Hospital for children, London, UK;

10. Department of Pediatrics, Clinic of Coagulation Disorders, Karolinska Hospital, Stockholm, Sweden;

11. St. Sophia Children’s Hospital, Haemophilia-Haemostasis Unit, Athens, Greece;

12. Unitat Hemofilia, Hospital Traumatologica, Hospital Vall d’Hebron, Barcelona, Spain;

13. Institut für Experimentelle Hämatologie und Transfusionsmedizin, Universitätsklinikum Bonn, Bonn, Germany;

14. Department of Paediatric Haematology, St. James's Hospital, Dublin, Ireland;

15. Hospital General Unidad de Hemofilia, Hospitales Universitarios Virgen del Rocio, Sevilla, Spain;

16. Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico Ca’ Granda, Ospedale Maggiore Policlinico, Milan, Italy;

17. Centre Régional de Traitement de l'Hémophilie et Autres Maladies Bicetre, Service Hématologique, Paris, France;

18. Department of Haematology, The Children’s Hospital, Birmingham, UK;

19. Department of Pediatrics, University Hospital of Aarhus at Skejby, Aarhus, Denmark;

20. Center for Clinical Transfusion Research, Sanquin Foundation, Leiden, The Netherlands;

21. Department of Pediatrics and Malmö Centre for Thrombosis and Haemostasis, Skånes Universitetssjukhus, Malmö, Sweden; and

22. Department of Clinical Epidemiology, Leiden University Medical Center, Leiden, The Netherlands

Abstract

Key Points High-dose intensive factor VIII treatment increases the risk for inhibitor development in patients with severe hemophilia A. In patients with severe hemophilia A, factor VIII prophylaxis decreases inhibitor risk, especially in patients with low-risk F8 mutations.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

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