ITI Treatment is not First-Choice Treatment in Children with Hemophilia A and Low-Responding Inhibitors: Evidence from a PedNet Study
- Resource Type
- Authors
- Elena Santagostino; Christoph Königs; Jayashree Motwani; Roseline d'Oiron; Beatrice Nolan; Maria Elisa Mancuso; Torben Stamm Mikkelsen; H. Marijke van den Berg; Helen Platokouki
- Source
- Van Den Berg, H M, Mancuso, M E, Königs, C, D'Oiron, R, Platokouki, H, Mikkelsen, T S, Motwani, J, Nolan, B, Santagostino, E & European Pediatric Network for Haemophilia Management (PedNet) 2020, ' ITI Treatment is not First-Choice Treatment in Children with Hemophilia A and Low-Responding Inhibitors : Evidence from a PedNet Study ', Thrombosis and Haemostasis, vol. 120, no. 8, pp. 1166-1172 . https://doi.org/10.1055/s-0040-1713097
- Subject
- 0301 basic medicine
medicine.medical_specialty
MEDLINE
Hemorrhage
030204 cardiovascular system & hematology
Severe hemophilia A
Hemophilia A
Drug Administration Schedule
Immune tolerance
03 medical and health sciences
0302 clinical medicine
Isoantibodies
Internal medicine
Medicine
Humans
Registries
immune tolerance induction low titer
Child
low-responding inhibitors
Factor VIII
business.industry
Disease Management
Hematology
Antibodies, Neutralizing
PedNet
Regimen
030104 developmental biology
Desensitization, Immunologic
hemophilia A
business
Follow-Up Studies
- Language
- ISSN
- 2567-689X
Background Limited data exist on the clinical impact of low-responding inhibitors and the requirement for immune tolerance induction (ITI) treatment to establish tolerance, reduce bleeding, and improve outcome. The aim of this article is to describe the therapeutic management of children with severe hemophilia A and low-responding inhibitors and its effect on bleeding phenotype. Methods The REMAIN (Real-life Management of Inhibitors) study is a satellite study of the PedNet registry. It included unselected children with severe hemophilia A (factor VIII [FVIII] Results A total of 260 patients with inhibitors were identified and 68 of them (26%) had low-responding inhibitors (peak Conclusion In children with low-responding inhibitors negative titers were reached with regular FVIII treatment irrespective of the regimen (i.e., prophylaxis or ITI).