ITI Treatment is not First-Choice Treatment in Children with Hemophilia A and Low-Responding Inhibitors: Evidence from a PedNet Study
| Autor: | Elena Santagostino, Christoph Königs, Jayashree Motwani, Roseline d'Oiron, Beatrice Nolan, Maria Elisa Mancuso, Torben Stamm Mikkelsen, H. Marijke van den Berg, Helen Platokouki |
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| Rok vydání: | 2020 |
| Předmět: |
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 |
| Zdroj: | 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 |
| ISSN: | 2567-689X |
| Popis: | 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). |
| Databáze: | OpenAIRE |
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