ITI Treatment is not First-Choice Treatment in Children with Hemophilia A and Low-Responding Inhibitors: Evidence from a PedNet Study

European Pediatric Network for Haemophilia Management (PedNet), H. Marijke Van Den Berg, Maria Elisa Mancuso, Christoph Königs, Roseline D'Oiron, Helen Platokouki, Torben Stamm Mikkelsen, Jayashree Motwani, Beatrice Nolan, Elena Santagostino

Research output: Contribution to journalArticlepeer-review

Abstract

Introduction 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] < 0.01 IU/mL) born between January 1, 1990 and December 31, 2009 who developed clinically relevant inhibitors and were followed-up for at least 3 years after the first positive inhibitor test. Results A total of 260 patients with inhibitors were identified and 68 of them (26%) had low-responding inhibitors (peak < 5 BU/mL). Five patients were lost to follow-up and 63 were included in this study. The median follow-up was 3.7 years (interquartile range: 3.0-7.5). ITI was started in 51/63 (81%) patients. The median time from ITI start to first negative inhibitor titer was similar with low-dose and high-dose ITI regimens (2.5 and 3.1 months, respectively). Ten of the 12 patients who did not receive ITI were treated with regular prophylaxis and reached a negative titer after a median of 6.5 months. Bleeding rate was low in all patients with no difference between treatment regimens. Conclusion In children with low-responding inhibitors negative titers were reached with regular FVIII treatment irrespective of the regimen (i.e., prophylaxis or ITI).

Original languageEnglish
Pages (from-to)1166-1172
JournalThrombosis and Haemostasis
Volume120
Issue number8
DOIs
Publication statusPublished - 2020

Keywords

  • hemophilia A
  • immune tolerance induction low titer
  • low-responding inhibitors
  • PedNet

ASJC Scopus subject areas

  • Hematology

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