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

H Marijke van den Berg1, Maria Elisa Mancuso2, Christoph Königs3

  • 1PedNet Foundation, Baarn, The Netherlands.

Thrombosis and Haemostasis
|June 24, 2020
PubMed

Insights

Children with severe hemophilia A and low-responding inhibitors achieve negative inhibitor titers with regular factor VIII (FVIII) treatment. Both immune tolerance induction (ITI) and prophylaxis are effective, with similar outcomes in reducing bleeding events.

Area of Science:

  • Pediatric Hematology
  • Hemophilia Treatment
  • Immunology

Background:

  • Limited data exist on the clinical impact of low-responding inhibitors in severe hemophilia A.
  • Understanding the efficacy of immune tolerance induction (ITI) is crucial for managing these patients.
  • This study addresses the therapeutic management and outcomes for children with severe hemophilia A and low-responding inhibitors.

Purpose of the Study:

  • To describe the therapeutic management of children with severe hemophilia A and low-responding inhibitors.
  • To evaluate the effect of treatment regimens on bleeding phenotype.
  • To assess the time to achieve negative inhibitor titers.

Main Methods:

  • The REMAIN study analyzed data from children with severe hemophilia A (factor VIII [FVIII] < 0.01 IU/mL) and clinically relevant inhibitors.
  • Included were children born between 1990-2009, followed for at least 3 years post-diagnosis.
  • 63 patients with low-responding inhibitors (peak < 5 BU/mL) were included.

Main Results:

  • 81% of patients initiated immune tolerance induction (ITI).
  • Negative inhibitor titers were reached similarly with low-dose and high-dose ITI (median 2.5-3.1 months).
  • Patients not receiving ITI achieved negative titers with regular prophylaxis (median 6.5 months); bleeding rates were low across all regimens.

Conclusions:

  • Children with low-responding inhibitors can achieve negative titers with regular factor VIII (FVIII) treatment.
  • Both prophylaxis and ITI regimens are effective in managing low-responding inhibitors in severe hemophilia A.
  • Treatment choice did not significantly impact bleeding rates in this cohort.
Abstract

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