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Immune tolerance in children with factors VIII and IX inhibitors: a single centre experience
1Haemophilia Centre, Great Ormond Street Hospital for Children NHS Trust, London, UK.
Insights
Immune tolerance effectively treats alloimmune inhibitors in severe hemophilia (FVIII/FIX). Successful treatment in 80% of boys depended on early intervention and low initial inhibitor levels. This approach offers a viable management strategy.
Area of Science:
- Hematology
- Immunology
- Pediatrics
Background:
- Alloimmune inhibitors against Factor VIII (FVIII) and Factor IX (FIX) are critical complications in hemophilia management.
- Optimal strategies for managing these inhibitors remain a significant clinical challenge.
Purpose of the Study:
- To evaluate the efficacy of immune tolerance therapy in boys with severe hemophilia and alloimmune inhibitors.
- To identify factors associated with successful immune tolerance induction.
Main Methods:
- Retrospective analysis of 15 boys with severe hemophilia (14 A, 1 B) undergoing immune tolerance over 9 years.
- Varying dose intensities of FVIII/FIX concentrate were used to induce tolerance.
- Key factors analyzed included inhibitor titre, duration of inhibitor presence, and time to tolerance.
Main Results:
- Successful immune tolerance was achieved in 12 out of 15 boys (80%).
- Factors predicting success included lower maximum inhibitor titres and shorter intervals between inhibitor diagnosis and initiation of immune tolerance.
- Time to tolerance ranged from 1 to 27 months, with no recurrences observed.
Conclusions:
- Immune tolerance is an effective treatment for alloimmune FVIII and FIX inhibitors in severe hemophilia.
- Early initiation of immune tolerance and management of low inhibitor titres are crucial for successful outcomes.
- Longer inhibitor duration prior to treatment was associated with treatment failure.
Abstract:
Alloimmune FVIII and FIX inhibitors are the most serious complication of haemophilia in the postviral contamination era and their optimal management remains controversial. We present 15 boys with severe haemophilia (14 with haemophilia A and 1 with haemophilia B) who have received immune tolerance at our centre over a 9-year period. Twelve of them (80%) were successfully tolerized with varying dose intensities, but three of them (including the boy with haemophilia B) failed tolerization. The factors, which were associated with successful tolerance in our group, were a low maximum inhibitor titre and a short interval between diagnosis of the inhibitor and the start of immune tolerance. The time taken to achieve immune tolerance varied from 1 to 27 months and none of the inhibitors have recurred. Two of the three boys who failed immune tolerance had had their inhibitor for 72 and 69 months, respectively before tolerance was attempted.
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