Preliminary Study Of Immune Tolerance Induction In The Treatment Of Paediatric Haemophilia A With Inhibitors
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Date
2017
Authors
Aziz, Azmanira
Journal Title
Journal ISSN
Volume Title
Publisher
Universiti Sains Malaysia
Abstract
Introduction
One of the primary complications in the treatment of haemophilia A patient is the development of inhibitor. Over the last 30 years, Immune Tolerance Induction (ITI) has been used to eliminate inhibitor as well as to restore FVIII pharmacokinetics. ITI is the best long term device in eliminating inhibitors with its success rate varying from 60% to 90%. The purpose of this study is to investigate the patient’s demography, clinical characteristics and the result from ITI therapy at the National Blood Centre.
Method
This cross sectional retrospective study is undertaken by reviewing the files of 18 severe haemophilia A paediatric patients with inhibitors who underwent ITI therapy at the National Blood Centre from 2002 till June 2016. All information had been obtained from the patient’s file and entered into a standardized research proforma. The data were included patient’s demographics, type of FVIII gene mutation, type of FVIII product, FVIII dosing, inhibitor history, inhibitor level during ITI, duration of ITI therapy and the outcomes. The successful outcome was defined as negative titre inhibitors, FVIII level were normalized and no trace of amnestic upon subsequent FVIII exposure.
Result
Twelve (66.6%) patients out of 18 from this study successfully eliminated inhibitors. This study also demonstrated that statistically there was a significant association with the historical peak inhibitor before start ITI (p = 0.015), peak of inhibitor during ITI (p = 0.018), inhibitor titre before ITI (p = 0.036), inhibitor at the start of ITI (p = 0.011) and the duration between inhibitor detection and the start of ITI (p = 0.046) with the final outcome of ITI therapy.
Conclusion
This study has demonstrated that ITI therapy can be an effective treatment and can be accepted as inhibitor eliminator. The successful ITI factors includes the history of low inhibitor titre peak prior to therapy (< 200BU/ml), low inhibitor titre peak during ITI, low inhibitor titre before and at the beginning of ITI (<10BU/ml). The shorter duration of starting ITI therapy after inhibitor detection could influence the ITI outcome with higher success rate. This is a good preliminary study that provides an understanding and helping the physicians in patient selection to start ITI therapy for a better success rate.
Description
Keywords
Immune Tolerance Induction , Treatment of Paediatric Haemophilia.