Factor VIII inhibitors – Results of immunotolerance induction at the National Haemophilia Centre; new options and fi rst experience with treatment using a bispecifi c antibody (emicizumab) in patients with inhibitors in Slovakia

Czech version

Authors: A. Bátorová; T. Prigancová; D. Jankovičová; A. Kyselová
Authors‘ workplace: Univerzitná nemocnica Bratislava, Slovensko ; Klinika hematológie a transfuziológie LF UK, SZU, Národné hemofi lické centrum – Európske hemofi lické centrum komplexnej starostlivosti
Published in: Transfuze Hematol. dnes,27, 2021, No. 1, p. 63-71.
Category: Case Report
doi: https://doi.org/10.48095/cctahd202163

Overview

Factor VIII (FVIII) inhibitors are the most challenging complication of haemophilia treatment. The priority is to eradicate the inhibitors and induce immune tolerance (ITI) to allow resumption of therapy with FVIII. The success rate of ITI is 60–85%, which means that some patients with inhibitors remain dependent on less effective treatment with bypassing agents. New, non-factor drugs are currently under development for effective prophylaxis in inhibitor patients. The first non-factor agent already in use is emicizumab, a bispecific antibody against factors IXa and X, which has been authorized for prophylaxis not only in patients with inhibitors but also in those without inhibitors. The availability of non-factor treatment can fundamentally change the current paradigm of haemophilia treatment, including access to early prophylaxis and ITI management. We discuss here the importance of ITI and we present the results of ITI in our centre and our first experience with non-factor treatment using emicizumab in three patients aged 3.5–7 years. Emicizumab was indicated in two patients with ITI failure and in one patient after successful ITI because of objective obstacles to pharmacokinetic guided prophylaxis with FVIII. In the absence of bleeding for 7–13 months, we evaluate bispecific antibody prophylaxis as being highly effective

Keywords:

haemophilia A – FVIII inhibitors – immunotolerance induction – emicizumab

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