Abstract Number: PB1045
Meeting: ISTH 2020 Congress
Background: Immune tolerance induction (ITI) is the only proven method to eradicate inhibitors in haemophilia A patients who develop a neutralising antibody to FVIII. While emicizumab or other bypassing agents are used to control bleeding in patients with inhibitors, they do not eliminate inhibitors. Although a single-centre study has been conducted, there are limited data on the use of ITI and emicizumab in combination and the outcomes of different treatment strategies.
Aims: The overall objectives of MOTIVATE are to characterise different approaches to the treatment and management of patients with haemophilia A and inhibitors and to evaluate the efficacy and safety of ITI and/or emicizumab.
Methods: MOTIVATE is comprised of two multicentre, investigator-initiated trials in the US and Europe. Data from the two trials will be pooled. Participants of any age with haemophilia A of any severity who have developed inhibitors to any FVIII product will be observed for a maximum of five years. Target enrolment is 120. The treatment approaches evaluated are standard ITI with FVIII (with Nuwiq®, octanate® or wilate®) (Group 1), ITI with emicizumab prophylaxis (Group 2), and prophylaxis with emicizumab or bypassing agents (Group 3). Participants are not randomised to any treatment group, and all treatments are at the discretion of the physician.
Results: The primary endpoints are the success of ITI in treatment groups that include ITI and the annualised bleeding rates across all groups. Optional sub-studies will explore factors that may influence ITI outcomes as well as the impact of different treatment approaches on diverse aspects of patient health, including joint and bone health and the risk of thrombosis.
Conclusions: MOTIVATE will collect real-world clinical experience on current approaches for the management of haemophilia A with inhibitors. The results will help facilitate discussions on the optimal treatment approach for people with haemophilia A and inhibitors.
To cite this abstract in AMA style:Escuriola-Ettingshausen C, Sidonio RF, Jr . MOdern Treatment of Inhibitor-PositiVe PATiEnts with Haemophilia A – MOTIVATE [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/modern-treatment-of-inhibitor-positive-patients-with-haemophilia-a-motivate/. Accessed December 11, 2023.
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