Abstract Number: PB0608
Meeting: ISTH 2021 Congress
Background: Immune tolerance induction (ITI) is the only proven method to eradicate inhibitors against FVIII in haemophilia A patients. While emicizumab and bypassing agents may be used to control bleeding in patients with inhibitors, they do not eradicate the inhibitor.
Aims: To capture different approaches to the management of patients with haemophilia A and inhibitors, document current ITI approaches from real-world clinical experience, and evaluate the efficacy and safety of ITI, emicizumab prophylaxis, and ITI with emicizumab prophylaxis.
Methods: The investigator-initiated, international MOTIVATE study (NCT04023019; EudraCT 2019-003427-38) is investigating in real-life clinical practice the management of patients with haemophilia A and inhibitors. Participants of any age with haemophilia A of any severity and inhibitors will be observed for up to five years. The treatment approaches evaluated (Figure 1) are standard ITI with FVIII (Group 1), FVIII ITI with emicizumab prophylaxis (Group 2) and prophylaxis with emicizumab or bypassing agents (Group 3). Participants are not randomised and all treatments are at the discretion of the physician. The primary endpoints are success of ITI and annual bleeding rates. Optional sub-studies will explore factors that may influence ITI outcome and the impact of different treatment approaches on aspects of patient health, including joint and bone health and the risk of thrombotic events.
Results: Target enrolment is 120 patients. As of 1 March 2021, 15 patients have been enrolled at seven study sites in Croatia, France, Germany and Switzerland. A total of 35 sites have been selected so far in 13 counties, namely the US, Canada, UK, Finland, France, Germany, Switzerland, Croatia, Norway, Spain, Sweden, Colombia and Taiwan.
Conclusions: The MOTIVATE study is collecting real-world data on current approaches for management of patients with inhibitors. These data will provide guidance to healthcare professionals for the optimal management of patients with haemophilia A and inhibitors.
To cite this abstract in AMA style:Escuriola Ettingshausen C, Sidonio RF, J. MOdern Treatment of Inhibitor-PositiVe PATiEnts with Haemophilia A – MOTIVATE (www.motivate-study.com) [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/modern-treatment-of-inhibitor-positive-patients-with-haemophilia-a-motivate-www-motivate-study-com/. Accessed October 19, 2021.
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