PB1158 - MOdern Treatment of Inhibitor-PositiVe PATiEnts with Haemophilia A – MOTIVATE (www.motivate-study.com)

Background: A number of treatment options are available for haemophilia A patients with inhibitors. While emicizumab and bypassing agents may control bleeding in patients with inhibitors, immune tolerance induction (ITI) is the only method proven to eradicate inhibitors.

Aims: The investigator-initiated, international MOTIVATE study (NCT04023019; EudraCT 2019-003427-38) is investigating the management of haemophilia A patients with inhibitors in real-life clinical practice. The objectives are to capture the different management approaches for haemophilia A patients with inhibitors, document current ITI approaches, and evaluate the efficacy and safety of ITI, emicizumab prophylaxis, and ITI with emicizumab prophylaxis.

Methods: Patients of any age with haemophilia A of any severity and inhibitors will be observed for up to five years. The treatment approaches evaluated are standard ITI with FVIII, FVIII ITI with emicizumab prophylaxis and prophylaxis with emicizumab or bypassing agents (Figure 1). Participants are not randomised, and treatment decisions are at the discretion of the investigators. Primary endpoints are ITI success and annualised bleeding rates. Optional sub-studies will explore factors that influence ITI outcome and the impact of different treatment approaches on patient health, including joint and bone health and the risk of thrombotic events.

Results: Target enrolment is 120 patients. As of December 2021, 29 patients have been recruited at 13 study sites in Croatia, France, Germany, Norway, Spain, Sweden, Switzerland, UK and the US. A total of 48 sites have been selected in Canada, Colombia, Croatia, Finland, France, Germany, Guatemala, Italy, Norway, Spain, Sweden, Switzerland, UK, Ukraine and the US. Data on enrolment by country and baseline characteristics as well as demographics of enrolled patients will be presented.

Conclusion(s): The MOTIVATE study is collecting real-world data on current approaches for management of patients with haemophilia A and inhibitors with the goal of informing decision making for the optimal management of such patients.