PB1248 - Patient-reported outcomes using the Patient-Reported Outcomes, Burdens and Experiences survey following Emicizumab initiation in patients with severe Hemophilia A without inhibitors at a single centre

Background: Patients with hemophilia A have conventionally required treatment with factor VIII infusions as bleeding prophylaxis. More recently, subcutaneous injections of a monoclonal antibody (Emicizumab/ Hemlibra®) have been demonstrated to decrease bleeding episodes and function as an alternative for prophylaxis without the presence of FVIII inhibitors available to patients in Canada since October 2021.

Aims: The project aim was to gain insight into patient outcomes and experience following the introduction of Emicizumab in patients with severe Hemophilia A as part of regular clinical care and quality improvement in one Centre.

Methods: As of January 2022, 9 patients completed initial full PROBE then shortened surveys at 0, 3 and 6 months after initiation of Emicizumab. Patient-reported outcomes were analyzed using descriptive statistics to assess the patient experience following Emicizumab introduction and to explore the feasibility of this approach. PROBE and EQ5D5L scores are both reported out of 1 and higher scores are associated with improvement in functional outcomes.

Results: Mean PROBE scores at 0, 3 and 6 months were 0.76, 0.82 and 0.81 with median PROBE scores at 0, 3 and 6 months were 0.82, 0.88 and 0.88, respectively. Mean EQ5DL scores at 0, 3 and 6 months of 0.54, 0.59 and 0.60, with median EQ5D5L scores of 0.61, 0.74 and 0.70, respectively.

Conclusion(s): PROBE and EQ5DL scores appeared to increase at 3 months post-Emicizumab initiation and remained either stable or slightly decreased at 6 months on Emicizumab. It was feasible to use the PROBE survey in this aspect of regular clinical care to ensure outcomes beyond bleeding rates are continuously assessed. This project furthers our understanding of the need for patient-reported outcomes for those living with hemophilia.