OC 70.3 - Unmet Medical Needs in Patient-Reported Outcomes and Sports Activity in People with Haemophilia A/B with or without Inhibitors: Historical Data from Patients Entering a Non-Interventional Study

Background: Preventing and treating bleeding episodes while reducing treatment burden, improving quality of life (QoL) and increasing physical activity is the aim of a holistic approach to haemophilia management.

Aims: To describe unmet medical needs of patients with HA/HB with or without inhibitors using their historical treatment burden, QoL and sports activity.

Methods: Historical data were collected from patients entering the non-interventional study, explorer6 (NCT03741881). Male patients ≥12 years old with severe HA (FVIII activity < 1%), severe/moderate HB (FIX activity ≤2%) or HA/HB with inhibitors of any severity were included. Patients with previous/current concizumab or emicizumab treatment were excluded. Each patient was treated according to their country’s standard-of-care (SoC). Historical treatment burden (assessed by Haemophilia Treatment Experience Measure [Hemo-TEM]), QoL (assessed by Short Form 36 Health Survey Version 2 [SF-36v2]) and sports activity (assessed by National Hemophilia Association’s “Playing It Safe: Activity Ratings Chart” [stepsforliving.hemophilia.org]) were collected and described.

Results: Overall, 231 patients (147 without inhibitors and 84 with inhibitors) with a median age of 28 years (range, 12–78 years) were enrolled. Mean Hemo-TEM total scores [standard deviations] tended to be higher in patients without inhibitors treated episodically (HA: 36.1 [30.2]; HB: 28.5 [14.2]) than patients treated with prophylaxis (PPX; HA: 20.9 [14.9]; HB: 16.2 [11.9]). SF-36v2 summary scores were lowest in patients with inhibitors and/or treated episodically (Figure 1). In the month before enrolment, lower proportions of patients with inhibitors and/or treated episodically performed sports activities and rarely those considered high-risk (Figure 2).

Conclusion(s): We describe treatment burden, QoL and sports activity in patients with HA/HB, with or without inhibitors, from >30 countries. Patients treated episodically had a higher treatment burden and rarely participated in sports in high-risk categories. Emerging treatments may help to address unmet needs and treatment burden and improve QoL for patients with haemophilia.