Treatment of Haemophilia A Without Inhibitors: Real-World Treatment Patterns and Clinical Outcomes in the US.

INTRODUCTION

Despite available prophylactic therapies for haemophilia A, breakthrough bleeding and consequent pain and joint damage still occur.

AIM: To provide insights into the unmet treatment needs of people with moderate-to-severe haemophilia A in the US.

METHODS

For this descriptive, observational cohort study, eligible participants diagnosed with moderate-to-severe haemophilia A were enrolled in the PicnicHealth database on or before 3 October 2022. Three patient groups were established to identify: (1) treatment patterns; (2) clinical outcomes by therapy class (standard half-life [SHL] factor VIII [FVIII]; extended half-life FVIII [EHL]; emicizumab); (3) health-related quality of life (HRQoL) using the Patient-Reported Outcomes Measuring Instruments System (PROMIS)-29 survey and a supplemental pain survey. Patients switching treatment classes were included in multiple treatment groups.

RESULTS

Of 211 participants in the 'treatment patterns population', 123 (58.3%) used SHL FVIII prophylaxis, 81 (38.4%) EHL FVIII prophylaxis, and 83 (39.3%) emicizumab prophylaxis. In the 'clinical outcomes population', 63 participants (70.8%) receiving SHL, 43 (61.4%) EHL and 36 (62.1%) emicizumab had ≥1 bleed at Year 1. Pain-related events within the past year occurred in 18 (12.8%), 16 (18.2%) and 10 (12.2%) participants, respectively. The most frequent patient-reported symptoms were 'sleep disturbance' (84.5%), 'inability to participate in social roles and activities' (69.7%) and 'fatigue' (66.9%). The most impacted PROMIS scores were anxiety and pain interference.

CONCLUSION

Regardless of the prophylaxis product class used, most participants experienced ≥1 bleeding event per year and reduced QoL. Further treatment optimisation may be needed to prevent bleeding episodes and improve patient QoL.