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Poster Networking

Inherited Bleeding Disorders and Inhibitors

PB0250 - Exploring clinical profile and patient-reported outcomes of people with non-inhibitor severe hemophilia A: insights from the Spanish cohort of ‘Cost Haemophilia: Socioeconomic Survey’ (CHESS)

Sunday, June 23, 2024
13:45 – 14:45 ICT
Room: Exhibition Hall
Background: Treatment approaches for people with severe haemophilia A (PwSHA) have evolved considerably in recent years.

Aims: This analysis aimed to describe clinical status and patient-reported outcomes (PRO) in a Spanish sample of PwSHA, alongside historical and current clinical characteristics and treatment approaches.

Methods: Descriptive analysis examined retrospective data on non-inhibitor PwSHA receiving factor or emicizumab prophylaxis from the CHESS data platform (2019-2022) (European cross-sectional retrospective, burden-of-illness study) within two non-mutually-exclusive groups: one comprising individuals with a physician questionnaire (full-cohort) and another with patient forms (PRO-cohort).

Results: Of 123 PwSHA enrolled in Spain with ≥6 months on current treatment, 60 (49%) were on emicizumab and 63 (51%) on FVIII prophylaxis, with a mean age of 33.1 and 45.6 (Table 1). Of these, 39 (32%) completed a patient-form (10 on emicizumab and 29 on FVIII) (Table 2). Mean all-bleed annualized bleeding rate (ABR) was 1.0 and 2.4, number of problem joints 1.3 and 1.1, and number of target joints 0.7 and 0.8 for the emicizumab and FVIII cohorts, respectively. Proportions with no chronic pain were equivalent (37%), with moderate/severe pain slightly more common in the emicizumab cohort (52% vs. 48%). Among emicizumab patients with pre-switch bleeding data (n=56), a 2.2 reduction in ABR was observed vs. previous treatment regimen.
PRO analysis revealed similar physical activity participation (~50%), EQ-5D scores and anxiety levels (GAD-7) between cohorts, with lower work (17% vs. 39%) and activity (38% vs. 46%) impairment, depression levels (5.8 vs. 7.20; measured via PHQ-8) and caregiver requirement (30% vs. 41%) for the emicizumab cohort (Table 2).

Conclusion(s): These findings suggest that PwSHA experiencing sub-optimal bleed control and switching to emicizumab generally experience reductions in ABR despite the presence of chronic joint damage. EQ-5D, physical activity and anxiety levels were comparable, with the emicizumab cohort reporting favorable impairment, caregiver requirement and depression levels.