OBJECTIVES: The use of different treatment options to manage people with severe haemophilia A (PwSHA) continues to evolve. This analysis aimed to quantify the clinical burden across treatment classes among adult PwSHA receiving prophylaxis in Europe and the Unites States.

METHODS: Cross-sectional data on PwSHA with no inhibitor history on prophylaxis from the ‘Cost of Haemophilia: a Socioeconomic Survey III’ (CHESS III) dataset were described, stratified by current treatment class (extended half-life [EHL] factor VIII (FVIII), standard half-life/plasma-derived [SHL/PD] FVIII, and non-factor treatment [NFT]). Demographic/clinical information was reported by treating physicians, whilst impairment was reported by PwSHA via the Work Productivity and Activity Impairment (WPAI) tool.

RESULTS: Overall, 333 PwSHA were included, with a mean [SD] age of 35.4 [13.7] and BMI of 25.3 [4.0]. Annual bleed rate and annual spontaneous bleed rate were lowest for the EHL cohort (n=62) at 1.3 [1.3] and 0.6 [1.0], respectively, followed by SHL/PD (n=68; 1.7 [3.3]; 1.4[2.8]) and NFT (n=203; 2.4 [4.9]; 1.6 [3.3]). In the latter cohort, target joints and problem joints were more common compared to the other cohorts, which may be related with the higher bleeding frequencies.

Seventy percent (n=232) of all patients were reported to suffer from some level of chronic pain, with moderate/severe levels reported in 16% (n=11), 19% (n=12) and 27% (n=54) of the SHL/PD, EHL and NFT cohorts, respectively.

Of the PwSHA with available work productivity loss (WPL) (n=53) and activity impairment (AI) (n=75) data, WPL ranged from 56.0 (28.6) for EHL to 15.0 (21.2) for SHL/PD, while AI ranged from 56.7 (24.1) for EHL to 42.9 (29.3) for NFT.

CONCLUSIONS: Clinical outcomes showed variation across treatment classes and were observed to be worse for NFTs, particularly for bleeding outcomes. Despite a range of therapeutic options being available, these results suggest remaining unmet need among non-inhibitor SHA patients receiving prophylaxis.