OBJECTIVES: Understanding how treatment decisions impact the clinical and humanistic burden of people with severe hemophilia A (PwSHA) is crucial in order to evaluate how treatment decisions impact chronic and acute outcomes. This analysis aimed to evaluate changes in outcomes for PwSHA receiving continuous prophylactic treatment with Factor VIII (FPX) and non-factor replacement therapies (NRTs).

METHODS: Longitudinal data on PwSHA with no inhibitor history treated with continuous prophylaxis from waves II and III (2019-2020;2022) of the ‘Cost of Hemophilia: a Socioeconomic Survey’ (CHESS) study were analyzed descriptively. Demographic, clinical and treatment pattern information was reported by treating physicians, whilst humanistic outcomes were reported by PwSHA.

RESULTS: Thirty-five PwSHA (nearly all from Spain and Italy) met the inclusion criteria. Mean (SD) age at baseline was 37.1 (13.4) years and, between waves, 57% (n=20) remained on FPX, while 43% (n=15) switched from FPX to NRTs. Between waves, annual bleed rate (ABR) showed a mean reduction 3.5 (2.9), with those switching to NRTs experiencing the largest improvement (4.3 [2.4]), albeit from a higher baseline (5.5 [2.7]); PwSHA remaining on FPX had an ABR reduction of 2.9 (3.1) from a 3.7 (2.7) baseline. Target joint number reductions were reported in 51% (n=18) of the sample, with 34% (n=12) experiencing no changes. Despite this, 66% experienced an increase in the number of problem joints. General reductions in chronic pain levels were observed, however, the proportion with some level of pain remained consistent (n=6; 83%).

Although adherence increased overall, sub-optimal and non-adherence was observed (n=8; 23%). Among those who remained on FVIII prophylaxis, 12-month FVIII usage increased, on average, by 62,860 IUs, while it decreased substantially for those switching to NRTs.

CONCLUSIONS: This study highlights that chronic areas of unmet need remain despite improvement of outcomes have been observed with evolution in treatment.