OBJECTIVES:

Prophylactic Factor VIII treatment, recommended for severe hemophilia A (SHA) to control bleeding events, may be associated with significant treatment burden. This analysis aims at quantifying impact of treatment burden on patient-reported outcomes (PROs; EQ-5D, EQ-VAS, work productivity and activity impairment [WPAI]) in adult non-inhibitor SHA patients in the United States.

METHODS:

Cross-sectional data from the ‘Cost of Haemophilia across the US+: a Socioeconomic Survey’ (CHESS US+) patient study were used, with treatment burden defined as “high” (HB) for prophylaxis and “low” (LB) for on-demand. A one-to-one Propensity Score (PS) Matching model with replacement was specified to estimate Average Treatment Effects (ATE), the mean outcome difference between groups. Variables selected for PS calculations included age, BMI, annualized bleeding rate (ABR), Problem Joint number (PJn), specific comorbidities and education, based on statistical and clinical relationships to the PROs. Sensitivity analyses were conducted.

RESULTS:

239 SHA patients were included, 49(20%) and 190(80%) with LB and HB, respectively. PS matching resulted in more balanced cohorts, particularly regarding age, BMI, ABR, and PJn. Pre-matching, mean difference in EQ-5D between HB (0.713) and LB (0.747) was -0.034. Post-matching sample size was n=239 for both cohorts and ATE of HB on EQ-5D was -0.107 (p<0.05) relative to the LB cohort, a clinically meaningful difference. ATE was -3.55 (p=0.208) for EQ-VAS, 2.42 (p=0.666) for activity and -6.13 (p=0.416) for work impairment. Magnitude and statistical significance of results varied in sensitivity analyses (including chronic pain or employment or removing ABR as matching variables for PS generation).

CONCLUSIONS:

This analysis suggests that routine prophylactic infusions may be associated with reduced quality of life after controlling for key clinical parameters, though the impact on work productivity is unclear. Variability in the impact of treatment burden between PROs warrants additional research, particularly in light of the advent of novel therapies with less frequent administration.