The humanistic and economic burden of problem joints for children and adults with moderate or severe haemophilia A: Analysis of the CHESS population studies

INTRODUCTION: Adequate prophylactic treatment and physical activity improve joint health and clinical outcomes for people with haemophilia A (HA). However, non-clinical joint-related burden of moderate (MHA) and severe (SHA) HA has not been well characterised. AIM: To quantify the joint health-related humanistic and economic burden of MHA and SHA in Europe. METHODS: A retrospective analysis of the cross-sectional CHESS population studies using a patient-centric measure of joint health (problem joints, PJs: chronic joint pain and/or limited range of movement due to compromised joint integrity with or without persistent bleeding) was conducted. Descriptive statistics summarised health-related quality of life (HRQoL), work productivity/activity impairment and costs by number of PJs (0, 1 or ≥2) and HA severity. RESULTS: A total of 1171 patients were included from CHESS-II (n = 468) and CHESS-PAEDs (n = 703). In both studies, 41 and 59% of patients had MHA and SHA, respectively. Prevalence of ≥2 PJs was similar with MHA and SHA (CHESS-II: 23 and 26%; CHESS-PAEDs: 4 and 3%, respectively). HRQoL was worse with an increasing number of PJs (CHESS-II: .81 vs. .66 with 0 and ≥2 PJs, respectively, for MHA; .79 vs. .51 for SHA; CHESS-PAEDs: .64 vs. .26 and .72 vs. .14). Total costs increased with increasing PJs regardless of severity in CHESS-II (€2923 vs. €22,536 with 0 and ≥2 PJs, respectively, for MHA; €11,022 vs. €27,098 for SHA) and CHESS-PAEDs (€6222 vs. €11,043 for MHA; €4457 vs. €14,039 for SHA). CONCLUSION: Presence of PJs was associated with a substantial humanistic and economic burden on patients with MHA or SHA across the lifespan.