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.

Cost of non-alcoholic steatohepatitis in Europe and the USA: The GAIN study.

BACKGROUND & AIMS: Non-alcoholic steatohepatitis (NASH) leads to cirrhosis and is associated with a substantial socioeconomic burden, which, coupled with rising prevalence, is a growing public health challenge. However, there are few real-world data available describing the impact of NASH. METHODS: The Global Assessment of the Impact of NASH (GAIN) study is a prevalence-based burden of illness study across Europe (France, Germany, Italy, Spain, and the UK) and the USA. Physicians provided demographic, clinical, and economic patient information via an online survey. In total, 3,754 patients found to have NASH on liver biopsy were stratified by fibrosis score and by biomarkers as either early or advanced fibrosis. Per-patient costs were estimated using national unit price data and extrapolated to the population level to calculate the economic burden. Of the patients, 767 (20%) provided information on indirect costs and health-related quality of life using the EuroQOL 5-D (EQ-5D; n = 749) and Chronic Liver Disease Questionnaire - Non-Alcoholic Fatty Liver Disease (CLDQ-NAFLD) (n = 723). RESULTS: Mean EQ-5D and CLDQ-NAFLD index scores were 0.75 and 4.9, respectively. For 2018, the mean total annual per patient cost of NASH was €2,763, €4,917, and €5,509 for direct medical, direct non-medical, and indirect costs, respectively. National per-patient cost was highest in the USA and lowest in France. Costs increased with fibrosis and decompensation, driven by hospitalisation and comorbidities. Indirect costs were driven by work loss. CONCLUSIONS: The GAIN study provides real-world data on the direct medical, direct non-medical, and indirect costs associated with NASH, including patient-reported outcomes in Europe and the USA, showing a substantial burden on health services and individuals. LAY SUMMARY: There has been little research into the socioeconomic burden associated with non-alcoholic steatohepatitis (NASH). The GAIN study provides real-world data on the direct medical, direct non-medical, and indirect costs associated with NASH, including patient-reported outcomes in five European countries (UK, France, Germany, Spain, and Italy) and the USA. Mean total annual per patient cost of NASH was estimated at €2,763, €4,917, and €5,509 for the direct medical, direct non-medical, and indirect cost categories, respectively.