Clinical, humanistic, and economic burden of severe haemophilia B in adults receiving factor IX prophylaxis: findings from the CHESS II real-world burden of illness study in Europe

Abstract

Background Real-world studies of the burden of severe haemophilia B in the context of recent therapeutic advances such as extended half-life (EHL) factor IX (FIX) products are limited. We analysed data from the recent CHESS II study to better understand the clinical, humanistic, and economic burden of severe haemophilia B in Europe. Data from male adults with severe haemophilia B receiving prophylaxis were analysed from the retrospective cross-sectional CHESS II study conducted in Germany, France, Italy, Spain and the United Kingdom. Inhibitors were exclusionary. Patients and physicians completed questionnaires on bleeding, joint status, quality of life, and haemophilia-related direct and indirect costs (2019–2020). All outcomes were summarised using descriptive statistics. Results A total of 75 CHESS II patients were eligible and included; 40 patients (53%) provided self-reported outcomes. Mean age was 36.2 years. Approximately half the patients were receiving EHL versus standard half-life (SHL) prophylaxis (44% vs 56%). Most patients reported mild or moderate chronic pain (76%) and had ≥ 2 bleeding events per year (70%), with a mean annualised bleed rate of 2.4. Mean annual total haemophilia-related direct medical cost per patient was €235,723, driven by FIX costs (€232,328 overall, n = 40; €186,528 for SHL, €290,620 for EHL). Mean annual indirect costs (€8,973) were driven by early retirement or work stoppage due to haemophilia. Mean quality of life (EQ-5D) score was 0.67. Conclusions These data document a substantial, persistent real-world burden of severe haemophilia B in Europe. Unmet needs persist for these patients, their caregivers, and society.

Association of factor expression levels with annual bleeding rate in people with haemophilia B

“…This cross‐sectional study utilized the data and infrastructure of the ‘Cost of Haemophilia in Europe: a Socioeconomic Survey’ (CHESS EU I‐II) and the ‘Cost of Severe Haemophilia across the US: a Socioeconomic Survey’ (CHESS US/US+) bottom‐up burden of illness studies. The design, methods and primary findings from CHESS EU I‐II and CHESS US/US+ have been reported previously 15,16 . The CHESS EU I and II studies included European males aged ≥18 years with haemophilia A or B from the five largest European countries (France, Germany, Italy, Spain, and the United Kingdom; EU5), with CHESS EU II adding physicians and patients from Denmark, Romania, and The Netherlands.…”

“…The design, methods and primary findings from CHESS EU I‐II and CHESS US/US+ have been reported previously. 15 , 16 The CHESS EU I and II studies included European males aged ≥18 years with haemophilia A or B from the five largest European countries (France, Germany, Italy, Spain, and the United Kingdom; EU5), with CHESS EU II adding physicians and patients from Denmark, Romania, and The Netherlands. CHESS EU I enrolment was limited to people with severe haemophilia A or B; CHESS EU II included people with mild or moderate disease.…”

Recombinant factor IX Fc prophylaxis reduces pain and increases levels of physical activity, with sustained, long-term improvements in patients with hemophilia B: post hoc analysis of phase III trials using patient-reported outcomes

“…[17][18][19][20][21][22][23] Haem-A-QoL improvements in adults and adolescents were maintained over time for the total score and the domains sports and leisure, physical health, feeling, and view of self; confirming both the results from the initial B-LONG study, 21 and the interim analysis of the BYOND study. 22 There is a paucity of data on the impact of prophylaxis with EHL FIX products on HRQoL in patients with hemophilia B, 24 making it difficult to ratify these results against previous studies. As the emphasis shifts away from focusing solely on bleed prevention toward a more holistic approach for treating hemophilia B, 14 the impact of successful treatment on HRQoL will be revealed.…”

Prevalence and perceptions of pain in people with haemophilia: A UK study

“…Up to 70% of PWH report living with chronic pain, 5–9 estimates of prevalence vary depending on the definitions used and the population studied. Most current data for pain in PwH in the UK is amalgamated with multi‐country results 10,11 . To date, no published pain study has focused only on PWH in the UK.…”

“…Most current data for pain in PwH in the UK is amalgamated with multi-country results. 10,11 To date, no published pain study has focused only on PWH in the UK.…”