Exploring the Relationship Between Prophylactic Infusion Frequency and Patient Utility for People With Haemophilia in Europe

OBJECTIVES: Haemophilia is an inherited, lifelong bleeding disorder characterised by prolonged traumatic or spontaneous bleeding due to a lack of clotting factor. The objective of the study is to explore how treatment convenience, measured by frequency of prophylactic factor therapy infusions, impacts patients’ health related quality of life (HRQoL).

METHODS: The study used cross-sectional real-world data from the ‘Cost of Haemophilia in Europe: a Socioeconomic Survey’ (CHESS II), a retrospective study of male adults (≥18 years old) in eight European countries. Only participants with severe haemophilia on prophylaxis with factor concentrates and no inhibitors were included in the analytic sample. Treatment convenience was determined by frequency of infusions per week (one vs. two or more infusions/week) and HRQoL was patient reported by means of EQ-5D-5L, later mapped to EQ-5D-3L utility scores. The relationship between treatment convenience and HRQoL was explored descriptively and by means of multivariate regression analysis (Tobit model) and Propensity score matching (PMS). Control variables were age, Body Mass Index, Annualised Bleed Rate, joint morbidity, comorbidities, haemophilia type and education.

RESULTS:: Analytic sample was 241 severe haemophilia patients, mean age was 35 and 83% (N=199) were receiving two or more infusions/week. Average EQ-5D-3L utility was larger for those receiving one infusion/week (0.71) vs those receiving two or more (0.67). Regression estimates showed a negative relationship between being on two or more infusions (vs one infusion/week) and EQ-5D-3L (-0.0596; p<0.05). PMS results showed that the average EQ-5D-3L if all participants were to receive one infusion/week would be 0.107 (p<0.001) higher than receiving two or more infusions.

CONCLUSIONS: Results show that having two or more infusion/week is negatively associated to HRQoL. This suggests that therapies that do not compromise efficacy and improve treatment convenience by reducing the number of prophylactic factor therapy infusions, would improve HRQoL of patients with haemophilia.