Abstract
Background - Hemophilia, a rare bleeding disorder, raises significant pharmacoeconomic challenges, particularly with the emergence of innovative therapies. This study evaluates clinical and patient-reported outcomes and expenditures in hemophilia patients focusing on those who underwent therapeutic switches between 2017 and 2023.
Materials and methods - This retrospective study included severe hemophilia patients. Expenditure was analyzed based on clotting factor consumption, while clinical well-being was assessed using ABR, NRS for pain, joint health scores (HJHS, HEAD-US) and trough plasma levels. Sub-analysis was conducted by applying different therapeutic groups. Statistical significance of differences between paired measures (pre [T0] vs post [T1]) was examined using the Wilcoxon Sum Rank test.
Results - Seventy patients were included. Total expenditure was € 12,947,580.44 in 2017 and € 12,967,576.92 in 2023. Among 45 patients who switched therapy, the median number of infusions/year significantly decreased from 156 to 91 (p<0.001), ABR from 1 to 0 (p<0.001) and NRS from 3,5 to 0 (p<0.001). Joint health did not show improvement with a slight but not statistically significant increase (HJHS from 10 to 15; HEAD-US from 7 to 8). Median trough increased from 3 UI/mL to 5 UI/mL (p=0.181). Patients were grouped for analysis based on homogeneous treatment regimens.
Discussion - This study is the first effort to conduct comprehensive analysis of the impact of therapeutic choices on well-being and cost of care in hemophilia Center. The study underscores the need for value-based approach to hemophilia care, integrating expenditure and clinical outcomes. Adopting validated economic weight outcome in future pharmacoeconomic evaluations will be essential for decision making.
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