Treatment costs and lost production were compared using Dutch pri

Treatment costs and lost production were compared using Dutch prices for the year

2010. Costs were translated to USD (1 euro = 1.326 USD). A total of 78 Dutch (intermediate-dose) and 50 Swedish (high-dose) patients were included and assessed during regular clinic visits. Patients were evaluated at a mean age of 24.5 years (range 14–37).The majority (90%) of patients had haemophilia A. Treatment and outcome according to prophylactic regimen are shown in Table 1. Overall, the prophylactic treatment regimens were very different: patients treated with the Dutch intermediate-dose regimen started prophylaxis later, and used a significantly lower dose throughout life. During evaluation, 78% of Dutch and 96% of Swedish patients were on full-time prophylaxis. Both cohorts showed normal physical activity levels (data not shown). Differences in outcome were small but statistically significant: patients treated with the intermediate-dose regimen had slightly higher HJHS scores (median 7.0 vs. 4.0 points out of 144) and reported slightly more bleeding (7–8 additional joint bleeds in 5 years) and more limitations in daily activities (median HAL scores of 93/100 vs. 99/100). These small differences in outcome did not result in a difference in quality of Midostaurin ic50 life or employment status. For the 5-year period, median total costs

per patient were 73% higher isothipendyl for high-dose prophylaxis. Clotting factor consumption accounted for >97% of costs. This study showed a statistically significant

but small improvement in outcome at age 24 after nearly doubling the annual prophylactic dose. This small incremental benefit was observed in all outcome parameters, except quality of life. This may reflect the limited clinical effects of one additional joint bleed per year, or the inability of the generic Euroqol questionnaire to pick up small differences. In addition, it must be noted that these joint bleeds were treated at a very early stage, usually requiring only a single infusion of FVIII/IX. From a life-long perspective, it is expected that differences in outcome between these two cohorts will increased in the next decades. However, we do not know the clinical and functional implications of such an increase. Is the difference attributable to dose difference only? One of the drivers of the slightly better outcome in the high-dose group may be the earlier start of prophylaxis, as was shown in both Swedish and Dutch patients [24, 25]. Multivariable regression analysis of these data suggested that the effect of dose was more important than the effect of early initiation of prophylaxis. For clinical practice, it will always be important to prevent bleeding, especially in the joints.

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