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Patients with hemophilia experience recurrent hemorrhages into soft tissues and joints, resulting in crippling and disability. Bleeding can be prevented by the prophylactic administration of clotting-factor concentrates, but the most cost-effective method of using these expensive biologics is unclear (NEJM JW Oncol Hematol Apr 17 2012).
To address this issue, European investigators examined outcomes and costs in patients with severe hemophilia who received one of two different prophylactic regimens of factor concentrate and were followed for 20 to 30 years: 78 Dutch patients received intermediate-dose prophylaxis (46 IU/kg/week) and 50 Swedish patients received high-dose prophylaxis (88 IU/kg/week).
During a 5-year period, patients who received the intermediate- versus high-dose regimen experienced more joint bleeds (median, 10.0 vs. 2.5; P<0.01) and greater loss of function (mean score, 9 vs. 4; P<0.01). However, the number of joints affected in the two groups was similar (median, 2 and 3, respectively), as were health-related quality-of-life scores. Patients in the high-dose groups used greater amounts of factor (median, 4000 vs. 2100 IU/kg/year; P<0.01) and had higher associated costs (mean per patient per 5 years, US$1,452,000 vs. US$867,000; P<0.01).
Fischer K et al. Intermediate-dose versus high-dose prophylaxis for severe hemophilia: Comparing outcome and costs since the 1970s. Blood 2013 Jun 18; [e-pub ahead of print]. (http://dx.doi.org/10.1182/blood-2012-12-470898)
Comment
Prophylaxis with clotting-factor concentrates can prevent permanent joint damage, increase school attendance, enhance work productivity, and improve quality of life. However, even though recombinant concentrates have been available for more than 15 years, they are extraordinarily expensive. Until alternative methods of providing protection against bleeding become available, the prophylactic regimen for each patient will be determined by trade-offs between effectiveness and cost tolerance.