Cost of care and quality of life for patients with hemophilia complicated by inhibitors: the COCIS Study Group

Abstract

Inhibitors in patients with hemophilia are a rare complication of a rare disease causing pain and disability in patients and impairment to the quality of their lives. Recent advances in treatment have brought improvements, but they have done so by absorbing larger amounts of financial resources. This study involved 52 Italian patients with hemophilia with high-responding inhibitors who were longitudinally observed for 18 months to evaluate concomitantly cost of care and quality of life. Overall, 0.6 bleeding episodes per patient per month were recorded. This frequency of events was lower than that reported in other cohorts of patients with hemophilia who were not taking inhibitors. The average monthly cost of care was, in euros, €18 000 (US $18 000) per patient, mainly because of treatment products. Recombinant activated factor VII, mostly used for orthopedic surgery, represented 50% of the expenses. Quality of life, measured through validated questionnaires, was similar to that of patients with severe hemophilia without inhibitors. In particular, physical quality of life was similar to that in patients with diabetes and on dialysis, whereas mental quality of life was comparable to that in the general population. This study shows that hemophilia complicated by inhibitors, a prototype of rare disease, requires high amounts of resources for management that provides a satisfactory quality of life. (Blood. 2003;

Anti-Inhibitor Coagulant Complex Prophylaxis in Hemophilia with Inhibitors

“…5 Consequently, patients with inhibitors are at increased risk for bleeding that is difficult to control. 6 Poorly controlled hemarthroses result in the early onset of chronic joint disease and physical disability, which can substantially impair the quality of life. 7 Prophylaxis, the routine scheduled replacement of factor VIII, is standard care for patients who have severe hemophilia A without inhibitors, because of its ability to prevent bleeding.…”

Longitudinal Changes in Health-Related Quality of Life for Chronic Diseases: An Example in Hemophilia A

“…However, none has attempted to describe the effects of changes in the frequency of symptoms or clinical status on HrQoL. [3][4][5] Over 18 months, Gringeri et al 3 found that HrQoL remained stable in a group of 41 hemophilia A and B patients with inhibitors when measured at 6-month intervals; they therefore reported only baseline HrQoL results. Similarly, over a 5-year period with annual follow-up, Lindvall et al 4 found no statistically significant changes in HrQoL among a group of ten patients with severe hemophilia.…”

Back to the future: a recent history of haemophilia treatment

“…The first and main goal is to maintain the high levels of treatment that may be jeopardized even in highincome countries by the increase in the costs of the welfare state. Treatment of the haemophiliacs, particularly of those with inhibitors [53], entails usually the highest per capita cost in these countries. On the other hand, in any country the costs of haemophilia are truly a tiny part of the whole budget for health care, and the cost effectiveness of haemophilia care is well proven.…”