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Study 2 Citation:
Assessing
the Effectiveness and Cost-Effectiveness of Prophylaxis Against Bleeding in
Patients With Severe Haemophilia and severe von Willebrand's Disease
Journal
Authors
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Abstract:
This study looked at the importance of preventing joint bleeds and their relationship with joint arthropathy later in life. The ability to keep this arthritis to a minimum, and their consequences, were the focus of this study.
Both hemophilia and von Willebrand’s disease are characterized by a deficiency in clotting factor VIII (hemophilia A), clotting factor IX (hemophilia B), or von Willebrand factor. Severe hemophilia, defined as <1 u/dL –1, is typically treated with on-demand or prophylactically-infused clotting factor. It has been found that as long as clotting factor levels are kept above 1 u/dL –1, permanent injury to the joints can be avoided. Prophylaxis has been shown to be more effective at doing this, and so has become the treatment regimen of choice. However, price is an issue, and has kept prophylactic treatment under debate for some time. This study looks at the clinical effectiveness of prophylaxis, as well as its cost-effectiveness.
Key words:
Prophylaxis, Arthropathy Willebrand’s disease
The authors' intent was to determine the effectiveness and cost-efficiency of clotting factors used prophylactically against bleeding in severe hemophilia. By retrospectively analyzing records of 179 patients, factors such as type and amount of treatment administered, the medical reason for treatment, the day and time, and the number and location of bleeds were all investigated. Clotting factor usage was grouped according to the reason it was given (demand vs prophylaxis). Participants were tracked over a 16 year period by comparing bleeding patterns for both treatment on-demand and those receiving prophylaxis. Cost effectiveness was then calculated by looking at costs per bleed avoided.
Results:
The results were conclusive: Bleeding frequency was reduced within one year in 41 of 47 patients who switched from treatment on demand to prophylactic treatment with clotting factor (the median annual number of bleeds decreased from 23.5 in 1980 to 14 in 1995. Although prophylactic treatment resulted in fewer bleeds, it was substantially more costly than treatment on demand (approximately $875 per averted bleed). The authors also noted that this figure was highly sensitive to certain variables, such as variation in the unit price of clotting factor. More detailed proof of cost-effectiveness may require the use of modeling techniques.
Summary of Study 2:
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Prophylaxis can reduce bleeding frequency, but it requires more clotting factor than treatment on demand, resulting in higher costs. Although avoiding bleeding could lead to a future reduction in health care costs the decision maker must evaluate whether the expected benefits justify the immediate additional treatment expenses incurred with prophylactic treatment. The authors noted that a complete cost-effectiveness analysis would include data that were unavailable in this retrospective study. Most importantly, a model will be required in the future that will link information relating to joint bleeds with the development of arthropathy. Until this relationship is better understood and quantified, it will be difficult to produce an accurate estimate of long-term cost-effectiveness. |