Hemophilia lives: the impact of prophylaxis

In this issue of Blood, Plug and colleagues in the Netherlands use their ongoing, country-wide, epidemiologic hemophilia studies to evaluate the effect of the introduction of prophylaxis on patients' ability to work and complete an education, as compared with the general population. More than 721 patients were divided into 2 groups: patients from 16 to 31 years of age after prophylaxis was introduced, and those from 31 to 64 years of age before its introduction.

As shown through the findings of this group and others, prophylaxis decreases disabilities. Of the hemophilia patients tested, 36% versus 5% of those with severe hemophilia had occupational disability before versus after introduction of prophylaxis. The study demonstrates that this health-care innovation not only affects joint outcomes, but also allows patients to achieve a higher level of education and career, such as by holding academic or managerial positions.

Health-related quality of life tools designed specifically for hemophilia are evolving. Plug and colleagues used a well-known scale, Short-Form 36, to evaluate their patient population. Patients with mild and moderate hemophilia were similar to the general population in both groups. In all 8 categories of measurement, there was marked improvement once prophylactic treatment was introduced.

These data strongly suggest that social integration into society is correlated, in the main, with the disabling sequelae of this disease. What is not addressed is the impact of transfusion-transmitted diseases on the quality of life, disability, and educational and/or work level. By using only questionnaires, one cannot evaluate the impact of the family structure, nor of socioeconomics, on issues such as educational expectations, academic achievements, and occupation. The authors do not comment on aspects of life not covered by the instrument they used. How did the change in therapy affect self-esteem, or patients' satisfaction with their life, family, and work?

The costs of prophylaxis are significantly higher than those of on-demand therapy.¹  The findings of Plug and colleagues, obtained in the Holland health-care system in which these costs are covered, may not be easily extrapolated to the United States, which has a pluralistic health-care reimbursement system and high costs of higher education. The message delivered by the authors is clear, nevertheless: their findings should inspire expansion of prophylaxis. The quality-of-life data they have presented will encourage the development of instruments to better understand the outcomes of the health-care delivery system, but translating them into meaningful financial paybacks to society will require further work.