A modeling approach to evaluate long-term outcome of prophylactic and on demand treatment strategies for severe XXXX .

@entity1 @entity548 @entity447
BACKGROUND: Severe hemophilia requires life-long treatment with expensive clotting factor concentrates; studies comparing effects of different therapeutic strategies over decades are very difficult to perform. A simulation model was developed to evaluate the long-term outcome of on demand, prophylactic and mixed treatment strategies for @entity1 with severe @entity447 . DESIGN AND METHODS: A computer model was developed based on individual @entity1 ' data from a Dutch cohort study in which intermediate dose prophylaxis was used and a French cohort study in which on demand treatment was used, and multivariate regression analyses. This model simulated individual @entity1 ' life expectancy, onset of @entity548 , life-time joint @entity548 , radiological outcome and concentrate use according to the different treatment strategies. RESULTS: According to the model, life-time on demand treatment would result in an average of 1,494 joint @entity548 during the hemophiliac's life, and consumption of 4.9 million IU of factor VIII concentrate. In contrast, life-time intermediate dose prophylaxis resulted in a mean of 357 joint @entity548 and factor consumption of 8.3 million IU. A multiple switch strategy (between prophylactic and on demand treatment based on @entity548 pattern) resulted in a mean number of 395 joint @entity548 and factor consumption of 6.6 million IU. The estimated proportion of @entity1 with Pettersson scores over 28 points was 32% for both the prophylactic and the multiple switching strategies, compared to 76% for continuous on demand treatment. CONCLUSIONS: The present model allows evaluation of the impact of various treatment strategies on @entity1 ' joint @entity548 and clotting factor consumption. It may be expanded with additional data to allow more precise estimates and include economic evaluations of treatment strategies.
