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November 16th, 2004 WFH Joint Symposium Review – Natural Proteins in the Treatment of Blood Disorders: Return to Reason
The objectives of the study were to follow haemophilia A patients in regular treatment who had been receiving pdFVIII and were switched to rFVIII, or vice versa, in a unique opportunity to evaluate efficacy under conditions where inter-individual patient response is minimised. The study period is 6 months on one preparation before crossover to 6 months on the other. An electronic Case Report Form was used to capture patient demographic and treatment data, type of product and treatment regimen, either on demand, prophylaxis, immune tolerance induction (ITI) or surgery. To minimise the impact of inhibitor development, only patients are enrolled who have had at least 150 exposure days (ED) of treatment. The principal outcome variables were mean bleeding frequency and mean duration of days per bleed. To date, about 200 patients have been included of which 126 (121 severe, 4 moderate, 1 mild) have completed the treatments and could be evaluated. Mean age of the patients is 23.84 (2.71-62.77) years. The cumulative treatments so far have given 893 months on pdFVIII and 909 months on rFVIII with comparable mean treatment periods per patient of around 220 ED. Two statistical methods of analysis, Student’s paired t-test and the non parametric Wilcoxon signed rank test, were used. The results were confirmed using Poisson regression analysis for the covariate, type of product. The number of bleeds per year and the mean number of bleeding days per year were similar in both arms of the study, indicating there was no significant difference between the types of product. The most significant covariate is prophylactic treatment. This had a dramatic impact on the use and efficacy of both types of product. This interim analysis thus suggests that plasmatic and recombinant FVIII have similar efficacies but that treatment modality strongly influences study outcome. © Octapharma AG, 2004 |
