Factor VIII prophylaxis effects outweigh other hemostasis contributors in predicting severe haemophilia A joint outcomes.

摘要: Introduction The Joint Outcome Study (JOS) demonstrated that previously untreated children with severe haemophilia A treated prophylactic factor VIII (FVIII) concentrate had superior joint outcomes at age 6 years compared to those episodically for bleeding. However, variation in outcome within each treatment arm was not well explained. Aim In this study, we sought better understand participants of the JOS. Methods We evaluated influence FVIII half-life, adherence, constitutional coagulant and anticoagulant proteins, global assays on (number bleeds, total number MRI score physical exam score). Logistic regression used evaluate association variables failure status MRI, defined as presence subchondral cyst, surface erosion or joint-space narrowing. Each parameter also correlated using Spearman correlations. Results Prophylaxis trough were found reduce risk univariate logistic analysis. When controlling arm, no longer significant, likely because high level covariation between these variables. consistent correlation any laboratory assay performed measured. Conclusion JOS, effect prescribed infusions overshadowed contribution coagulation proteins. (ClinicalTrials.gov number, NCT00207597).