Clinical experience over decades and numerous retrospective and, recently, also prospective studies clearly demonstrate that prophylactic treatment, albeit much more expensive, is superior to on-demand treatment regardless of whether the outcome is number of joint- or life-threatening bleeds or arthropathy optimal prophylactic treatment should be started early (primary prophylaxis) but various opinions exist on the model. The optimal model should be to individualize prophylaxis taking into account the bleeding phenotype and the individual susceptibility to arthropathy and further develop monitoring by global coagulation assays and pharmacokinetic parameters. This review covers proof of concept of primary prophylaxis in children, comparisons between prophylaxis and on demand treatment, and future trends of prophylactic treatment of hemophilia. Pediatr Blood Cancer © 2012 Wiley Periodicals, Inc.
|Tidskrift||Pediatric Blood & Cancer|
|Status||Published - 2013|