Long-term prophylaxis: what are our options and how to define success?

Abstract

Currently, we are at an enviable place in hemophilia treatment. Although full prophylaxis with standard half-life recombinant or plasma-derived factor concentrates has been definitively shown to be inadequate for full protection against bleeding and arthropathy, a number of novel therapies with improved hemostatic enhancement are clinically available or in promising clinical trials. In order to compare outcomes among a number of very efficacious therapies, it is necessary to have sensitive tools employed in long-term follow-up for several years for participants with no or minimal joint disease. The tool kit must be comprehensive, with outcomes of bleeding, factor level restoration or hemostatic capacity, joint structure, joint function, pain, quality of life, and patient satisfaction. This article reviews the history of prophylaxis, the promise of emerging therapies, and the sensitive tools used to assess long-term efficacy for joint structure and function.