Source
Mountain States Regional Hemophilia & Thrombosis Center, University of Colorado Health Sciences Center and The Children's Hospital, Denver, Colorado, USA. marilyn.manco-johnson@uchsc.edu
Abstract
PURPOSE OF REVIEW:
Progress in hemophilia management over the past year has focused on improving understanding of the most common complications of genetic bleeding disorders and building upon available therapeutic strategies.
RECENT FINDINGS:
Research continued to link factor VIII structure with immune recognition and inhibitor formation. Clinical regimens of immune tolerance induction confirmed and expanded basic understanding. Barriers to optimal prevention using prophylaxis were explored allowing future refinements to address unmet needs. Outcome tools to assess joint health and overall quality of life were developed and validated. The inclusion of standardized instruments in assessment of outcome will allow meaningful comparison of available therapies. Use and complications of central venous access devices (CVAD), needed to deliver aggressive infusion regimens, were exhaustively reviewed. Finally, continued progress was achieved in development of improved vectors for future gene therapy of the hemophilias.
SUMMARY:
A general theme of recent progress in hemophilia management is harmonization in definitions and assessments of complications and outcomes, facilitating more rigorous and ultimately more useful interpretation of laboratory and clinical research.