New developments in the treatment of pediatric hemophilia and bleeding disorders.

PURPOSE OF REVIEW: Disorders of hemostasis such as hemophilia, von Willebrand disease (VWD), and other clotting protein deficiencies lead to significant morbidity in the pediatric population. Because of the limitations of current treatment options, novel therapies are being developed, many of which are reviewed here.

RECENT FINDINGS: Several new observations about the nature of clotting protein physiology have been made recently, creating novel perspectives on the treatment options. This review will mostly focus on the current therapy as well as new progress in hemophilia care (particularly strategies to prolong half-life of clotting factor replacements, the management of inhibitors, gene therapy, and novel therapeutic approaches), and briefly mention some progress in VWD and fibrinogen deficiency therapies.

SUMMARY: New therapeutic developments have the potential to dramatically decrease morbidity and improve the quality of life of children with bleeding disorders.