title: New developments in the treatment of pediatric hemophilia and bleeding disorders.
abstract: OBJECTIVE
  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.
  
  
  RESULTS
  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.
  
  
  CONCLUSIONS
  New therapeutic developments have the potential to dramatically decrease morbidity and improve the quality of life of children with bleeding disorders.
authors.affiliation: Department of Pediatrics, Section of Hematology/Oncology/Bone Marrow Transplantation, Children's Hospital Colorado, University of Colorado - Anschutz Medical Campus, Aurora, Colorado 80045, USA.
authors.forename: Brian R
authors.initials: BR
authors.lastname: Branchford
authors.forename: Paul E
authors.initials: PE
authors.lastname: Monahan
authors.forename: Jorge
authors.initials: J
authors.lastname: Di Paola
mesh_terms: D025861:Blood Coagulation Disorders, Inherited / Q000628:therapy*; D002648:Child; D003029:Coagulants / Q000627:therapeutic use; D005164:Factor IX / Q000627:therapeutic use; D005169:Factor VIII / Q000627:therapeutic use; D015316:Genetic Therapy / Q000379:methods; D006467:Hemophilia A / Q000628:therapy; D006474:Hemorrhagic Disorders / Q000628:therapy*; D006801:Humans; D008297:Male
chemical_list: D003029:Coagulants; D005169:Factor VIII; D005164:Factor IX