title: Factor VIII/factor IX prophylaxis for severe hemophilia.
abstract: Experience with clotting factor concentrate (CFC) replacement products over several decades has shown that regular replacement (prophylaxis) is the only way to prevent musculoskeletal damage in hemophilia and impact the natural history of hemophilia. Yet there is a lack of data on the optimal age to start such replacement therapy and the regimens to be used. While very early administration of high doses is certainly more effective in preventing bleeding, cost and compliance are major constraints all over the world. Starting prophylaxis with even lower doses comparable to that used in episodic therapies leads to major reduction in bleeding. Recognition of the clinical heterogeneity of hemophilia even among patients with a label of severe hemophilia in terms of their spontaneous bleeding has led to efforts aimed at individualizing CFC replacement, based on clinical responses or pharmacokinetic data of the CFC. The importance of long-term outcome assessment being combined with CFC replacement therapy cannot be overemphasized.
authors.affiliation: Department of Paediatrics; Division of Haematology/Oncology and Child Health Evaluative Sciences, Research Institute, Hospital for Sick Children, Toronto, Ontario, Canada. Electronic address: manuel.carcao@sickkids.ca.
authors.forename: Manuel
authors.initials: M
authors.lastname: Carcao
authors.affiliation: Department of Haematology and Centre for Stem Cell Research (a unit of inStem, Bengaluru), Christian Medical College, Vellore, India. Electronic address: aloks@cmcvellore.ac.in.
authors.forename: Alok
authors.initials: A
authors.lastname: Srivastava
mesh_terms: D005164:Factor IX / Q000191:economics / Q000627:therapeutic use*; D005169:Factor VIII / Q000191:economics / Q000627:therapeutic use*; D006467:Hemophilia A / Q000188:drug therapy* / Q000628:therapy; D006470:Hemorrhage / Q000517:prevention & control*; D006801:Humans; D055118:Medication Adherence; D017063:Outcome Assessment, Health Care; D010641:Phenotype
chemical_list: D005169:Factor VIII; D005164:Factor IX
keywords: Hemophilia; individualization; prophylaxis; tailoring