title: Pilot study of once-a-day prophylaxis for youth and young adults with severe haemophilia A.
abstract: BACKGROUND
  Standard prophylaxis has been shown to be an effective treatment for severe haemophilia A. According to pharmacokinetic principles, daily factor infusions of smaller doses can maintain similar trough factor VIII (FVIII) levels, and perhaps the same protection as standard prophylaxis.
  
  
  OBJECTIVE
  This multicentre study examined the feasibility of daily prophylaxis for youth and young adults with severe haemophilia A in Montreal and Toronto.
  
  
  METHODS
  Bleeding rates, joint status, quality of life and physical activity were monitored for 14 patients during this study. At baseline, subjects continued their regular treatment regimen and switched to daily prophylaxis after 4 months; nine had begun daily prophylaxis before enrolment. Additional visits occurred at 8 and 12 months which included a physical examination, inhibitor testing, HJHS and FISH assessments, the CHO-KLAT/Haemo-QoL-A and PDPAR. Treatment satisfaction was assessed using the Treatment Satisfaction Questionnaire for Medication ver.II and perceived difficulty questions at the end of study.
  
  
  CONCLUSIONS
  There were no significant changes in quality of life except for concerns with the demanding daily infusion schedule. The number of bleeds did not statistically differ from the initial 4 months of the study to the last 8 months. Monthly bleeding rates from the year prior to the study and during the intervention phase were not statistically different. It was also found that daily prophylaxis used 24% less FVIII compared to standard prophylaxis. Taking all of this into account, we have found that providing daily prophylaxis is feasible and that it is feasible to prospectively study daily prophylaxis in youth and young adults.
authors.affiliation: Department of Chemistry, University of Toronto, Toronto, ON, Canada.
authors.forename: V
authors.initials: V
authors.lastname: Crivianu-Gaita
authors.affiliation: Division of Haematology/Oncology, Department of Paediatrics, Université de Montréal, CHU Sainte-Justine, Montreal, QC, Canada.
authors.forename: G E
authors.initials: GE
authors.lastname: Rivard
authors.affiliation: Division of Haematology/Oncology, Department of Paediatrics and Child Health Evaluative Sciences, Research Institute, The Hospital for Sick Children, University of Toronto, Toronto, ON, Canada.
authors.forename: M
authors.initials: M
authors.lastname: Carcao
authors.affiliation: Division of Hematology and Oncology, Department of Medicine, St. Michael's Hospital, University of Toronto, Toronto, ON, Canada.
authors.forename: J
authors.initials: J
authors.lastname: Teitel
authors.affiliation: Hematology and Oncology Service, CHU Sainte-Justine, Department of Medicine, Université de Montréal, Montreal, QC, Canada.
authors.forename: J
authors.initials: J
authors.lastname: St-Louis
authors.affiliation: Department of Pediatrics, University of Toronto, Pediatric Comprehensive Care Hemophilia Program, Division of Hematology/Oncology, Hospital for Sick Children, Toronto, ON, Canada.
authors.forename: V
authors.initials: V
authors.lastname: Blanchette
authors.affiliation: Child Health Evaluative Sciences, The Hospital for Sick Children, The Dalla Lana School of Public Health, University of Toronto, Toronto, ON, Canada.
authors.forename: E
authors.initials: E
authors.lastname: Pullenayegum
authors.affiliation: Child Health Evaluative Sciences, The Hospital for Sick Children, The Dalla Lana School of Public Health, University of Toronto, Toronto, ON, Canada.
authors.forename: A
authors.initials: A
authors.lastname: Abad
authors.affiliation: Department of Pediatrics, Faculty of Medicine and the Institute of Health Policy Management & Evaluation, Dalla Lana School of Public Health, University of Toronto, Child Health Evaluative Sciences, Toronto, ON, Canada. brian.feldman@sickkids.ca.|Division of Rheumatology, The Hospital for Sick Children, Toronto, ON, Canada. brian.feldman@sickkids.ca.
authors.forename: B M
authors.initials: BM
authors.lastname: Feldman
mesh_terms: D000293:Adolescent; D000328:Adult; D002170:Canada; D018450:Disease Progression; D005169:Factor VIII / Q000627:therapeutic use*; D005240:Feasibility Studies; D005500:Follow-Up Studies; D006467:Hemophilia A / Q000150:complications / Q000188:drug therapy*; D006470:Hemorrhage / Q000209:etiology / Q000517:prevention & control*; D006801:Humans; D010865:Pilot Projects; D011788:Quality of Life; D011795:Surveys and Questionnaires; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D005169:Factor VIII
keywords: factor VIII; haemophilia A; joint bleeds; pilot study; prophylaxis; quality of life