title: A longitudinal study of orthopaedic outcomes for severe factor-VIII-deficient haemophiliacs. The Orthopaedic Outcome Study Group.
abstract: OBJECTIVE
  Arthropathy is the major cause of morbidity in haemophilia. In an attempt to establish optimal therapeutic regimens for persons with haemophilia, we hypothesized that no direct relationship exists between increasing factor dosage and orthopaedic outcomes over time.
  
  
  METHODS
  A longitudinal uncontrolled 6 year study was carried out. Ankles, knees and elbows of patients were studied using physical and X-ray examination scores. Bleeding episodes and treatment regimens were determined. The amount of time lost from work and school, as well as days in hospital, was also monitored.
  
  
  METHODS
  Twenty-one international haemophilia centres were used to accrue and follow patients over the 6 year period.
  
  
  METHODS
  Severe (< 1%) factor VIII deficient patients under the age of 25 without inhibitors were recruited into the study.
  
  
  METHODS
  The status of the six major joints of these patients were measured annually for mobility. X-ray evaluation of these joints was carried out at the beginning and end of the study. Using a World Federation of Haemophilia joint and X-ray score, stability, progression or regression of arthropathy was evaluated.
  
  
  RESULTS
  Physical and X-ray examination scores increased significantly with age, and the number of joint bleeds were significant in determining the delta score. Approximately 10% of patients entered with all six joints normal. Of these, 50% remained so. Year-long prophylaxis significantly reduced the rate at which joints deteriorate both on physical (P = 0.02) and X-ray examination (P = < 0.001). Patients on prophylaxis had significantly fewer days lost from work or school, as well as fewer days spent in hospital (P = < 0.01).
  
  
  CONCLUSIONS
  Higher doses of factor per se do not necessarily produce improved orthopaedic outcomes. However, full time prophylaxis is likely to produce the best orthopaedic outcome. The most critical factor for a good orthopaedic outcome is the reduction of joint bleeds.
authors.affiliation: Mount Sinai Medical Center, Department of Hematology, New York.
authors.forename: L M
authors.initials: LM
authors.lastname: Aledort
authors.forename: R H
authors.initials: RH
authors.lastname: Haschmeyer
authors.forename: H
authors.initials: H
authors.lastname: Pettersson
mesh_terms: D005169:Factor VIII / Q000627:therapeutic use*; D006395:Hemarthrosis / Q000000981:diagnostic imaging / Q000209:etiology / Q000517:prevention & control*; D006467:Hemophilia A / Q000150:complications / Q000188:drug therapy*; D006801:Humans; D016014:Linear Models; D008297:Male; D010808:Physical Examination; D011446:Prospective Studies; D011859:Radiography; D016896:Treatment Outcome
chemical_list: D005169:Factor VIII