title: Choice of factor VIII/IX regimen in adolescents and young adults with severe or moderately severe haemophilia. A French national observational study (ORTHem 15-25).
abstract: BACKGROUND
  The value and challenges of long-term prophylaxis (LTP) in adolescents and young adults need further characterisation.
  
  
  OBJECTIVE
  To determine the proportions of adolescents and young adults with severe or moderately severe haemophilia in France under LTP and treatment on demand (OD).
  
  
  METHODS
  Patients 15 to 25years old with haemophilia A or B, factor VIII/IX ≤2% and no current inhibitor could be included if they had been under factor VIII/IX treatment at least 12months and kept a treatment and bleeding diary.
  
  
  RESULTS
  LTP was administered to 169/212 patients (79.7%) and OD treatment to 40/212 patients (18.9%). The most frequent reasons for initiating LTP were joint bleeding, target joints and frequent bleeds; whereas OD treatment was most often selected on the basis of mild bleeding phenotype or because of constraints on LTP. The mean annual bleed rate (ABR) in the OD group (6.33) was higher than in the LTP group (3.07, p<0.001). Mean ABR did not differ significantly between age strata (15-18, >18-21 and >21-25years), but was significantly higher for patients with severe haemophilia (4.02) as compared to those with moderate haemophilia (1.97, p=0.002). No significant difference was observed in mean ABR for joint bleeds between the LTP and OD groups. Physician reported LTP compliance was good or excellent in 97.0% of patients.
  
  
  CONCLUSIONS
  LTP is the predominant factor VIII/IX treatment among adolescents and young adults with severe or moderately severe haemophilia in France. LTP was associated with low ABR and high compliance.
authors.affiliation: Haemophilia Treatment Centre, Louis Pradel Hospital, Bron, France. Electronic address: sandrine.meunier@chu-lyon.fr.
authors.forename: Sandrine
authors.initials: S
authors.lastname: Meunier
authors.affiliation: Centre for Haemophilia and Rare Congenital Bleeding Disorders, AP-HP University Hospitals Paris-Sud, Bicêtre Hospital, Le Kremlin-Bicêtre, France.
authors.forename: Roseline
authors.initials: R
authors.lastname: d'oiron
authors.affiliation: Haemophilia Treatment Centre, AP-HM La Timone Hospital and Inserm UMR S 1062, Aix Marseille University, Marseille, France.
authors.forename: Hervé
authors.initials: H
authors.lastname: Chambost
authors.affiliation: Baxalta France S.A.S., now part of Shire, Puteaux, France.
authors.forename: Edita
authors.initials: E
authors.lastname: Dolimier
authors.affiliation: Haemophilia Treatment Centre, Pontchaillou Hospital, CHU Rennes and Inserm IRSET U1085, Rennes University 1, Rennes, France.
authors.forename: Benoît
authors.initials: B
authors.lastname: Guillet
mesh_terms: D000293:Adolescent; D000328:Adult; D003029:Coagulants / Q000627:therapeutic use*; D005164:Factor IX / Q000627:therapeutic use*; D005169:Factor VIII / Q000627:therapeutic use*; D005260:Female; D005602:France / Q000453:epidemiology; D006395:Hemarthrosis / Q000209:etiology / Q000517:prevention & control; D006467:Hemophilia A / Q000150:complications* / Q000188:drug therapy* / Q000453:epidemiology; D006470:Hemorrhage / Q000209:etiology / Q000517:prevention & control*; D006801:Humans; D008297:Male; D012189:Retrospective Studies; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D003029:Coagulants; D005169:Factor VIII; D005164:Factor IX
keywords: Age groups; Factor IX; Factor VIII; Haemorrhage; On-demand treatment; Prophylaxis