title: European retrospective study of real-life haemophilia treatment.
abstract: BACKGROUND
  Haemophilia treatment varies significantly between individuals, countries and regions and details of bleed rates, factor consumption and injection frequency are often not available.
  
  
  OBJECTIVE
  To provide an overview of the FVIII/FIX treatment practice and outcome for patients with haemophilia A (HA) or haemophilia B (HB) across Europe.
  
  
  METHODS
  Non-interventional, 12-month retrospective study where anonymized data were retrieved from haemophilia centres/registers in Belgium, France, Germany, Italy, Spain, Sweden and the United Kingdom. Male patients (all ages) receiving coagulation factor treatment 24 months prior to the study, with basal FVIII/FIX levels ≤5 IU dL-1, without inhibitors, were included. Data were summarized descriptively.
  
  
  RESULTS
  In total, 1346 patients with HA and 312 with HB were included in the analysis; 75% and 57% had severe disease (FVIII/FIX < 1 IU dL-1) respectively. Prophylaxis was most common for severe haemophilia, especially for children, whereas on-demand treatment was more common for moderate haemophilia in most countries. The mean (SD) prescribed prophylactic treatment ranged from 67.9 (30.4) to 108.4 (78.1) (HA) and 32.3 (10.2) to 97.7 (32.1) (HB) IU kg-1per week, across countries. Most patients on prophylaxis were treated ≥3 times/week (HA) or two times/week (HB). The median annual bleeding rate (ABR) for patients on prophylaxis ranged from 1.0 to 4.0 for severe HA, and from 1.0 to 6.0 for severe HB, while those with moderate haemophilia generally had slightly higher ABRs. Median ABRs for on-demand-treated severe HA ranged from 4.5 to 18.0, and for HB, 1.5 to 14.0.
  
  
  CONCLUSIONS
  Treatment practice varied greatly between centres and countries and patients treated on-demand and prophylactically both experienced bleeds, emphasizing the need for further optimization of care.
authors.affiliation: Clinical Coagulation Research Unit, Lund University, Malmö, Sweden.
authors.forename: E
authors.initials: E
authors.lastname: Berntorp
authors.affiliation: St Thomas' Hospital Haemophilia Centre, St Thomas' Hospital, London, UK.
authors.forename: G
authors.initials: G
authors.lastname: Dolan
authors.affiliation: Department of Haematology, Manchester Royal Infirmary, Manchester, UK.
authors.forename: C
authors.initials: C
authors.lastname: Hay
authors.affiliation: Center for Bleeding Disorders, Careggi University Hospital, Florence, Italy.
authors.forename: S
authors.initials: S
authors.lastname: Linari
authors.affiliation: Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, IRCCS Ca' Granda Foundation, Maggiore Hospital Policlinico, Milan, Italy.
authors.forename: E
authors.initials: E
authors.lastname: Santagostino
authors.affiliation: Department of Cell Therapy and Hematology, Hemophilia and Thrombosis Center, San Bortolo Hospital, Vicenza, Italy.
authors.forename: A
authors.initials: A
authors.lastname: Tosetto
authors.affiliation: Center for Bleeding Disorders, Careggi University Hospital, Florence, Italy.|Department of Cell Therapy and Hematology, Hemophilia and Thrombosis Center, San Bortolo Hospital, Vicenza, Italy.
authors.forename: G
authors.initials: G
authors.lastname: Castaman
authors.affiliation: Hospital Universitario La Paz, Thrombosis and Haemostasis Unit, Madrid, Spain.
authors.forename: M T
authors.initials: MT
authors.lastname: Álvarez-Román
authors.affiliation: Unidad de Hemofilia, Hospital Vall d'Hebrón, Barcelona, Spain.
authors.forename: R
authors.initials: R
authors.lastname: Parra Lopez
authors.affiliation: Institut für Experimentelle Hämatologie und Transfusionsmedizin, Universitätsklinik Bonn, Bonn, Germany.
authors.forename: J
authors.initials: J
authors.lastname: Oldenburg
authors.affiliation: Institut für Experimentelle Hämatologie und Transfusionsmedizin, Universitätsklinik Bonn, Bonn, Germany.
authors.forename: T
authors.initials: T
authors.lastname: Albert
authors.affiliation: Center of Haemostasis, Leipzig, Germany.
authors.forename: U
authors.initials: U
authors.lastname: Scholz
authors.affiliation: Coagulation Unit, Hematology Centre Karolinska, Karolinska University Hospital, Stockholm, Sweden.|Department of Medicine, Karolinska Institutet, Solna, Sweden.
authors.forename: M
authors.initials: M
authors.lastname: Holmström
authors.affiliation: Hôpital Saint Eloi, Laboratoire d'hématologie, Montpellier, France.
authors.forename: J-F
authors.initials: JF
authors.lastname: Schved
authors.affiliation: Centre Régional de Traitement de l'Hémophilie CHU, Nantes, France.
authors.forename: M
authors.initials: M
authors.lastname: Trossaërt
authors.affiliation: Haemostasis and Thrombosis Unit, Haemophilia Clinic St-Luc University Hospital, Brussels, Belgium.
authors.forename: C
authors.initials: C
authors.lastname: Hermans
authors.affiliation: Haemostasis and Thrombosis Unit, Haemophilia Clinic St-Luc University Hospital, Brussels, Belgium.|Department of Hematology, Medical School of Zagreb, University Hospital Center Zagreb, Zagreb, Croatia.
authors.forename: A
authors.initials: A
authors.lastname: Boban
authors.affiliation: Sobi, Stockholm, Sweden.|University of Edinburgh, Edinburgh, UK.
authors.forename: C
authors.initials: C
authors.lastname: Ludlam
authors.affiliation: Sobi, Stockholm, Sweden.|Copenhagen University, Copenhagen, Denmark.
authors.forename: S
authors.initials: S
authors.lastname: Lethagen
mesh_terms: D000328:Adult; D005060:Europe; D006467:Hemophilia A / Q000628:therapy*; D006801:Humans; D008297:Male; D012189:Retrospective Studies
keywords: factor IX; factor VIII; haemophilia A; haemophilia B; retrospective study; treatment