title: Evaluation of the utility of the ISTH-BAT in haemophilia carriers: a multinational study.
abstract: BACKGROUND
  There has been increasing recognition in recent years that female carriers of haemophilia manifest abnormal bleeding; however, data on the use of bleeding assessment tools in this population are lacking.
  
  
  OBJECTIVE
  Our objective was to validate the ISTH-BAT in haemophilia carriers to describe bleeding symptoms and allow for comparisons with factor levels and other patient groups.
  
  
  METHODS
  This was a prospective, observational, cross-sectional study performed by members of Global Emerging HEmostasis Panel (GEHEP). Unselected consecutive haemophilia carriers were recruited and a CRF and the ISTH-BAT were completed by study personnel.
  
  
  RESULTS
  A total of 168 haemophilia carriers were enrolled: 155 haemophilia A and 13 haemophilia B. The mean age was 40 years (range: 20-82). Carriers had higher mean bleeding scores (BS) compared with age-matched controls (n = 46; 5.7 vs. 1.43; P < 0.0001) and Type 3 VWD OC (n = 32; 3.0; P = 0.009), but lower BS compared with women with Type 1 VWD (n = 83; 8.7; P < 0.0001). Fifteen carriers reported haemarthrosis, and of those six had normal FVIII/FIX levels. There was a significant but weak negative correlation between BS and factor level (Spearman's r2= -0.36, P < 0.001).
  
  
  CONCLUSIONS
  Our results show that haemophilia carriers experience abnormal bleeding, including haemarthrosis. Overall, BS in women with Type 1 VWD > haemophilia carriers > Type 3 VWD OC > controls. Understanding the performance of the ISTH-BAT in this population is a critical step in future research aimed at investigating the underlying pathophysiology of abnormal bleeding, with the ultimate goal of optimizing treatment.
authors.affiliation: Queen's University, Kingston, ON, Canada.
authors.forename: P D
authors.initials: PD
authors.lastname: James
authors.affiliation: Faculty of Health Sciences, University of the Witwatersrand, Johannesburg, South Africa.
authors.forename: J
authors.initials: J
authors.lastname: Mahlangu
authors.affiliation: Dr. von Hauner's Children's University Hospital, Munich, Germany.
authors.forename: C
authors.initials: C
authors.lastname: Bidlingmaier
authors.affiliation: Regional University Hospital of Malaga, Malaga, Spain.
authors.forename: M E
authors.initials: ME
authors.lastname: Mingot-Castellano
authors.affiliation: Children's Hospital of Michigan, Detroit, MI, USA.
authors.forename: M
authors.initials: M
authors.lastname: Chitlur
authors.affiliation: Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
authors.forename: P F
authors.initials: PF
authors.lastname: Fogarty
authors.affiliation: Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
authors.forename: A
authors.initials: A
authors.lastname: Cuker
authors.affiliation: Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy.
authors.forename: M E
authors.initials: ME
authors.lastname: Mancuso
authors.affiliation: Department of Hematology and Institute of Clinical Medicine, University Hospital, University of Oslo, Oslo, Norway.
authors.forename: P A
authors.initials: PA
authors.lastname: Holme
authors.affiliation: Queen's University, Kingston, ON, Canada.
authors.forename: J
authors.initials: J
authors.lastname: Grabell
authors.affiliation: Queen's University, Kingston, ON, Canada.
authors.forename: N
authors.initials: N
authors.lastname: Satkunam
authors.affiliation: Queen's University, Kingston, ON, Canada.
authors.forename: W M
authors.initials: WM
authors.lastname: Hopman
authors.affiliation: Bayer Health Care, Whippany, NJ, USA.|University of New Mexico, Albuquerque, NM, USA.
authors.forename: P
authors.initials: P
authors.lastname: Mathew
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D003430:Cross-Sectional Studies; D005260:Female; D006467:Hemophilia A; D006470:Hemorrhage / Q000175:diagnosis*; D006801:Humans; D008875:Middle Aged; D011446:Prospective Studies; D055815:Young Adult
keywords: ISTH-BAT; bleeding; haemophilia carriers