Evaluation of the utility of the ISTH‐BAT in haemophilia carriers: a multinational study. (November 2016)

• Record Type: Journal Article
• Authors: James, P. D.; Mahlangu, J.; Bidlingmaier, C.; Mingot‐Castellano, M. E.; Chitlur, M.; Fogarty, P. F.; Cuker, A.; Mancuso, M. E.; Holme, P. A.; Grabell, J.; Satkunam, N.; Hopman, W. M.; Mathew, P.
• Other Names: Kulkarni Roshni investigator.; Iorio Alfonso investigator.; Gomez Keith investigator.; Kasthuri Raj investigator.; Kruse‐Jarres Rebecca investigator.; Ozelo Margareth investigator.; Shida Yasuaki investigator.; Kenet Gili investigator.
• Is Part Of: Haemophilia. Volume 22:Number 6(2016:Nov.)
• Page Start: 912
• Page End: 918
• Publication Date: 2016-11
• Subjects: bleeding -- haemophilia carriers -- ISTH‐BAT; Hemophilia -- Periodicals; 616.1572005
• DOI: 10.1111/hae.13089
• Languages: English
• ISSNs: 1351-8216

Abstract

Abstract : Introduction: 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. Aim: 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 r 2 = −0.36, P < 0.001). Conclusion: 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.