Bleeding symptoms at disease presentation and prediction of ensuing bleeding in inherited FVII deficiency^{[ * ]}

Summary

Individuals with inherited factor VII (FVII) deficiency display bleeding phenotypes ranging from mild to severe, with 30% of patients having always been asymptomatic (non-bleeding). In 626 FVII-deficient individuals, by analysing data from the International Factor VII (IF7) Registry and the Seven Treatment Evaluation Registry (STER), we determined whether bleeding type at disease presentation and FVII coagulant activity (FVIIc) predict ensuing bleeds. At disease presentation/diagnosis, 272 (43.5%) individuals were non-bleeding, 277 (44.2%) had minor bleeds, and 77 (12.3%) had major bleeds. During a median nine-year index period (IP) observation, 87.9% of non-bleeding individuals at presentation remained asymptomatic, 75.1% of minor-bleeders had new minor bleeds, and 83.1% of major-bleeders experienced new major bleeds. After adjusting for FVIIc levels and other clinical and demographic variables, the relative risk (RR) for ensuing bleedings during the IP was 6.02 (p <0.001) and 5.87 (p <0.001) in individuals presenting with major and minor bleeds, respectively. Conversely, compared to non-bleeding individuals, a 10.95 (p = 0.001) and 28.21 (p <0.001) RR for major bleedings during the IP was found in those with minor and with major bleeds at presentation, respectively. In conclusion, in FVII deficiency, the first major bleeding symptom is an independent predictor of the risk of subsequent major bleeds.

Keywords

Asymptomatic individuals - minor bleeds - major bleeds - factor VII deficiency

^* Presented as an oral presentation at the 53rd Annual Meeting of the American Society of Hematology (ASH), San Diego, California (Blood, ASH Annual meeting abstracts) 2011; 118: Abstract 30.

^** A complete list of the members of the Seven Treatment Evaluation Registry (STER) Study Group appears in the Appendix.

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