Bleeding Data Across Baseline FIX Expression Levels in People With Hemophilia B: An Analysis Using the ‘Factor Expression Study
Highlights From the 63rd ASH Annual Meeting

Bleeding Data Across Baseline FIX Expression Levels in People With Hemophilia B: An Analysis Using the ‘Factor Expression Study

Tom Burke, MSc1,2*, Anum Shaikh2*, Talaha Ali3*, Nanxin Li, PhD, MBA4, Barbara A Konkle, MD5, Declan Noone, MSc6*, Brian O'Mahony, FACSLM7,8*, Steven W. Pipe, MD9, and Jamie O'Hara, MSc1,2*

1Faculty of Health and Social Care, University of Chester, Chester, United Kingdom
2HCD Economics, Daresbury, United Kingdom
3uniQure, Lexington, MA
4uniQure, Lexington, MA
5University of Washington, Washington Center for Bleeding Disorders, Seattle, WA
6European Haemophilia Consortium, Brussels, Belgium
7Trinity College Dublin, Dublin, Ireland
8Irish Haemophilia Society, Dublin, Ireland
9Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor, MI

Key Data Points

Predicted Bleeds in a Generalized Linear Model of Factor Expression Level

Data from two burden of illness studies (CHESS EU I-II and CHESS US/US+) were used in a generalized linear model (GLM), with log link to evaluate the association between ABR and FIX expression levels (FEL) in patients with hemophilia B. Across all patients included in the analysis (N = 407), the mean FEL (SD) was 9.95 IU/dL (10.5) and the mean ABR (SD) was 2.4 bleeds/year (3.6). The predicted bleeds as a function of FEL produced by the model are shown above. After adjusting for age, BMI, and blood-borne viruses, the average ABR in the model decreased by 0.8 units for each 1% increase in FEL (P < 0.001).

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Supported by educational grants from Bayer, BioMarin, Freeline Therapeutics Limited, Pfizer Inc., Shire, Spark Therapeutics, and uniQure, Inc.

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