Inhibitors and mortality in persons with nonsevere hemophilia A in the United States

• Type of Material: Article
• Authors: Ming Y. Lim, University of Utah; Dunlei Cheng, American Thrombosis and Hemostasis Network; Michael Recht, American Thrombosis and Hemostasis Network; Christine Kempton, Emory University; Nigel S. Key, University of North Carolina
• Language: English
• Date: 2020-10-13
• Publisher: AMER SOC HEMATOLOGY
• Publication Version: Final Published Version
• Copyright Statement: © 2020 by The American Society of Hematology
• Final Published Version (URL): http://dx.doi.org/10.1182/bloodadvances.2020002626
• Title of Journal or Parent Work: BLOOD ADVANCES
• Volume: 4
• Issue: 19
• Start Page: 4739
• End Page: 4747
• Grant/Funding Information: M.Y.L. received a 2015 HTRS/ATHN DREAM Award from the Hemostasis and Thrombosis Research Society and the American Thrombosis and Hemostasis Network, which was supported by an independent medical educational grant from Shire.
• Keywords: RATES; AIDS; INFECTION; RISK; Hematology; Science & Technology; DEATH; COHORT; FACTOR-VIII; Life Sciences & Biomedicine; POPULATION; HCV
• Research Categories: Health Sciences, Medicine and Surgery

Abstract

Although persons with nonsevere hemophilia A (NSHA) account for about one-half of the hemophilia A population, epidemiological data in this subset of individuals are scarce. We set out to describe the clinical characteristics of persons with NSHA with inhibitors, and to determine mortality rates, predictors of mortality, and primary causes of death in persons with NSHA in the United States over a 9-year period (2010-2018). We queried the American Thrombosis and Hemostasis Network dataset (ATHNdataset) for information on demographics, inhibitor status, and date and cause of death. A total of 6624 persons with NSHA (86.0% men; 14.0% women) were observed for an average of 8.5 years; total 56 119 person-years. The prevalence of inhibitors was 2.6% (n = 171), occurring at a median age of 13 years. At the end of follow-up, 136 persons died at a median age of 63 years; an age-adjusted mortality rate of 3.3 deaths per 1000 person-years. Three deaths occurred in inhibitor participants. Presence of inhibitors was not associated with increased mortality risk (hazard ratio [HR], 0.7, 95% confidence interval [CI], 0.2-2.3). Factors independently associated with increased risk of death (HR, 95% CI) were the following: age (10-year increase) (2.1, 2.0-2.4); male (2.6, 1.0-6.4); hepatitis C (2.2, 1.5-3.1); and HIV (3.6, 2.2-6.0). The most common primary cause of death was malignancy (n - 27, 20.0%). In persons with NSHA, the development of inhibitors occurred at an early age and was not associated with increased mortality.