Psychosocial impact and disease management in patients with congenital factor VII deficiency

• Type of Material: Article
• Authors: Skye Peltier, University of Minnesota; Angela Kellum, Tulane University; Janet Brewer, Comprehensive Health Education Services; Alexander Duncan, Emory University; David L. Cooper, Novo Nordisk Inc.; Hossam Saad, Novo Nordisk Inc.
• Language: English
• Date: 2020-01-01
• Publisher: DovePress
• Publication Version: Final Published Version
• Copyright Statement: © 2020 Peltier et al.
• License: Creative Commons Attribution-Noncommercial 3.0 Unported
• Final Published Version (URL): http://dx.doi.org/10.2147/JBM.S259909
• Title of Journal or Parent Work: Journal of Blood Medicine
• Volume: 11
• Start Page: 297
• End Page: 303
• Keywords: bleeding disorder; survey; psychosocial impact; recombinant activated factor VII
• Research Categories: Psychology, Social; Sociology, Public and Social Welfare

Abstract

Purpose: Congenital factor VII (FVII) deficiency is a rare bleeding disorder of variable phenotype with predominantly mucocutaneous bleeding. The aim of this study was to identify the burden of FVII deficiency on patients and caregivers through a better understanding of the management and psychosocial impact of this disease. Materials and Methods: A rare disease specialty recruiter from Comprehensive Health Education Services recruited participants for this online survey, which was conducted from January 31 to March 12, 2019. A moderator-assisted questionnaire was used to collect data on demographics, diagnosis, treatment, and psychosocial impact. Results: Of the 45 respondents (25 patients and 20 caregivers), the majority were female (56%). Respondents reported a wide variety of initial bleeding symptoms, including bruising (58%), epistaxis (56%), and menorrhagia (36% of females). Because symptoms varied between individuals and were not always severe, diagnosis was often delayed. Mean time to obtain a diagnosis was 6.5 years and mean age at first diagnosis was 12.9 years. One-quarter (24%) of the respondents reported more than 100 bleeds of any severity over the previous year. When treating bleeds, 44% of patients reported using antifibrinolytics, and 42% reported using recombinant activated factor VII. Almost 31% of respondents reported missing schooldays as children, and 16% reported losing or resigning from a job in adulthood as a direct result of their disease. Notably, 29% of caregivers and 10% of their partners had also experienced issues with employment. Forty percent of respondents reported not participating in contact sports during childhood, and 22% continued to avoid contact sports in adulthood. Conclusion: Overall, FVII deficiency has a substantial psychosocial impact, but most patients are satisfied with their disease management and are optimistic about their future. Patients desire additional educational, social, and financial support.