Presentation Details
| Disease burden, quality of life, and treatment patterns in patients with hereditary factor X deficiency: Analysis of data from the Hereditary Factor X Deficiency in America Survey Kim Clark1, Denise A.Garner2, Amy Wu2, Lorie Mody2, Jaymin Patel2, Brian Branchford3. 1Kedrion Biopharma, INC., Fort Lee, NJ, USA.2AESARA, INC., Chapel Hill, NC, USA.3Versiti Blood Research Institute, Medical College of Wisconsin, and Children's Wisconsin, Milwaukee, WI, USA |
Abstract
Background: Hereditary factor X deficiency (HFXD) is a rare genetic coagulation disorder leading to delayed hemostasis and potentially life-threatening bleeding symptoms. Variability in disease burden may be associated with the number of HXFD treatments a patient has ever received. However, burden of disease and quality of life (QoL) may differ in patients who receive different types of treatments throughout their lifetime. Objectives: Examine disease burden, QoL, and treatment patterns among HFXD patients that have used different numbers and forms of treatments over their lifetime. Methods: A prospective cross-sectional web-based survey of patients with HFXD and their caregivers was conducted. The survey assessed disease burden, QoL, and treatment patterns. Patient-level disease burden was measured using the Hemophilia Well-being Index (HWBI) and Short-Form Health Survey 12 (SF-12). HWBI has a maximum score of 32; lower scores indicate worse well-being. The SF-12 is an overall QoL measure; scores range from 0-100 for each component (physical and mental) with lower scores representing worse QoL. This analysis stratified individuals by number of treatments ever received: 1-2, 3-4, and 5+. Descriptive results are presented. Results: Thirty HXFD patients were included: 10 (33.3%), 14 (46.7%), and 6 (20.0%) patients had ever received 1-2, 3-4, and 5+ treatments, with mean ages of 16 years (40.0% female, 55.6% Asian, and median age at diagnosis 0 years), 24.1 years (71.4% female, 21.4% Asian; median age at diagnosis 1.25 years), and 40.5 years (66.7% female, 33.3% White, 33.3% Black/African-American; median age at diagnosis 2 years), respectively (Table 1). The 1-2 treatment cohort reported an HWBI score of 24 (SD: 11.1). In contrast, the cohorts of 3-4 and 5+ treatments reported lower well-being scores of 20.5 (SD: 7.7) and 14.6 (SD: 5.9), respectively (Table 1). Patients who reported 5+ treatments had lower physical function mean (SD) scores for the SF-12, 36.1 (8.8) relative to 1-2 and 3-4 treatments (47.2 [13.0] and 47.5 [10.6], respectively). Mean (SD) mental function scores were also lower for 5+ treatments, 42.2 (6.7), relative to 1-2 treatments 52.3 (10.3) and 3-4 treatments 49.5 (11.5). Most (≥70%) patients in each treatment cohort reported ever receiving fresh frozen plasma (FFP). The 5+ cohort had the lowest proportion (16.7%) currently receiving FFP; 40% and 21.4% of the 1-2 and 3-4 treatments cohorts, respectively, were currently receiving FFP. All patients in the 5+ cohort had ever received prothrombin complex concentrate (PCC) and single-factor replacement (SFR). In contrast, in the 1-2 treatments cohort, 20% and 50% had ever received PCC and SFR, respectively; however, in the 3-4 treatments cohort, it was 50% and 64.3%, respectively. Few (0-10%) patients in the 1-2 and 3-4 cohorts were currently receiving PCC while 30-57.1% were currently receiving SFR (Table 2). Conclusion: Increased patient burden and lower QoL were more likely in HFXD patients that have received multiple treatments over their lifetime. In addition, more patients who ever received SFR were still on SFR treatment while FFP and PCC were less likely to be continued as current treatment.
No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author.
No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author.