Presentation Details
| Evaluation of Real-world Treatment Patterns and Outcomes in Patients with Von Willebrand Disease Treated Prophylactically with Recombinant Von Willebrand Factor Across Treatment Centers in the United States Jonathan C Roberts1, Maissaa Janbain2, Jessica R Marden3, Sanjana Sundaresan3, Natalia Nieto4, Bethany Jones4, Jorge Caicedo4. 1Bleeding & Clotting Disorders Institute, Peoria, IL, USA.2Tulane University School of Medicine, New Orleans, LA, USA.3Analysis Group, Inc., Boston, MA, USA.4Takeda Pharmaceuticals U.S.A., Inc, Lexington, MA, USA |
Abstract
Background: Von Willebrand disease (VWD) is a bleeding disorder caused by deficient or defective von Willebrand factor (VWF). People with VWD experience increased bleeding, which can lead to decreased quality of life, and may benefit from prophylactic treatment. Real-world effectiveness of prophylaxis with recombinant VWF (rVWF; vonicog alfa; Takeda Pharmaceuticals U.S.A., Inc., Lexington, MA, USA) was investigated in a retrospective center-based chart review. Objective: To evaluate the real-world clinical characteristics, treatment patterns, and outcomes of rVWF prophylaxis in people with VWD in the United States. Methods: This observational study included participants aged ≥12 years with a confirmed VWD diagnosis from US healthcare centers who have received either routine or intermittent (for heavy menstrual bleeding [HMB]) prophylactic rVWF treatment. Eligibility criteria included availability of medical records during the baseline period (≥6 months pre-rVWF initiation) and rVWF period (≥6 months post-rVWF initiation). Annualized bleed rate (ABR), healthcare resource utilization (HCRU), and treatment patterns were the main outcomes of interest. Baseline characteristics were summarized descriptively. Outcomes were compared between the baseline and rVWF periods using Wilcoxon signed-rank tests and McNemar’s tests. Results: Data from 30 participants across 11 sites were analyzed; 23 (76.7%) participants received routine rVWF prophylaxis (mean ± SD age: 42.0 ± 21.4 years; 52.2% female), and 7 (23.3%) participants received intermittent rVWF prophylaxis (mean ± SD age: 27.6 ± 11.6 years; 100% female). All three VWD subtypes were represented (Type 1: 7 [23.3%]; Type 2: 13 [43.3%]; Type 3: 10 [33.3%]). Mean duration of prophylactic treatment was 2.9 years in the routine cohort and 2.2 years in the intermittent cohort; treatment is ongoing in most study participants (Table 1). Seventeen (73.9%) participants in the routine cohort received prior prophylaxis with plasma-derived VWF. Mean weekly initial dose in the routine cohort was 132.2 IU/kg and decreased by 14.2 IU/kg during the study period; participants received an average of 1.8 doses per week (Table 1). Mean initial dose in the intermittent cohort was 44.8 IU/kg and increased by 4.4 IU/kg during the study period; most participants received treatment during menses (Table 1). ABR decreased in the routine cohort, mainly driven by reductions in major bleeds (Table 2). Decreased ABR in the intermittent cohort was nonsignificant (Table 2). The number of participants without bleeds increased in both cohorts despite the longer timeframe of the rVWF period (Table 2). Total and bleed-related inpatient visits and number of surgeries decreased in the routine cohort (Table 2). Conclusions: Study sample size is limited, owing to the rarity of people with VWD using prophylaxis. Participants receiving routine rVWF prophylaxis in this study experienced reduced ABR and HCRU relative to the baseline period, indicating that rVWF prophylaxis may result in fewer bleeds and less hospitalization. This study adds to the growing body of real-world evidence investigating the use of rVWF prophylaxis, including 2 clinical trials (Leebeek, 2022, Blood; Ragni, 2023, Lancet Haematol) and 1 observational study (ATHN 9: NCT03853486), and suggests potential effectiveness of routine and intermittent rVWF prophylaxis in people with VWD across all subtypes.
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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.