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Thank you for attending THSNA 2026. The virtual meeting is now closed.
Presentation Details
| Sex and age-based evaluation of a newly formulated intranasal DDAVP compared with intranasal DDAVP and subcutaneous DDAVP in type 1 von Willebrand disease Sanjay Ahuja1, Sweta Gupta1, Magdalena Lewandowska1, Jennifer Maahs1, Sonia Nasr2, Amy Shapiro1. 1Innovative Hematology, Indiana Hemophilia & Thrombosis Center, Indianapolis, IN, USA.2GLOVAL LLC, Broomfield, CO, USA |
Abstract
Introduction: DDAVP (desmopressin) is an efficacious, safe, and cost-effective treatment for bleeding in type 1 von Willebrand disease (VWD). DDAVP is administered via intranasal (IN), subcutaneous (SC), or intravenous (IV) routes. IN DDAVP is preferred for convenience and accessibility, enabling at-home use and decreased treatment burden. Global estimates suggest 84,000 type 1 VWD patients could treat 210,000 bleeds using DDAVP annually. In 2020, production of intranasal desmopressin acetate (Stimate®) was discontinued and ongoing shortages of all DDAVP formulations have further limited global access. In 2021, the Hemophilia Alliance partnered with a registered 503b outsourcing facility to produce a newly formulated IN DDAVP currently available only in the U.S. Objectives: To evaluate the efficacy of newly formulated IN DDAVP compared with the discontinued IN DDAVP and SC DDAVP by age and sex in type 1 VWD. Methods: Retrospective DDAVP responsiveness data were analyzed in 981 type 1 VWD patients at the Indiana Hemophilia and Thrombosis Center. SC DDAVP data were collected from 2007-2025, IN DDAVP from 2000-2020, and newly formulated IN DDAVP from 2021-2025. SC DDAVP dosing was 0.3 mcg/kg. IN DDAVP dosing was 150 mcg for patients <50 kg and 300 mcg for ≥50 kg. Plasma FVIII:C, VWF:Ag, and VWF:Act (VWF:RCo or VWF:GPIbM) were measured at baseline and 90 minutes post-dose. Criteria for responsiveness were based on ASH ISTH NHF WFH guidelines: ≥2x baseline VWF:Act; VWF:Act, and FVIII:C >0.5 IU/mL. This study was exempted from full IRB review. Results: In pediatric patients (<18 years), responsiveness was 74.7% with newly formulated DDAVP (N=170), 80.2% with IN DDAVP (N=440), and 80% with SC DDAVP (N=30); adults (≥18 years) showed comparable responsiveness (63.7%, N=91; 70.9%, N=248; 100%, N=2; respectively). Differences were not statistically significant (Figure 1). In pediatric patients, newly formulated IN DDAVP showed a higher fold-change in FVIII:C (p=0.001) but not VWF:Ag (p=0.75) or VWF:Act (p=0.91) compared with IN DDAVP. Newly formulated IN DDAVP showed a higher fold-change in FVIII:C (p=0.034), VWF:Ag (p=0.039) and VWF:Act (p=0.016) than SC DDAVP. Among adults treated with newly formulated IN DDAVP, FVIII:C fold-change was increased compared with IN DDAVP (p<0.001); no other differences were statistically significant (Figure 2). Differences in fold-change were within expected clinical variability and not considered clinically relevant. In female patients of all ages, responsiveness was 80.3% with newly formulated DDAVP (N=183), 78.3% with IN DDAVP (N=507), and 75.0% with SC DDAVP (N=16); similar responsiveness was observed in males of all ages (66.1%, N=59; 75.0%, N=172; and 86.7%, N=15; respectively). Differences were not statistically significant. In females, newly formulated IN DDAVP showed a higher FVIII:C fold-change (p<0.001) not considered clinically relevant. No other sex-based between-product comparisons were statistically significant. Conclusions: No clinically-relevant sex- or age-dependent differences in DDAVP responsiveness between newly formulated IN DDAVP, IN DDAVP, and SC DDAVP were observed. These results support the use of newly formulated DDAVP for treatment of bleeding in responsive patients with type 1 VWD. Improved availability and full regulatory approval of newly formulated IN DDAVP will broaden global access to effective and safe treatment.
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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.