Trial reportResearch and practice in thrombosis and haemostasis2026
Efficacy and safety of recombinant von Willebrand factor in on-demand treatment of children with von Willebrand disease: up to 4 years of phase 3/3b follow-up.
Trial report in Research and practice in thrombosis and haemostasis, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Not yet cited in PubMed.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Phase 3, Prospective, Multicenter, Uncontrolled, Open-Label Clinical Study to Determine the Efficacy, Safety, and Tolerability of rVWF With or Without ADVATE in the Treatment and Control of Bleeding Episodes, the Efficacy and Safety of rVWF in Elective and Emergency Surgeries, and the Pharmacokinetics (PK) of rVWF in Children Diagnosed With Severe Von Willebrand Disease
A Phase 3b, Prospective, Open-Label, Uncontrolled, Multicenter Study on Long-Term Safety and Efficacy of rVWF in Pediatric and Adult Subjects With Severe Von Willebrand Disease (VWD)
Who cites it
0 citing papers in PubMed.
No citing paper in PubMed yet.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
15 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Background: Recombinant von Willebrand factor (rVWF) is approved to treat von Willebrand disease (VWD) in adults and was recently approved in the United States for pediatric on-demand and perioperative treatment. Objectives: To evaluate the efficacy and safety of rVWF, without or with recombinant factor VIII (rFVIII), for on-demand treatment of bleeding events (BEs) in pediatric patients with VWD. Methods: A prospective, open-label, phase 3 study (NCT02932618) evaluated efficacy and safety of on-demand rVWF, without or with rFVIII, for treating nonsurgical BEs (for up to 18 months) in patients aged <18 years with severe VWD (VWF:ristocetin cofactor <20 IU/dL). The primary end point was treatment success (mean efficacy, <2.5; 4-point scale, 1 [excellent] to 4 [none]). Patients completing the study could enter a phase 3b continuation study (NCT03879135), evaluating safety and efficacy of rVWF for 3 additional years. Results: Of 25 patients in the phase 3 study, 11 (44%) had type 3 VWD. Eighteen patients reported 104 BEs treated with on-demand rVWF; all were treated successfully (mean [SD] efficacy, 1.01 [0.04]). All 98 BEs with efficacy ratings were rated excellent or good. Most BEs (82%) were treated with 1 rVWF infusion; 27% were treated with additional rFVIII. rVWF was well tolerated, with no adverse events leading to study or treatment discontinuation. No thromboembolic events, severe hypersensitivity reactions, or treatment-related serious adverse events occurred. No neutralizing or binding antibodies to rVWF were observed. Efficacy and tolerability of on-demand rVWF were maintained in the continuation study. Conclusion: rVWF, without or with rFVIII, was efficacious for treating nonsurgical BEs in pediatric patients with severe VWD, with efficacy maintained for up to 4 years and no new safety findings.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.