Type 2A von Willebrand disease and systemic sclerosis: Vonicog alfa reduced gastrointestinal bleeding

Korsten, Peter; Wallbach, Manuel; Binder, Claudia R.

doi:10.1002/rth2.12426

Cited by 3 publications

(3 citation statements)

References 14 publications

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“…Thus, because of the presence of ultra‐large multimers in rVWF and the proposed relationship between absence of HMWMs and pathophysiology of GI bleeding, it has been hypothesized that bleeding associated with angiodysplasia may respond better to rVWF than replacement pdVWF, which contains lower–molecular‐weight multimers 34–36 . Case reports have shown successful bleed resolution in previously refractory mucosal BEs, including refractory GI bleeding, with rVWF 37–39 . While most BEs treated with rVWF in the current report successfully resolved after rVWF, the extremely small sample size precludes drawing conclusions about the effectiveness of rVWF in treating GI BEs; prospective/registry studies are needed to fully explore rVWF effectiveness in GI BE.…”

Section: Discussionmentioning

confidence: 64%

“…[34][35][36] Case reports have shown successful bleed resolution in previously refractory mucosal BEs, including refractory GI bleeding, with rVWF. [37][38][39] While most BEs treated with rVWF in the current report successfully resolved after rVWF, the extremely small sample size precludes drawing conclusions about the effectiveness of rVWF in treating GI BEs; prospective/registry studies are needed to fully explore rVWF effectiveness in GI BE.…”

Section: Discussionmentioning

confidence: 86%

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Retrospective chart review of GI bleeding in people with von Willebrand disease

Roberts,

Escobar,

Acharya

et al. 2024

Haemophilia

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IntroductionGastrointestinal (GI) bleeding events (BEs) in von Willebrand disease (VWD) are difficult to diagnose and often recurrent. Limited data from clinical trials has led to lack of consensus on treatment options.AimDescribe current treatments and outcomes for GI BEs in people with VWD.MethodsThis retrospective, observational, multicentre chart review study was conducted from January 2018 through December 2019 and included patients with inherited VWD with ≥1 GI BE in the preceding 5 years. Baseline characteristics, number and aetiology of BEs, associated GI‐specific morbidities/lesions, treatment and outcomes were analysed descriptively.ResultsSixty bleeds were reported in 20 patients with type 1 (20%), type 2 (50%) and type 3 (30%) VWD. During the 5‐year study period, 31 (52%) BEs had one identified or suspected cause; multiple causes were reported in 11 (18%). Most GI BEs (72%) were treated with a combination of von Willebrand factor (VWF), antifibrinolytics and/or other haemostatic or non‐haemostatic treatments. Time to resolution did not differ by VWF treatment use; however, BEs treated with non‐VWF treatments tended to resolve later. In patients with GI‐specific morbidities/lesions, 84% resolved with first‐line treatment; time to resolution tended to be longer than in patients without such morbidities/lesions. Thirteen BEs occurred in patients receiving prophylaxis and 47 in patients receiving on‐demand treatment; 18 BEs resulted in a switch to prophylaxis after bleed resolution.ConclusionsThis study confirms the unmet need for the management of recurrent GI BEs in people with VWD and the need for prospective data, especially on prophylaxis.

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Section: Discussionmentioning

confidence: 64%

Section: Discussionmentioning

confidence: 86%

Retrospective chart review of GI bleeding in people with von Willebrand disease

Roberts,

Escobar,

Acharya

et al. 2024

Haemophilia

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“…Our search identified 10,019 unique studies. After screening by title and abstract, and full‐text review, 61 studies were included in the review of which 54 had patient‐level data comprising 146 patients 8,10,15–66 and nine reported data usable at the population or survey level 9,11,19,51,67–71 (Figure 1). There were no new studies for inclusion identified by review of reference lists.…”

Section: Resultsmentioning

confidence: 99%

Vascular abnormalities in patients with von Willebrand disease: A scoping review

Chornenki

Shanjer

James

2021

Journal of Thrombosis and Haemostasis

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Background: Qualitative or quantitative defects of von Willebrand factor (VWF) such as in von Willebrand disease (VWD) are associated with vascular abnormalities, especially in the gastrointestinal (GI) tract. However, the locations, extent, and natural history of vascular abnormalities in patients with VWD is not well understood. To summarize the existing literature on the topic, we conducted a scoping review of vascular abnormalities in patients with VWD. Methods:We searched MEDLINE and EMBASE from inception to September 1, 2020, for studies clinically describing vascular abnormalities in VWD patients. Screening and data extraction was completed independently and in duplicate and each abnormality was documented individually.Results: After screening, 54 studies that reported patient level data comprising 146 patients were included. Type 2A (39%) and type 3 (14.4%) were the most common VWD subtypes. The most common site of vascular malformation was the GI tract, occurring in 124 patients (84.9%), whereas 18 (12.3%) had non-GI vascular abnormalities and 4 (2.7%) had both GI and non-GI vascular abnormalities. With respect to outcomes, the clinical course was not specified in the majority (55.5%) of patients.Survey and population level data were reported in nine studies, demonstrating vascular abnormalities occurred at higher rates in VWD and that VWD patients are overrepresented among those with those abnormalities. Conclusion:Vascular malformations in patients with VWD occur primarily in the gastrointestinal tract. Type 2A and type 3 VWD are the most common subtypes affected.The clinical treatment and natural history of these abnormalities remains understudied and further research is needed.

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Ambrisentan/factor-VIII/von-Willebrand factor

2021

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Type 2A von Willebrand disease and systemic sclerosis: Vonicog alfa reduced gastrointestinal bleeding

Cited by 3 publications

References 14 publications

Retrospective chart review of GI bleeding in people with von Willebrand disease

Retrospective chart review of GI bleeding in people with von Willebrand disease

Vascular abnormalities in patients with von Willebrand disease: A scoping review

Ambrisentan/factor-VIII/von-Willebrand factor

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