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Retrospective Chart Review of Gastrointestinal Bleeding in Patients with von Willebrand Disease: Study Design and Initial Demographic Results

1Bleeding and Clotting Disorders Institute, Peoria, United States, 2University of Texas Health Science Center at Houston, Houston, United States, 3Northwell Health Bleeding Disorders and Thrombosis Program, Cohen Children's Medical Center, Zucker School of Medicine at Hofstra/ Northwell, New Hyde Park, United States, 4Center for Inherited Blood Disorders, CHOC Children's Hospital, Orange, United States, 5School of Medicine Hemophilia and Thrombosis Center, Anschutz Medical Campus, University of Colorado, Aurora, United States, 6Baxalta US Inc., a Takeda Company, Cambridge, United States, 7HCD Economics, Daresbury, United Kingdom, 8Mary M. Gooley Hemophilia Center, Department of Medicine, Rochester, United States

Abstract Number: PB1555

Meeting: ISTH 2020 Congress

Theme: Platelet Disorders and von Willebrand Disease » VWF and von Willebrand Factor Disorders - Clinical Conditions

Background: Specific etiology and treatment of gastrointestinal (GI) bleeds in patients with von Willebrand disease (VWD) is challenging and compounded by limited published data. Despite von Willebrand Factor (VWF) replacement therapy, GI bleeding may be refractory resulting in various treatment approaches with no consensus regarding the most effective therapeutic approach.

Aims: To describe etiology and management of GI bleeds, and outcomes following VWF replacement products and adjuvant therapy, including recombinant VWF (rVWF).

Methods: This retrospective, multicenter, observational chart review included patients with confirmed congenital VWD with ≥1 GI bleed within last 5 years (abstraction initiated 2019 and ongoing). The target sample size is 20 patients from 6 US centers. Demographics and clinical information, including treatment regimens, will be abstracted from patient records on all recorded GI bleeds within the last 5 years. Clinical effectiveness will be defined by treatment response, change in duration of treatment or time to bleed resolution across treatment cohorts (e.g. prophylaxis, on-demand; rVWF, plasma-derived VWF) at the time of a GI bleed and for any subsequent period of prophylactic treatment to prevent GI bleed recurrence. Data will be analyzed descriptively.

Results: To date, data on 22 bleeds in 7 patients with type 1 (14%), type 2 (43%) or type 3 (43%) have been abstracted. 57% were female, mean (±SD) age was 55.71 (18.86) years, and all patients had ≥1 recorded GI-specific morbidity. Treatments used for GI bleeding included aminocaproic acid, tranexamic acid, plasma-derived VWF-factor VIII concentrates, antihemophilic factor (recombinant), rVWF, corticosteroids, polypectomy, and thalidomide.

Conclusions: This retrospective chart review will describe real world experience to identify etiology and management of GI bleeds in VWD patients, and provide insights into the use and effectiveness of current treatments for GI bleeding in individuals with VWD. Results from this study may identify additional therapeutic approaches for the treatment of GI bleeding in VWD.

To cite this abstract in AMA style:

Roberts JC, Escobar M, Acharya S, Hwang NX, Wang M, Hale SA, Oladapo A, Asghar S, Kouides PA. Retrospective Chart Review of Gastrointestinal Bleeding in Patients with von Willebrand Disease: Study Design and Initial Demographic Results [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/retrospective-chart-review-of-gastrointestinal-bleeding-in-patients-with-von-willebrand-disease-study-design-and-initial-demographic-results/. Accessed November 30, 2020.

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