Abstract Number: PB1549
Meeting: ISTH 2020 Congress
Theme: Platelet Disorders and von Willebrand Disease » VWF and von Willebrand Factor Disorders - Clinical Conditions
Background: Gastro-intestinal (GI) bleeding is a characteristic feature of severe Von Willebrand disease (VWD), that arises generally in older patients, requiring in most cases blood transfusion and hospitalization. The presence of arteriovenous malformations is often described. Patients with congenital type 3, 2A and 2B are those most frequently affected by this symptom, which could be due to the loss of high-molecular-weight multimers (HMWM).
Aims: To evaluate the response to treatment in case of GI bleeding in patients with severe VWD.
Methods: Case-series. All charts from patients with severe VWD (defined as any type of VWD with VWF:RCo < 6 IU/dL) evaluated at the A. Bianchi Bonomi Hemophilia and Thrombosis Center in Milano between 2015 and 2019 were reviewed. GI bleeding was defined as occult (positive stool test) or evident (hematemesis or melena).
Results: A total of 116 patients were evaluated, finding 35 (30%) with at least one episode of GI bleeding during their life (occult in 4); 2 patients with severe GI bleeding (not responsive to treatment with FVIII/VWF concentrate) were excluded as they presented a bleeding comorbidity (Idiopathic Thrombocytopenia and Hereditary hemorrhagic telangiectasia), 1 patient was excluded because bleeding was due to hemorrhoids inflammation and was resolved by surgery. A blood transfusion was required in 62.5% (20/32 patients) and a local lesion (ulcer, gastric or intestinal polyps or mucosal inflammation) was associated to GI bleeding in 41% of patients (13/32). Recurrent bleeding was present in 56% of patients (18/32). Tables 1 and 2 present patients and GI bleeding characteristics.
Conclusions: One-third of the patients with severe VWD presented at least 1 episode of GI bleeding during their life. A fifth of the patients with GI bleeding (n=7, 22%), all characterized by loss of HMWM, needed long term prophylaxis or some rescue therapy (surgery, atorvastatin, octreotide, lenalinomide).
| All patients, N=116 | Patients with GI bleeding, N=32 | |||
| Female sex | number (%) | 58 (50) | 15 (47) | |
| Age at diagnosis, years | median (range) | 19 (0-69) | 34 (3-53) | |
| Age at last follow-up, years | median (range) | 49 (6-88) | 61 (7-87) | |
| FVIII:C, IU/dL | median (range) | 34 (1-110) | 29 (1-76) | |
| VWD type | with loss of HMWM (type 3, 2A and 2B) | number (%) | 68 (59) | 20 (62.5) |
| without loss of HMWM (type 2M and 1, not classified) | number (%) | 48 (41) | 12 (37.5) | |
| Age at last follow-up, years | with loss of HMWM (type 3, 2A and 2B), | median (range) | 45 (6-87) | 54 (7-87) |
| without loss of HMWM (type 2M and 1, not classified), | median (range) | 57 (7-86) | 65 (38-86) |
[Table 1. Patients characteristics.]
| Patients with GI bleeding N=32 | VWD with loss of HMWM N=20 | VWD without loss of HMWM N=12 | ||
| Age at first GI bleeding, years | median (range) | 45 (3-81) | 43 (3-75) | 49 (6-81) |
| GI bleeding event before diagnosis | number (%) | 12 (37.5) | 7 | 5 |
| Local lesion causing GI bleeding | number (%) | 13 (41) | 5 | 8 |
| Recurrent GI bleeding | number (%) | 18 (56) | 12 | 6 |
| Presence of angiodysplasia | number (%) | 8 (25) | 5 | 3 |
| No treatment with FVIII/VWF concentrate | number (%) | 8 (25) | 5 | 3 |
| Treatment (FVIII/VWF concentrate) with complete response | number (%) | 14 (44) | 5 | 6 |
| Long term prophylaxis (FVIII/VWF concentrate) with complete response | number (%) | 3 (9) | 3 | – |
| Need of rescue therapy associated with prophylaxis | number (%) | 4 (13) | 4 | – |
[Table 2. Characteristics of GI bleeding events.]
To cite this abstract in AMA style:
Biguzzi E, Siboni SM, Gualtierotti R, Braham S, Pagliari MT, Peyvandi F. Response to Treatment for Gastro-Intestinal Bleeding in von Willebrand Disease [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/response-to-treatment-for-gastro-intestinal-bleeding-in-von-willebrand-disease/. Accessed September 29, 2023.« Back to ISTH 2020 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/response-to-treatment-for-gastro-intestinal-bleeding-in-von-willebrand-disease/