AEs in patients who received rVWF prophylaxis (safety analysis set)*
| . | Prior on-demand group (n = 13) n (%)/events . | Switch group (n = 10) n (%)/events . | Total (n = 23) n (%)/events . |
|---|---|---|---|
| AE† | 10 (76.9)/26 | 7 (70.0)/15 | 17 (73.9)/41 |
| Mild | 7 (53.8)/18 | 4 (40.0)/12 | 11 (47.8)/30 |
| Moderate | 1 (7.7)/5 | 2 (20.0)/2 | 3 (13.0)/7‡ |
| Severe | 2 (15.4)/3 | 1 (10.0)/1 | 3 (13.0)/4§ |
| Serious AE | 1 (7.7)/1 | 2 (20.0)/2 | 3 (13.0)/3 |
| AE considered related to rVWF | 1 (7.7)/1 | 0 | 1 (4.3)/1 |
| Serious AE considered related to rVWF | 0 | 0 | 0 |
| AE considered related to study procedures | 0 | 0 | 0 |
| Serious AE considered related to study procedures | 0 | 0 | 0 |
| AE leading to discontinuation of rVWF | 1 (7.7)/1 | 0 | 1 (4.3)/1 |
| Fatal AE | 0 | 0 | 0 |
| Life-threatening AE | 0 | 0 | 0 |
| AE of special interest‖ | 1 (7.7)/1¶ | 1 (10.0)/1** | 2 (8.7)/2 |
| . | Prior on-demand group (n = 13) n (%)/events . | Switch group (n = 10) n (%)/events . | Total (n = 23) n (%)/events . |
|---|---|---|---|
| AE† | 10 (76.9)/26 | 7 (70.0)/15 | 17 (73.9)/41 |
| Mild | 7 (53.8)/18 | 4 (40.0)/12 | 11 (47.8)/30 |
| Moderate | 1 (7.7)/5 | 2 (20.0)/2 | 3 (13.0)/7‡ |
| Severe | 2 (15.4)/3 | 1 (10.0)/1 | 3 (13.0)/4§ |
| Serious AE | 1 (7.7)/1 | 2 (20.0)/2 | 3 (13.0)/3 |
| AE considered related to rVWF | 1 (7.7)/1 | 0 | 1 (4.3)/1 |
| Serious AE considered related to rVWF | 0 | 0 | 0 |
| AE considered related to study procedures | 0 | 0 | 0 |
| Serious AE considered related to study procedures | 0 | 0 | 0 |
| AE leading to discontinuation of rVWF | 1 (7.7)/1 | 0 | 1 (4.3)/1 |
| Fatal AE | 0 | 0 | 0 |
| Life-threatening AE | 0 | 0 | 0 |
| AE of special interest‖ | 1 (7.7)/1¶ | 1 (10.0)/1** | 2 (8.7)/2 |
Table displays the number and percentage of patients who had ≥1 AE and the number of AEs for a given parameter.
AEs starting or worsening after the first dose of rVWF.
Patients were counted once for the highest severity.
Joint (shoulder) injury, supraventricular tachycardia, and ventricular extrasystoles (all in the same patient); joint (knee) injury; headache; arthralgia; gastroenteritis; all events resolved; includes events in 2 patients who also had severe events and so are listed in the severe category.
Fall and multiple injuries from fall (2 events in the same patient and requiring hospitalization); toothache; rheumatoid arthritis. All events resolved except rheumatoid arthritis.
AEs of special interest defined as thromboembolic events, hypersensitivity reactions (including allergic or anaphylactic reactions), the development of neutralizing or binding antibodies to VWF and FVIII, and binding antibodies to trace proteins in rVWF (Chinese hamster ovary immunoglobulin G [IgG], murine IgG, and human Furin IgG).
Purpura, which developed due to trauma, was classified as a thromboembolic event (per broad SMQ search); considered nonserious and nonsevere by investigator, and resolved with no action taken.
Rash pruritic was classified as a hypersensitivity reaction (per broad SMQ search); considered nonserious and nonsevere by investigator, and resolved with no action taken.