Table 1.
Clinical and Laboratory Findings in Study Subjects
| Pt | Age/ Sex |
Dx | VWF: RCo (%) |
VWF: Ag (%l) |
FVIII: C (%) |
DDAVP Response (Historic) |
OCP Use |
Pre | VWF:RCo (%) Day 4 |
DDAVP | Pre | VWF:Ag (%) Day 4 |
DDAVP | Pre | FVIII:C (%) Day 4 |
DDAVP | Pre | FVIII:Ag (%) Day 4 |
DDAVP | VWF mRNA Fold- increase Pre - Day 4 |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| VWD Patients | ||||||||||||||||||||
| # 1 | 25/F | 1 VWD | 12.0 | 11.0 | 32.0 | Incomplete | − | ≤12.5±0.0 | 22.0±9.5 | 45.7±26.2 | 12.0±5.4 | 25.4±6.2 | 48.8±15.8 | 18.0±1.0 | 48.0±7.0‡ | 124.7±35.6† | 24.6±5.3 | 25.7±1.0 | 66.3±22.0 | 0.55-fold |
| # 2 | 22/F | 2B VWD | 20.0 | 6.0 | 39.0 | Not indicated |
− | ≤12.5±0.0 | ≤12.5±0.0 | NA | 24.7±2.6 | 35.4±18.0 | NA | 37.4±12.3 | 85.7±13.2‡ | NA | 29.7±1.3 | 50.9±5.4‡ | NA | NS |
| # 3 | 25/F | 1 VWD | 10.0 | 6.0 | 16.0 | Incomplete | + | ≤12.5±0.0 | ≤12.5±0.0 | 32.2±19.7 | 4.5±0.1 | 5.7±0.4 | 26.2±14.9 | 6.9±0.0 | 5.5±0.1‡ | 53.6±33.8 | 8.9±0.6 | 11.3±0.3‡ | 46.0±22.8 | −0.29-fold |
| # 4 | 26/F | 2M VWD | 10.0 | 48.0 | 99.0 | Allergic | + | 64.0±4.0 | 92.0±2.0‡ | NA | 36.7±1.3 | 71.7±18.3 | NA | 88.5±6.3 | 149.9±14.8‡ | NA | 46.1±0.3 | 86.6±19.1* | NA | −0.47-fold |
| Fold Increase | 1.30-fold | 2.33-fold | 1.69-fold | 3.25-fold | 1.86-fold | 6.17-fold | 1.47-fold | 3.32-fold | 0.07-fold | |||||||||||
| Hemophilia A Patients | ||||||||||||||||||||
| # 5 | 25/M | Mild HA | 58.0 | 65.0 | 8.0 | Incomplete | − | 78.0±5.0 | 121.0±25.0 | 227.0±11.0‡ | 80.8±7.1 | 138.9±2.7‡ | 207.1±5.9‡ | 10.2±0.3 | 19.2±1.2‡ | 78.2±12.4‡ | 5.8±0.6 | 12.7±0.7‡ | 36.1±6.2‡ | 2.07-fold |
| # 6 | 51/M | Mild HA | 128.0 | 92.0 | 12.0 | Incomplete | − | 73.5±3.5 | 151.0±43.0‡ | 191.0±25.0 | 80.2±5.7 | 171.3±8.9‡ | 222.0±19.7† | 24.8±0.7 | 37.1±1.4‡ | 74.0±3.7‡ | 12.5±0.4 | 25.5±0.3‡ | 43.3±3.5‡ | 0.40-fold |
| # 7 | 31/M | Mild HA | 135.0 | 141.0 | 11.0 | Responsive | − | 112.0±24.0 | 208.0±0.0‡ | 324.0±60.0 | 135.4±2.9 | 235.3±13.0‡ | 273.4±43.8 | 7.4±0.2 | 7.8±0.0* | 23.8±7.9* | 11.4±2.6 | 19.1±0.8‡ | 46.5±17.6 | 1.15-fold |
| # 8 | 28/M | Mild HA | NA | NA | 12.0 | Responsive | − | 92.0±10.0 | 140.0±4.0 | 290.0±10.0 | 122.1±11.1 | 141.9±30.0 | 277.9±7.0‡ | 26.9±0.4 | 32.6±0.1‡ | 103.1±11.0‡ | 15.0±0.3 | 21.2±1.1‡ | 53.3±4.3‡ | 0.40-fold |
| # 9 | 34/M | Mod HA | NA | NA | 4.0 | Incomplete | − | 58.0±8.0 | 97.0±3.0‡ | 171.0±9.0‡ | 80.3±6.6 | 135.7±16.1† | 198.1±11.8† | 1.7±0.2 | 1.9±0.2 | 5.2±1.3† | 6.9±0.0 | 6.9±0.0 | 6.9±0.0 | 2.65-fold |
| Fold increase | 1.73-fold | 1.71-fold | 1.69-fold | 1.47-fold | 1.48-fold | 3.00-fold | 1.66-fold | 2.10-fold | 1.33-fold | |||||||||||
Dx is diagnosis; VWD is von Willebrand disease; HA is hemophilia A; VWF:RCo is von Willebrandristocetin cofactor activity; VWF:Ag is von Willebrand factor antigen; FVIII:C is factor VIII activity; DDAVP is desmopressin; OCP is oral contraceptive use during study; VWF mRNA is VWF messenger RNA; NA is not applicable; NS is not sufficient. Results are expressed as mean ± standard error of the mean (SEM).
indicates p<0.05;
is p<0.01;
is p<0.001. Fold-increase in VWF and F.VIII values by Day 4 post rhIL-11 are comparedwith pre-rhIL-11, and Day 4 post DDAVP with Day 4 pre-DDAVP (post rhIL-11) values.For the “Pre” values, we averaged two baseline levels, one at screening and one before rhIL-11 injection on Day 1. For the “Day 4” values, we averaged the day 3 and day 4 values. For the “DDAVP” values, we averaged values obtained at 30 minutes and 4 hours after DDAVP.