Table 2.
Clinical and laboratory findings in study subjects.
| No./age (years) | BS at Dx/OCP Use (−/+) | VWF:RCo U/ml | VWF:Ag U/ml | VIII:C U/ml | RCo/Ag ratio/MULT | PBAC | Bleeding duration – Day | Cycle severity (0 – 3) | VWF:RCo U/ml | VWF:Ag U/ml | VIII:C U/ml | VIII:Ag U/ml | VWF mRNA fold- increase Pre to Day 4 | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Pre | Mo 2 | Mo 6 | Pre | Mo 2 | Mo 6 | Pre | Mo 2 | Mo 6 | Pre | Day 4 | Pre | Day 4 | Pre | Day 4 | Pre | Day 4 | |||||||
| #1/28 | 5 (−) c,e,m,p | 0.34 | 1.23 | 1.32 | 0.28/Nl | 1400 ± 0 | 17 ± 2* | 281 ± 345* | 28.5 ± 16.5 | 7.0 ± 0.0 | 12.6 ± 2.8 | 2.5 ± 0.5 | 0.0 ± 0.0* | 0.8 ± 0.5† | 1.61± ± 05 | 1.26 ± 0.00* | 1.76 ± 0.37 | 1.82 ± 0.10 | 1.56 ± 0.22 | 1.15 ± 0.25* | 1.57 ± 0.05 | 2.25 ± 0.07* | 1.2 (1.2–1.3) |
| #2/35 | 6 (−) c,d,e,m,mu | 0.37 | 0.58 | 0.46 | 0.64/Nl | 222 ± 18 | 92 ± 1* | 61 ± 11* | 7.0 ± 0.5 | 3.5 ± 0.5* | 4.0 ± 0.3* | 2.0 ± 0 | 1.0 ± 0.0* | 1.3 ± 0.2* | 1.25 ± 0.17 | 1.56 ± 0.10 | 0.78 ± 0.03 | 1.11 ± 0.09* | 0.81 ± 0.01 | 1.46 ± 0.08* | 0.72 ± 0.05 | 1.14 ± 0.01* | 13.5 (8.5–21.4) |
| #3/36 | 4 (−) c,g,m | 0.29 | 0.44 | 0.39 | 0.66/Nl | 145 ± 138 | 19 ± 14 | 15 ± 4 | 6.0 ± 1.0 | 4.0 ± 2.0 | 4.0 ± 0.6 | 1.5 ± 0.5 | 0.5 ± 0.5 | 0.2 ± 0.2* | 1.06 ± 0.12 | 1.30 ± 0.02‡ | 0.58 ± 0.29 | 0.98 ± 0.36 | 1.09 ± 0.10 | 1.95 ± 0.66 | 0.99 ± 0.22 | 2.19 ± 0.02* | 2.7 (1.7–4.3) |
| #4/29 | 6 (+) c,e,m,p | 0.22 | 0.72 | 0.82 | 0.30/Nl | 1025 ± 0 | 701 ± 49 | 557 ± 161* | 6.0 ± 0.0 | 12.0 ± 4.0 | 9.0 ± 1.8 | 2.0 ± 0 | 2.5 ± 0.5 | 1.8 ± 0.3 | 0.87 ± 0.17 | 1.42 ± 0.20‡ | 0.75 ± 0.13 | 1.71 ± 0.10* | 0.98 ± 0.11 | 1.35 ± 0.03* | 0.90 ± 0.02 | 2.37 ± 0.05* | 1.0 (0.9–1.1) |
| #5/40 | 5 (+) c,m,mu,w | 0.43 | 0.54 | 1.03 | 0.80/Nl | 410 ± 0 | 140 ± 60* | 94 ± 39* | 6.0 ± 0.0 | 4.0 ± 0.0* | 3.8 ± 0.2* | 2.0 ± 0 | 1.0 ± 0.0* | 1.0 ± 0.3* | 0.88 ± 0.05 | 1.37 ± 0.17* | 0.60 ± 0.01 | 0.69 ± 0.07 | 0.80 ± 0.18 | 0.84 ± 0.11 | 0.67 ± 0.21 | 0.80 ± 0.34 | 5.1 (4.5–5.8) |
| #6/42 | 4 (−) c,e,m | 0.10 | 0.15 | 0.24 | 0.67/Nl | 561 ± 4 | 46 ± 0.5* | 56 ± 24* | 6.5 ± 0.5 | 4.5 ± 0.5* | 4.2 ± 0.3* | 3.0 ± 0 | 0.0 ± 0.0* | 0.7 ± 0.3* | 0.12 ± 0.00 | 0.12 ± 0.00 | 0.12 ± 0.02 | 0.17 ± 0.01* | 0.31 ± 0.01 | 0.60 ± 0.12† | 0.25 ± 0.01 | 0.36 ± 0.01* | 1.6 (1.4–1.7) |
| #7/21 | 2 (+) m | 0.10 | 0.25 | 0.36 | 0.40/Nl | 299 ± 78 | 177 ± 59 | 171 ± 32 | 9.5 ± 1.5 | 6.5 ± 1.5 | 5.4 ± 0.7* | 3.0 ± 0 | 1.5 ± 0.5* | 1.4 ± 0.2* | 0.12 ± 0.00 | 0.12 ± 0.00 | 0.06 ± 0.02 | 0.12 ± 0.01* | 0.38 ± 0.02 | 0.29 ± 0.11 | 0.21 ± 0.01 | 0.31 ± 0.01* | ns |
BS is bleeding score; Dx is diagnosis; OCP is oral contraceptive use during study; VWF:RCo, von Willebrand ristocetin cofactor; Ag; antigen; FVIII:C, factor VIII activity; MULT is multimers; Nl is normal multimers; OCP is oral contraceptive use during study; c, cutaneous; d, dental; e, epistaxis; g, gastrointestinal; m, menorrhagia; mu, musculoskeletal; o, oral; p, postpartum; s, surgery; w, wound bleeding; PBAC is pictorial blood assessment chart. VWFmRNA is von Willebrand factor mRNA. ns is sample not sufficient. Clinical and laboratory values are expressed as mean ± standard error of the mean (SEM). Student’s t test was used to compare pre-treatment clinical parameters from two baseline cycles, with values after 2 cvcles (inclusive) and after 6 cycles (inclusive) post-treatment; and to compare pre-treatment laboratory assays with day 4 values post-treatment. Significance is indicated by
p < 0.001;
p < 0.01;
p < 0.05.