TABLE II.
DBPCFC SCD (mg) and change from baseline by treatment group (all participants)
| Time point | PLB-VP250 (N = 20) | VP100-VP250 cross-over (N = 24) | VP250 (N = 25) | Total (N = 69) | P value* |
|---|---|---|---|---|---|
| Baseline,† N | 20 | 24 | 25 | 69 | .585 |
| Median (IQR) | 14.0 (4.0 to 144.0) | 44.0 (4.0 to 144.0) | 14.0 (14.0 to 44.0) | 14.0 (4.0 to 144.0) | |
| (Min, Max) | (1.0, 2044.0) | (0.0, 444.0) | (0.0, 444.0) | (0.0, 2044.0) | |
| Week 52, N | 19 | 21 | 25 | 65 | .476 |
| Median (IQR) | 44.0 (44.0 to 444.0) | 144.0 (44.0 to 144.0) | 144.0 (44.0 to 1044.0) | 144.0 (44.0 to 444.0) | |
| (Min, Max) | (1.0, 2044.0) | (44.0, 2044.0) | (0.0, 2044.0) | (0.0, 2044.0) | |
| Week 130, N | 18 | 18 | 23 | 59 | .003 |
| Median (IQR) | 44.0 (14.0 to 144.0) | 144.0 (144.0 to 444.0) | 444.0 (144.0 to 1044.0) | 144.0 (44.0 to 444.0) | |
| (Min, Max) | (1.0, 2044.0) | (44.0, 2044.0) | (44.0, 2044.0) | (1.0, 2044.0) | |
| Change from baseline at week 52,‡ N | 19 | 21 | 25 | 65 | .535 |
| Median (IQR) | 40.0 (0.0 to 300.0) | 43.0 (0.0 to 140.0) | 130.0 (30.0 to 600.0) | 100.0 (0.0 to 300.0) | |
| (Min, Max) | (0.0, 1900.0) | (−300.0, 2040.0) | (−300.0, 2040.0) | (−300.0, 2040.0) | |
| Change from baseline at week 130,§ N | 18 | 18 | 23 | 59 | .003 |
| Median (IQR) | 11.5 (−3.0 to 40.0) | 141.5 (30.0 to 444.0) | 400.0 (100.0 to 1040.0) | 130.0 (3.0 to 444.0) | |
| (Min, Max) | (−1000.0, 2000.0) | (−100.0, 1600.0) | (−300.0, 2040.0) | (−1000.0, 2040.0) | |
| Change from week 52 at week 130,∥ N | 18 | 18 | 23 | 59 | .018 |
| Median (IQR) | −35.0 (−300.0 to 0.0) | 0.0 (0.0 to 100.0) | 0.0 (0.0 to 300.0) | 0.0 (−100.0 to 100.0) | |
| (Min, Max) | (−2000.0, 2000.0) | (−1000.0, 1600.0) | (−1600.0, 1000.0) | (−2000.0, 2000.0) |
*
The Kruskal-Wallis test was used for comparisons.
†
For PLB-VP250 subjects, the week 52 value was used as baseline.
‡
Wilcoxon pairwise test results for change from baseline at week 52 are P = .305 for PLB-VP250 vs VP250, P = .989 for PLB-VP250 vs VP100-VP250, and P = .410 for VP250 vs VP100-VP250.
§
Wilcoxon pairwise test results for change from baseline at week 130 are P = .002 for PLB-VP250 vs VP250, P = .020 for PLB-VP250 vs VP100-VP250, and P = .397 for VP250 vs VP100-VP250.
∥
Wilcoxon pairwise test results for change from week 52 at week 130 are P = .013 for PLB-VP250 vs VP250, P = .051 for PLB-VP250 vs VP100-VP250, and P = .384 for VP250 vs VP100-VP250.