TABLE 2.
Mean change in efficacy variables from baseline to Week 72 and Week 240
| Mean (95% CI) | Week 72 | Week 240 | ||||||
|---|---|---|---|---|---|---|---|---|
| FIC1 deficiency | t‐BSEP | nt‐BSEP | All BSEP deficiency a | Overall | FIC1 deficiency | nt‐BSEP b | Overall | |
| sBAs, μmol/L | +22.5 (−82.34, 127.28) | −3.3 (−135.55, 128.88) | −38.1 (−151.49, 75.23) | −30.4 (−117.84, 57.04) | −17.2 (−84.70, 50.34) | −17.6 (−106.10, 70.97) | −220.3 (−368.74, −71.84) | −152.7 (−264.35, −41.08) |
| n = 6 | n = 4 | n = 14 | n = 18 | n = 24 | n = 4 | n = 8 | n = 12 | |
| p = 0.605 | p = 0.941 | p = 0.480 | p = 0.473 | p = 0.604 | p = 0.573 | p = 0.010 | p = 0.012 | |
| ItchRO(Obs) score c | −0.7 (−1.75, 0.34) | −1.4 (−2.68, −0.17) | −1.0 (−1.42, −0.54) | −1.1 (−1.44, −0.67) | −1.0 (−1.33, −0.64) | −0.9 (−3.65, 1.79) | −1.7 (−2.60, −0.86) | −1.6 (−2.27, −0.87) |
| n = 6 | n = 4 | n = 18 | n = 22 | n = 28 | n = 2 | n = 8 | n = 10 | |
| p = 0.142 | p = 0.037 | p < 0.001 | p < 0.001 | p < 0.001 | p = 0.144 | p = 0.002 | p < 0.007 | |
| ItchRO(Pt) score c | −0.9 (−3.65, 1.79) | −1.6 (−11.77, 8.50) | −1.0 (−2.01, −0.07) | −1.2 (−1.98, −0.43) | −1.1 (−1.72, −0.57) | −1.1 (NA, NA) | −1.8 (−4.51, 0.94) | −1.6 (−2.69, −0.42) |
| n = 2 | n = 2 | n = 5 | n = 7 | n = 9 | n = 1 | n = 2 | n = 3 | |
| p = 0.144 | p = 0.290 | p = 0.041 | p = 0.009 | p = 0.002 | p = NA | p = 0.076 | p = 0.028 | |
| Height z score | −0.14 (−0.602, 0.327) | −0.16 (−0.840, 0.518) | −0.01 (−0.523, 0.510) | −0.04 (−0.441, 0.360) | −0.06 (−0.371, 0.241) | −0.52 (−2.338, 1.289) | +0.33 (−0.346, 1.010) | +0.05 (−0.573, 0.666) |
| n = 6 | n = 4 | n = 14 | n = 18 | n = 24 | n = 4 | n = 8 | n = 12 | |
| p = 0.481 | p = 0.506 | p = 0.979 | p = 0.832 | p = 0.664 | p = 0.425 | p = 0.285 | p = 0.871 | |
| Weight z score | +0.06 (−0.905, 1.020) | −0.11 (−0.657, 0.432) | −0.05 (−0.368, 0.266) | −0.06 (−0.314, 0.185) | −0.03 (−0.292, 0.224) | +0.29 (−2.195, 2.772) | 0.00 (−0.339, 0.347) | +0.10 (−0.466, 0.664) |
| n = 6 | n = 4 | n = 14 | n = 18 | n = 24 | n = 4 | n = 8 | n = 12 | |
| p = 0.884 | p = 0.558 | p = 0.734 | p = 0.592 | p = 0.787 | p = 0.736 | p = 0.977 | p = 0.707 | |
| PedsQL score | +16 (−0.7, 33.1) | −5 (−37.7, 27.7) | +11 (0.2, 21.2) | +7 (−2.4, 16.8) | +9 (1.2, 17.1) | +19 (−24.3, 62.3) | +22 (7.4, 36.7) | +21 (8.4, 33.8) |
| n = 5 | n = 4 | n = 14 | n = 18 | n = 23 | n = 4 | n = 8 | n = 12 | |
| p = 0.056 | p = 0.662 | p = 0.047 | p = 0.131 | p = 0.025 | p = 0.256 | p = 0.009 | p = 0.004 | |
Note: Shaded cells indicate statistical significance.
Abbreviations: CI, confidence interval; ItchRO(Pt), Itch Reported Outcome Patient.
a
Includes patients with nt‐BSEP and t‐BSEP.
b
No patients with t‐BSEP remained in the study at Week 240.
c
As per study protocol, ItchRO(Obs) data were acquired at Weeks 48 and 242.