TABLE 2.

Change from baseline to Week 48, Week 72, and EOT in key outcomes (combined studies and observed cases)

Characteristics	Change from baseline to Week 48 (observed cases) (n = 45) Mean (SEM) 95% CI	Change from baseline to Week 72 a (observed cases) (n = 41)	Change from baseline to EOT b (observed cases) (n = 45)
ItchRO(Obs)	42	16	45
	−1.61 (0.16)	−2.00 (0.25)	−1.88 (0.15)
	−1.92, −1.30	−2.48, −1.52	−2.18, −1.58
CSS	44	40	45
	−1.48 (0.23)	−1.43 (0.25)	−1.64 (0.21)
	−1.93, −1.02	−1.91, −0.94	−2.06, −1.23
Serum bile acid (umol/L)	43	37	45
	−62.43 (15.82)	−57.61 (16.50)	−74.68 (15.05)
	−93.43, −31.43	−89.95, −25.27	−104.18, −45.18
PedsQL total, parent	42	30	44
	10.15 (2.57)	10.69 (2.63)	8.31 (2.61)
	5.10, 15.19	5.54, 15.85	3.21, 13.42
Multidimensional fatigue scale	35	25	39
	14.33 (3.16)	12.97 (2.70)	11.27 (3.07)
	8.14, 20.51	7.69, 18.26	5.25, 17.29
Bilirubin (mg/dl)	44	37	45
	0.18 (0.33)	−0.30 (0.25)	−0.05 (0.47)
	−0.46, 0.83	−0.79, 0.19	−0.98, 0.87
Cholesterol (mg/dl)	42	35	43
	−31.19 (14.82)	−38.57 (21.14)	−64.58 (17.76)
	−60.23, −2.15	−80.00, 2.86	−99.39, −29.77
ALT (U/L)	44	37	45
	49.57 (11.34)	50.19 (10.18)	42.22 (14.20)
	27.35, 71.79	30.23, 70.15	14.40, 70.05
Albumin (g/dl)	44	37	45
	−0.04 (0.05)	−0.02 (0.05)	−0.10 (0.06)
	−0.14, 0.06	−0.11, 0.07	−0.21, 0.02
Platelet (103/ul)	42	36	45
	−32.24 (10.59)	−37.28 (9.80)	−62.53 (13.85)
	−53.00, −11.47	−56.49, −18.07	−89.68, −35.38
Height z‐score	44	39	45
	0.22 (0.06)	0.29 (0.08)	0.29 (0.09)
	0.11, 0.34	0.13, 0.44	0.11, 0.47
Weight z‐score	44	39	45
	0.13 (0.08)	0.17 (0.10)	0.13 (0.13)
	−0.02, 0.28	−0.02, 0.37	−0.12, 0.38

^a Week 72 is observed week.

^b For EOT analysis, only participants who have at least 48 weeks in IMAGINE or IMAGINE II are included. The EOT data were obtained as the last value that is before the date of last treatment dose +7 days.