Table 2.
Changes in Pruritus, Quality of Life, Sleep, and Patient Perception of Treatment Benefits from Baseline to Week 13/et
| Maralixibat 10 mg (n = 21) | Maralixibat 20 mg (n = 21) | Maralixibat Overall (n = 42) | Placebo (n = 24) | |
|---|---|---|---|---|
| 5‐D Itch score | ||||
| LS mean change from baseline (95% CI) | –6.9 | –6.7 | –6.8 | –6.3 |
| (–8.9, –4.8)‡ | (–8.8, –4.6)‡ | (–8.2, –5.3)‡ | (–8.3, –4.4)‡ | |
| LS mean difference from placebo (95% CI) | –0.5 | –0.4 | –0.5 | N/A |
| (–3.4, 2.3) | (–3.2, 2.5) | (–2.9, 2.0) | ||
| PBC‐40 domain score | ||||
| Itch (3 items) | ||||
| LS mean change from baseline (95% CI) | –4.0 | –4.1 | –4.1 | –3.8 |
| (–5.3, –2.8)‡ | (–5.4, –2.9)‡ | (–5.0, –3.2)‡ | (–5.0, –2.6)‡ | |
| LS mean difference from placebo (95% CI) | –0.3 | –0.4 | –0.3 | N/A |
| (–2.0, 1.5) | (–2.1, 1.4) | (–1.8, 1.2) | ||
| Emotional (3 items) | ||||
| LS mean change from baseline (95% CI) | –1.4 | –1.2 | –1.3 | –0.5 |
| (–2.2, –0.7)‡ | (–2.0, –0.4)† | (–1.9, –0.8)‡ | (–1.3, 0.2) | |
| LS mean difference from placebo (95% CI) | –0.9 | –0.7 | –0.8 | N/A |
| (–2.0, 0.2) | (–1.7, 0.4) | (–1.7, 0.1) | ||
| Cognitive (6 items) | ||||
| LS mean change from baseline (95% CI) | –1.5 | –0.5 | –1.0 | –1.6 |
| (–3.0, –0.1)* | (–2.0, 0.9) | (–2.0, 0.0)* | (–3.0, –0.3)* | |
| LS mean difference from placebo (95% CI) | 0.1 | 1.1 | 0.6 | N/A |
| (–1.8, 2.1) | (–0.8, 3.1) | (–1.1, 2.3) | ||
| Symptoms (7 items) | ||||
| LS mean change from baseline (95% CI) | –0.8 | –0.4 | –0.6 | –1.8 |
| (–2.3, 0.7) | (–1.9, 1.1) | (–1.6, 0.4) | (–3.2, –0.4)* | |
| LS mean difference from placebo (95% CI) | 1.0 | 1.4 | 1.2 | N/A |
| (–1.0, 3.0) | (–0.7, 3.4) | (–0.6, 2.9) | ||
| Social (10 items) | ||||
| LS mean change from baseline (95% CI) | –2.0 | –2.8 | –2.4 | –2.1 |
| (–4.4, 0.4) | (–5.2, –0.3)* | (–4.1, –0.7)† | (–4.5, 0.2) | |
| LS mean difference from placebo (95% CI) | 0.1 | –0.6 | –0.2 | N/A |
| (–3.2, 3.5) | (–4.0, 2.7) | (–3.1, 2.6) | ||
| Fatigue (11 items) | ||||
| LS mean change from baseline (95% CI) | –2.3 | –2.8 | –2.5 | –4.9 |
| (–5.5, 0.9) | (–6.0, 0.4) | (–4.8, –0.3)* | (–7.9, –1.8)† | |
| LS mean difference from placebo (95% CI) | 2.6 | 2.1 | 2.3 | N/A |
| (–1.8, 7.0) | (–2.3, 6.5) | (–1.4, 6.1) | ||
| MOS‐Sleep Scale score | ||||
| Sleep disturbances (4 items) | ||||
| LS mean change from baseline (95% CI) | –9.2 | –9.0 | –9.1 | –15.6 |
| (–18.8, 0.5) | (–18.6, 0.7) | (–15.9, –2.3)* | (–24.8, –6.4)† | |
| LS mean difference from placebo (95% CI) | 6.4 | 6.6 | 6.5 | N/A |
| (–6.9, 19.8) | (–6.7, 19.9) | (–4.9, 18.0) | ||
| Snoring (1 item) | ||||
| LS mean change from baseline (95% CI) | –8.5 | –5.7 | –7.1 | 0.3 |
| (–17.5, 0.6) | (–14.9, 3.6) | (–13.5, –0.6)* | (–8.4, 8.9) | |
| LS mean difference from placebo (95% CI) | –8.7 | –5.9 | –7.3 | N/A |
| (–21.2, 3.7) | (–18.8, 6.9) | (–18.2, 3.5) | ||
| Awaken short of breath or headache (1 item) | ||||
| LS mean change from baseline (95% CI) | –6.3 | –3.0 | –4.6 | –0.9 |
| (–16.4, 3.9) | (–13.2, 7.2) | (–11.8, 2.6) | (–10.5, 8.8) | |
| LS mean difference from placebo (95% CI) | –5.4 | –2.1 | –3.7 | N/A |
| (–19.4, 8.7) | (–16.2, 12.0) | (–15.8, 8.3) | ||
| Sleep adequacy (2 items) | ||||
| LS mean change from baseline (95% CI) | 7.1 | 2.1 | 4.6 | 3.9 |
| (–3.8, 18.0) | (–8.8, 13.0) | (–3.1, 12.3) | (–6.4, 14.2) | |
| LS mean difference from placebo (95% CI) | 3.2 | –1.8 | 0.7 | N/A |
| (–11.8, 18.2) | (–16.8, 13.2) | (–12.1, 13.5) | ||
| Somnolence (3 items) | ||||
| LS mean change from baseline (95% CI) | –5.0 | –10.3 | –7.7 | –11.7 |
| (–14.3, 4.2) | (–19.6, –1.1)* | (–14.2, –1.2)* | (–20.5, –3.0)† | |
| LS mean difference from placebo (95% CI) | 6.7 | 1.4 | 4.1 | N/A |
| (–6.0, 19.4) | (–11.3, 14.1) | (–6.8, 15.0) | ||
| Sleep problems index I (6 items) | ||||
| LS mean change from baseline (95% CI) | –8.3 | –5.7 | –7.0 | –8.8 |
| (–16.0, –0.6)* | (–13.4, 2.0) | (–12.5, –1.5)* | (–16.1, –1.4)* | |
| LS mean difference from placebo (95% CI) | 0.5 | 3.1 | 1.8 | N/A |
| (–10.2, 11.1) | (–7.6, 13.7) | (–7.4, 10.9) | ||
| Sleep problems index II (9 items) | ||||
| LS mean change from baseline (95% CI) | –7.8 | –7.2 | –7.5 | –10.9 |
| (–15.2, –0.4)* | (–14.6, 0.2) | (–12.7, –2.3)† | (–17.9, –3.9)† | |
| LS mean difference from placebo (95% CI) | 3.1 | 3.8 | 3.4 | N/A |
| (–7.1, 13.3) | (–6.5, 13.9) | (–5.3, 12.2) | ||
| Sleep quantity, number of hours (1 item) | ||||
| LS mean change from baseline (95% CI) | 0.1 | 0.5 | 0.3 | 0.3 |
| (–0.4, 0.6) | (0.0, 1.0) | (0.0, 0.6) | (–0.2, 0.7) | |
| LS mean difference from placebo (95% CI) | –0.2 | 0.2 | 0.0 | N/A |
| (–0.8, 0.5) | (–0.5, 0.9) | (–0.6, 0.6) | ||
| Optimal sleep indicator (1 item) | ||||
| Baseline | ||||
| n | 21 | 21 | 42 | 24 |
| Yes, n (%) | 6 (28.6) | 8 (38.1) | 14 (33.3) | 4 (16.7) |
| No, n (%) | 15 (71.4) | 13 (61.9) | 28 (66.7) | 20 (83.3) |
| P value versus placebo | 0.34 | 0.11 | 0.14 | N/A |
| Week 13/ET | ||||
| n | 21 | 21 | 42 | 23 |
| Yes, n (%) | 9 (42.9) | 5 (23.8) | 14 (33.3) | 8 (34.8) |
| No, n (%) | 12 (57.1) | 16 (76.2) | 28 (66.7) | 15 (65.2) |
| P value versus placebo | 0.59 | 0.42 | 0.91 | N/A |
| Patient Impression of Change | ||||
| Week 13/ET | ||||
| n | 21 | 21 | 42 | 22 |
| Responder (score ≤3), n (%) | 19 (90.5) | 17 (81.0) | 36 (85.7) | 15 (68.2) |
| Nonresponder (score >3), n (%) | 2 (9.5) | 4 (19.0) | 6 (14.3) | 7 (31.8) |
| P value versus placebo | 0.07 | 0.39 | 0.10 | N/A |
| Patient Global Therapeutic Benefit | ||||
| Week 13/ET | ||||
| n | 21 | 21 | 42 | 22 |
| Responder (score ≤2), n (%) | 14 (66.7) | 17 (81.0) | 31 (73.8) | 14 (63.6) |
| Nonresponder (score >2), n (%) | 7 (33.3) | 4 (19.0) | 11 (26.2) | 8 (36.4) |
| P value versus placebo | 0.80 | 0.24 | 0.40 | N/A |
*
P < 0.05; † P < 0.01; ‡ P < 0.001 (analysis of covariance model with treatment group, ALP level [strata] and treatment group by ALP level interaction as factors, and baseline value as a covariate). Categorical data were analyzed using a Cochran–Mantel−Haenszel test. All P values are nominal (not adjusted for multiplicity).
Abbreviation: N/A, not applicable.