TABLE 1.
Baseline demographics and characteristics
| Characteristic | Maralixibat cohort (n=84) | GALA control cohort (n=469) | P value |
|---|---|---|---|
| Sex | |||
| Male | 49 (58.3) | 274 (58.4) | 0.99 |
| Female | 35 (41.7) | 195 (41.6) | |
| Age at baseline, y | |||
| Median (Q1–Q3) | 5.6 (2.7–9.9) | 4.3 (2.2–9.6) | 0.078 |
| Year of birth | |||
| Median (Q1–Q3) | 2009 (2005–2012) | 2009 (2004–2013) | 0.25 |
| Region | |||
| Europe | 41 (48.8) | 229 (48.8) | 0.95 |
| North America | 34 (40.5) | 195 (41.6) | |
| Australia | 9 (10.7) | 45 (9.6) | |
| Variant | |||
| JAGGED1 | 81 (97.6) | 330 (95.1) | 0.55a |
| NOTCH2 | 2 (2.4) | 17 (4.9) | |
| Other/unknown | 1 (0.2) | 37 (9.6) | |
| Total bilirubin, mg/dL | |||
| Median (Q1–Q3) | 3.15 (1.00–8.15) | 1.99 (0.60–11.52) | 0.39 |
| <2 | 37 (44.0) | 235 (50.1) | 0.31 |
| ≥2 | 47 (56.0) | 234 (49.9) | |
| GGT, log10×ULN | |||
| Median (Q1–Q3) | 1.25 (0.93–1.44) | 1.24 (0.93–1.52) | 0.58 |
| GGT, ×ULN | |||
| <3 | 3 (3.6) | 6 (1.3) | 0.14a |
| ≥3 | 81 (96.4) | 463 (98.7) | |
| ALT, U/L | |||
| Median (Q1–Q3) | 145 (94–207) | 130 (75–203) | 0.12 |
| sBA,b µmol/L | |||
| Median (Q1–Q3) | 200 (81–371) | 125 (39–260) | 0.0033 |
All data are n (%) unless otherwise stated.
a
Due to >20% of the cells having expected counts <5, chi-square results may be invalid, and the Fisher exact test was used instead.
b
Baseline sBA data were available for 73 patients in the GALA control cohort. Approximately 85% of the sBA values were not available in the GALA clinical research database as frequent sBA measurement is not part of clinical practice.
Abbreviations: ALT, alanine aminotransferase; GALA, Global ALagille Alliance; GGT, gamma-glutamyl transferase; Q1, first quartile; Q3, third quartile; sBA, serum bile acid; ULN, upper limit of normal.