TABLE 2.

Probability of EFS and TFS in children with Alagille syndrome treated with maralixibat according to the threshold of predictor

	Variable
	Change in pruritusa from baseline to week 48		Total bilirubin levels at week 48		sBA levels at week 48
	Improved: > 1 point reduction n = 46	Worse: ≤ 1 point reduction n = 30	Improved: < 6.5 mg/dL n = 52	Worse: ≥ 6.5 mg/dL n = 24	Improved: < 200 µmol/L n = 56	Worse: ≥ 200 µmol/L n = 18
6-y EFS (%)	88	57	90	43	85	49
C-statistic	0.70		0.82		0.74
p b	0.0046		<0.0001		0.0010
6-y TFS (%)	93	57	94	42	90	49
C-statistic	0.77		0.85		0.79
p b	0.0007		< 0.0001		0.0001

Note: “improved” or “worse” refers to the predicted outcomes for either EFS or TFS.

^aAs assessed by ItchRO(Obs).

^b p-value from the log-rank test.

Abbreviations: EFS, event-free survival; ItchRO(Obs), Itch-Reported Outcome (Observer); sBA, serum bile acids; TFS, transplant-free survival.