Δ4-3-oxosteroid-5β-reductase deficiency: Responses to oral bile acid therapy and long-term outcomes

Table 2 Evolution of liver function tests and international normalized ratio during oral ursodeoxycholic acid therapy in nine children¹ with ∆4-3-oxosteroid 5β-reductase deficiency

Patient No.	Starting age	Dosage (d.kg)/duration of therapy	Before UDCA administration				At the end of UDCA therapy
			TB / DB (μmol/L)	ALT/AST (U/L)	ALB (g/L)	INR	TB/DB (μmol/L)	ALT/AST (U/L)	ALB (g/L)	INR
1	2.5 mo	20 mg/3 mo	241/177	73/172	38	1.23	292/214	169/309	33	1.46
3	2 mo	30 mg/4 mo	96/64	176/183	43	1.03	8.7/5.9	51/29	44	n.a.
4	1 mo	20 mg/1.5 mo; 40 mg/1 wk	150/58	76/159	35	1.43	310/218	76/273	36	1.52
5	10 mo	18 mg/1 mo; 40 mg/4 mo	126/66	340/262	49	n.a.	6.7/2.9	8/16	42	n.a.
6	8 mo	20 mg/6 mo	221/142	235/204	32	1.24	324/252	267/584	33	2.17
7	8 mo	10 mg/2 mo	268/121	532/897	38	1.16	19/15	100/124	52	0.9
8	1 mo	16 mg/6 mo	308/131	150/187	41	1.13	273/148	448/410	28	1.36
9	1 mo	18 mg/3 mo	316/243	1240/1565	37	1.71	358/258	195/283	32	n.a.
11	3 mo	18 mg/2 mo	122/87	106/155	n.a.	1.02	85/68	83/167	39	n.a.
		Normal range	5-21/0-3.4	0-40	35-52	0.8-1.2	5-21/0-3.4	0-40	35-52	0.8-1.2

¹Patients 2, 10, and 12 did not receive ursodeoxycholic acid therapy. n.a.: Not available; UDCA: Ursodeoxycholic acid; ALT: Alanine aminotransferase; AST: Aspartic transaminase; TB: Total bilirubin; DB: Direct bilirubin; INR: International normalized ratio; ALB: Albumin.