Understanding the role of SGLT2 inhibitors in glycogen storage disease type Ib: the experience of one UK centre

Table 1 Clinical and biochemical data at baseline and on treatment

Patient ID	1		2		3		4		5		6		7		8		Median
Current age (years)	7.0		15.17		8.58		4.33		15.42		15.83		1.5		6.75		7.79
Time on treatment (years)	1.67		1.25		1.83		2.33		0.75		1.83		0.58		2.08		1.75
Daily dose (mg/kg htwt)	0.18		0.09		1.28		0.38		0.24		0.09		0.21		0.24		0.22
Empagliflozin treatment	Pre	Post	Pre	Post	Pre	Post	Pre	Post	Pre	Post	Pre	Post	Pre	Post	Pre	Post	Pre	Post
Weight (Z-score)	− 0.54	− 0.09	2.04	1.9	0.93	0.9	0.84	0	− 1.4	− 0.76	0.1	0.05	1.1	1.34	− 0.34	− 0.45	0.47	0.03
Height (Z-score)	− 0.75	− 0.07	0.43	0.4	− 1.33	− 1.9	− 1.3	− 1	− 3.9	− 3.9	− 0.96	− 0.39	− 1.2	− 0.92	− 0.96	− 1.08	− 1.08	− 0.96
BMI (Z-score)	− 0.1	− 0.11	2.1	1.97	2	2.2	2.3	0.68	0.95	1.56	0.64	0.43	N/A	N/A	0.39	0.23	0.95	0.68
ANC (× 10⁹/L)	2	1	0.1	0.6	1.4	0.7	0.2	0.6	N/A	1.7	0.1	0.6	0.4	0.4	0.4	0.5	0.4	0.6
Haemoglobin (g/L)	107	126	90	123	101	116	105	130	N/A	144	110	140	96	113	103	126	103	126
Iron (μmol/L)	15.3	14.7	3.1	10.7	3.7	8.7	3.1	10.5	N/A	11	8.6	14.5	8.6	6.2	4.7	8.4	4.7	10.6
Urate (μmol/L)	0.47	0.16	0.33	0.25	0.41	0.38	0.39	0.25	N/A	0.16	0.35	0.25	0.35	0.26	0.27	0.14	0.35	0.25
Triglycerides (mmol/L)	6.26	2.81	0.96	0.8	2.72	6.3	2	5	N/A	2.53	1.37	1.49	3.16	2.35	1.92	2.6	2	2.57
PUCAI score	10	10	35	10	55	40	30	0	N/A	N/A	20	5	N/A	N/A	5	10	25	10
Daily GCSF (μg/kg htwt)	1.75	0	N/A	N/A	3.46	0	1.77	0	N/A	N/A	N/A	N/A	N/A	N/A	N/A	N/A	1.77	0

ANC: absolute neutrophil count; GCSF: granulocyte colony-stimulating factor; PUCAI: Pediatric Ulcerative Colitis Activity Index—Severe > 65, moderate 35–64, mild 10–34, in remission < 10. Haemoglobin—normal range < 110 g/L under 6 years, < 115 g/L > 6. Iron—normal range 14-25 μmol/L). Urate—normal range 120–430 μmol/L. Triglycerides – normal range 0.6–1.9 mmol/L

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