TABLE 1.
Clinical course and results of laboratory tests in patients with glycogen storage disease type 1b (GSD 1b) without versus with empagliflozin (EMPA)
| Patient/current age | P1/17 years | P2/13 years | P3/9 years | P4/17 months | ||||
| Without/with EMPA |
Without EMPA 1.5 years before EMPA |
EMPA ‐ Final dose: 0.3 mg/kg/24 h ‐ Treatment duration: 1.5 years |
Without EMPA 1 year before EMPA |
EMPA ‐ Final dose 0.4 mg/kg/24 h ‐ Treatment duration: 1 year |
Without EMPA 1 year before EMPA |
EMPA ‐ Final dose 0.4 mg/kg/24 h ‐ Treatment duration: 1 year |
Without EMPA 6 months before EMPA |
EMPA ‐ Final dose: 0.4 mg/kg/24 h ‐ Treatment duration: 6 months |
| Number of urgent hospitalisations due to infection | 9 | 0 | 1 | 0 | 6 | 3 | 4 | 0 |
| Number of infections requiring antibiotic therapy | 3 | 1 | 5 | 1 | 5 | 2 | 0 | 0 |
| Mean number of stools/24 h | 8–10 | 3–5 | 7–8 | 3–4 | 6–8 | 2–3 | 1–2 | 1–2 |
| Average number of days per month with mucosal lesions | 10–12 | 0 (from 1 year) | 10 | 0 (from 6 months) | 6 | 2 | 0 | 0 |
| Neutrophil count (cell/μl) a |
M: 131 R: 74–176 |
M: 440 R: 320–572 |
M: 470 R: 190–790 |
M: 1040 R: 550–1210 |
M: 625 R: 240–730 |
M: 815 R: 650–860 |
M: 890 R: 860–950 |
M: 950 R: 500–3000 |
| Mean G‐CSF dose (μg/kg/24 h) | 2.7 | 0 (from 6 months) | 2.5 | 0 (from 6 months) | 6 | 3 | 2 | 0 (from 3 months) |
| Lactate (mmol/L, N < 2.22) a |
M: 1.06 R: 1.03–1.09 |
M: 1.22 R: 1.14–1.30 |
M: 2.94 R: 1.11–5.88 |
M: 2.48 R: 1.93–3.28 |
M: 1.39 R: 1.05–2.15 |
M: 1.23 R: 1.04–1.4 |
M: 3.77 R: 2.66–5.66 |
M: 5.55 R: 5.49–6.1 |
| Uric acid (μmol/L, N < 416) a |
M: 371.7 R: 303–475.8 |
M: 280.75 R: 243.87–327.14 |
M: 713.8 b R: 374.72–933.84 |
M: 386.62 c R: 333.1–422.31 |
M: 401.5 R: 291.5–499 |
M: 237.92 R: 196.3–261.7 |
M: 410.4 R: 83.3–434 |
M: 136.8 R: 107–166.54 |
| Triglycerides (mmol/L, N < 1.71) a |
M: 0.71 R: 0.6–0.75 |
M: 0.68 R: 0.63–0.74 |
M: 8.74 R: 7.49–1.99 |
M: 4.69 d R: 3.37–14.31 |
M: 1.15 R: 0.81–1.93 |
M: 1.19 R: 1.0–1.31 |
M: 7.34 R: 2.32–12.11 |
M: 7.92 R: 7.91–7.92 |
Abbreviations: G‐CSF, granulocyte colony‐stimulating factor; M, median; N, normal; R, range.
Results of laboratory tests in patients without infection.
Patient additionally treated with allopurinol.
Patient additionally treated with febuxostat.
Patient additionally treated with fenofibrate.