Table 1 Summary of all four patients with medical conditions, treatment plan and observed side effects
Clinical characteristics | Patient #1 | Patient #2 | Patient #3 | Patient #4 |
|---|---|---|---|---|
Age at presentation | hypoglycaemia DOL 1 | hypoglycaemia DOL 1 | hypoglycaemia at birth | hypoglycaemia DOL1 |
Mutation | homozygous c.563A > G (ABCC8) | homozygous c.1176G > C (ABCC8) | mosaic paternal UPD 11 | mosaic paternal UPD 11 |
Associated syndromic condition | no | no | yes, BWS | yes, BWS |
Treatment prior to LAN-ATG | DZ p.o. 10 mg/kg/d OCT sc. 43 μg/kg/d GLUC sc. max. 50 μg/kg/d carbohydrates 154 g/d p.o. | DZ p.o. 14 mg/kg/d OCT sc. 36 μg/kg/d GLUC sc. max. 84 μg/kg/d carbohydrates 100 g/d p.o. | DZ p.o. 10 mg/kg/d carbohydrates 69 g/d p.o. | DZ p.o. 10 mg/kg/d OCT sc. 40 μg/kg/d carbohydrates 78 g/d p.o. and i.v. |
LAN-ATG treatment | 30–90 mg/month (4.8–6.9 mg/kg) | 30–90 mg/month (5–7.5 mg/kg) | 1× 30 mg (5.3 mg/kg) | 60 mg/14d-90 mg/month (9.5–13.6 mg/kg) |
Carbohydrates after LAN-ATG | 64.2Â g/d p.o. | 63.6Â g/d p.o. | 63Â g/d p.o. | 78Â g/d p.o. |
Duration of LAN-ATG treatment | 25Â months | 23Â months | single injection | 3Â months |
Additional medication | none | none | none | DZ 8Â mg/kg/d |
Side effects of LAN-ATG | nodules at injection site pre-existing cholelithiasis | nodules at injection site pre-existing biliary sludge | none | nodules at injection site |