Clinical Efficacy Evaluation of Sirolimus in Congenital Hyperinsulinism
Table 2
Assessment of response to treatment with sirolimus and outcome.
Patient no.
Age of starting sirolimus
Dose of sirolimus of starting (mg/m2)
Final dose of sirolimus (mg/m2)
Response to treatment with sirolimus
Duration of treatment
Side effects
Outcomes and follow-up
1
8 weeks
0.5
0.2 and, then, discontinued
Discontinued dextrose therapy after 10 days
3 months
None
Continue sirolimus therapy for 3 months, no hypoglycemic episodes for one year after tapering sirolimus. Eurodevelopmental growth compatible with the age
2
14 weeks
0.5
1.5
Discontinued dextrose therapy after 15 days and octreotide after 20 days
More than one year until the present
Mild transient elevation of liver aminotransferase levels
Reduced hypoglycemic episodes for 6 months after receiving sirolimus, continued plus diazoxide, and developmental delay
3
4 weeks
0.5
Discontinued dextrose therapy after 17 days and diazoxide/octreotide after 20–24 days
More than one year (continued until now)
None
Reduced hypoglycemic episodes for 6 months after receiving sirolimus. In 14 months of age, neurodevelopmental growth compatible with the age
4
6 weeks
0.5
1–1.5
Recurrent attacks of hypoglycemia and seizure, discontinued after 6 months
6 months
None
Recurrent hypoglycemia ended up in pancreatectomy after 6 months of receiving sirolimus, after pancreatectomy (focal hyperplasia in histologic findings) diazoxide therapy with minimal dose continued
5
3 weeks
0.5
0.5–0.7
Discontinued dextrose therapy after 25 days, continued diazoxide 15 mg/kg/d and octreotide 10 mcg/kg/d
8 months (discontinued follow-up after 8 months)
None
Reduced hypoglycemic episodes for 6 months after receiving sirolimus (no seizure or poor feeding) adequate developmental growth
6
5 years
0.5
0.5
Continued diazoxide (10–15 mg/kg/d)
More than one year, continued until now
None
After surgery, the hypoglycemia episode was controlled by sirolimus plus diazoxide. No attacks of seizures, reduced hypoglycemia less than 1 time in the week
