Table 3.
Onset of Management With Sirolimus in Patients With CHI
| Onset of Sirolimus Therapy | |
|---|---|
| Age (mo) sirolimus started, median (range) | 3.87 (1.5–198), 10 started during first trimester of life |
| Weight at sirolimus start, median (range), SDS | 0.81 g (0.05–3.31) |
| Starting dose of sirolimus, median (range), mg/m2/d | 0.96 (0.5–4) |
| Total number of mo receiving sirolimus, median (range) | 21.5 (3–59)a |
| Starting level of sirolimus, median (range), ng/mL | 4.7 (2–14.5) |
| Other medications at time of starting sirolimus | Glucagon infusion (5–10 μg/kg/h): 10 patients Diazoxide (6.2–18 mg/kg/d): 5 patients Octreotide (20–40 μg/kg/d): 17 patients |
| IV and feeds while on sirolimus | 8 IV dextrose with/without continuous feeds 6 continuous enteral feeds exclusively 8 frequent feeds exclusively |
| Maximum dose sirolimus, median (range), mg/m2/d | 6.5 (1.76–17) |
| Level of sirolimus at maximum dose, median (range), ng/mL | 16.35 (5.1–46.1) |
| Response to sirolimus | Partial response: 20 (91%) |
| Complete response: 1 (4.5%) | |
| Unresponsive: 1 (4.5%) | |
| Genetics vs response | Partial response: 5 compound heterozygous ABCC8; 5 autosomal dominant heterozygous ABCC8; 3 homozygous ABCC8; 4 negative genetics for known CHI genes; 1 BWS; 1 Rubinstein-Taybi syndrome; 1 GCK mutation |
| Complete response: 1 autosomal dominant heterozygous ABCC8 | |
| Unresponsive: 1 homozygous ABCC8 | |
a
Two stopped it transiently (1 from concerns for infection/parainfluenza, decision taken by medical team; 1 unspecified infections, decision taken by parents) and restarted; total number of months on sirolimus included.