Table 2.
Safety profile of mTOR inhibitor therapy
| Patient # | Age at start of treatment | Dosages | Comedications | AE | Vaccination status before therapy | Vaccination status during therapy |
|---|---|---|---|---|---|---|
| 1 | 19 months | 2.5 mg once daily | VGB, MAD | Recurrent URTI / LRTI Increase of cholesterol / TG | Complete | NA |
| 2 | 14 months | 2.5 mg 3 times per week | VGB, STM, VPA | None | Complete | No live vaccines |
| 3 | 15 months | 2.5 mg once daily | LTG, VGB | Transient stomatitis | Complete | NA |
| 4 | 17 months | 2 mg once daily | LEV, VPA | Transient stomatitis | Complete | NA |
| 5 | 12 months | 3 mg once daily | OXC | None | NA | No live vaccines |
| 6 | 14 days | 0.3 mg two times per week | flecainide, propranolol | None | Incomplete | NA |
| 7 | 5 months | 1 mg daily | ACTH, OXC, VGB, TMP | Transient anemia | No vaccinations | NA |
| 8 | 14 months | 2.5 mg daily | different AEDs, TMP | Recurrent URTI / LRTI Increase of cholesterol / TG | Complete | NA |
| 9 | 7 days | NA | TPM, VGB, VPA, propanolol, melatonin | Recurrent infections Reduction of phosphate Increase of cholinesterase Increase of TG Increase of LDH |
NA | Vaccinations under therapy |
| 10 | 2 months | NA | LEV, VGB, sotalol | Recurrent infections | NA | Vaccinations during 3 months pause of therapy |
| 11 | 7 days | 0.05 mg every other day | VPA, VGB, TPM, propranolol, propafenone | Worsening of infantile acne Increase of phosphate Increase of cholinesterase |
NA | Vaccinations under therapy |
| 12 | 1 month | 0.03 mg/m2twice per day | LEV, VGB | Transient neutropenia UTI |
NA | NA |
| 13 | 3 months | 0.1 mg twice per day | VGB, flecainide, metoprolol, amiodarone, metildigoxin | Transient neutropenia Recurrent URTI |
NA | NA |
| 14 | 2 days | 1.5-2 mg/m2 daily | VGB, LEV, digoxin, TMP/SMX, nystatin | Increase of cholesterol / TG Transient lymphopenia |
NA | No live vaccines |
| 15 | 2 days | 0.25 mg daily | TMP/SMX, nystatin | None | NA | No live vaccines |
| 16 | 7 months | 0.5 mg twice per day | PB, VGB | Recurrent infections | NA | No live vaccines |
| 17 | 10 months | 5 mg daily | OXC, VGB, KD | None | NA | No live vaccines |
ACTH adrenocorticotropic hormone, AE adverse event, CR cardiac rhabdomyoma, DD developmental delay, KD ketogenic diet, LDH lactate dehydrogenase, LEV levetiracetam, LRTI lower respiratory tract infection, LTG lamotrigine, MAD modified Atkins diet, NA not available, OXC oxcarbazepine, PB phenobarbital, SEGA subependymal giant cell astrocytoma, SEN subependymal nodule, SMX sulfamethoxazole, STM sulthiame, TG triglycerides, TMP trimethoprim, TPM topiramate, URTI upper respiratory tract infection, UTI urinary tract infection, VGB vigabatrin, VPA valproic acid