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Table 2 Safety profile of mTOR inhibitor therapy

From: Safety and efficacy of mTOR inhibitor treatment in patients with tuberous sclerosis complex under 2 years of age – a multicenter retrospective study

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

  1. 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