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Table 4 Adverse effects with dose modification over time during the treatment period

From: Effectiveness and safety of everolimus treatment in patients with tuberous sclerosis complex in real-world clinical practice

 

1st year

All grades

n = 64

n/%

Grade

3–4

n = 64

n/%

2st years

All grades

n = 54

n/%

Grade

3–4

n = 54

After 2sd years

All grades

n = 41

n/%

Grade

3–4

n = 41

n/%

Whole period

(all grades and non-graded)

n = 64

n/%

Grade

3–4

n/N/%

Dose modification

whole period

n = 64

n/N/%

Infections

         

 Stomatitis/mouth ulceration

28/44

1/2

18/33

 

17/42

 

40/63

2/35/6

10/40/25

 Upper respiratory tract infection

19/30

1/2

11/20

 

3/7

 

24/38

 

1/24/4

 Rash

9/14

 

2/4

 

5/12

 

17/27

 

3/17/18

 Pyrexia

10/16

4/6

9/17

3/6

1/2

1/2

13/20

4/12/33

4/13/31

 Skin infection included erythema nodusum

7/11

1/2

 

1/2

5/12

1/2

11/17

3/12/25

4/11/37

 Nausea/vomiting

7/11

 

1/2

 

3/7

 

9/14

1/9/11

2/9/22

 Diarrhoea

6/10

1/2

2/4

 

1/2

 

9/14

 

1/9/11

 Dermatitis acne

4/6

 

6/11

 

3/7

 

8/13

 

2/8/25

 Pneumonia

5/8

5/8

3/6

2/4

5/12

4/10

8/13

8/8/100

5/8/63

 Gastroenteritis

1/2

 

3/10

 

1/2

 

7/11

 

3/7/43

 Urinary tract and/or pyelonephritis infections

3/5

 

4/7

 

2/5

 

6/9

1/6/17

2/6/33

 Otitis

4/6

 

1/2

 

2/5

 

5/8

  

 Genital infection*

    

1/2

 

3/5

  

 Immune deficiency

1/2

1/2

2/4

2/4

1/2

1/2

2/3

2/64/3

 

 Abscess

1/2

     

1/2

  

 Infected polyp

    

1/2

 

1/2

  

 Mononucleosis

1/2

     

1/2

 

1/1/100

 Hepatitis B virus

  

1/2

   

1/2

  

 Appendicitis

1/2

     

1/2

  

 Acute encephalitis

1/2

     

1/2

1/64/2

1/1/100

 Chronic osteomyelitis

  

1/2

 

1/2

1/2

1/2

1/64/2

 

Other adverse effects

         

 Tiredness

9/14

1/2

4/7

1/2

5/12

 

14/22

2/64/14

1/14/7

 Pruritus

  

1/2

   

7/11

  

 Amenorrhea or irregular menses n/N/%

4/21/19

 

1/21/5

   

6/21/29

  

 Cysts **

2/4

 

3/6

2/4

  

6/9

2/64/14

 

 Constipation

3/5

 

1/2

   

5/8

  

 Headache

      

2/3

  

 Diabetes mellitus

1/2

     

1/2

 

1/1/100

 Cerebral oedema

1/2

1/2

    

1/2

1/64/2

1/1/100

Laboratory abnormalities

         

 Any lab abnormality***

35/55

 

29/4

 

16/39

 

46/72

  

 Increased cholesterol

25/39

 

22/41

 

18/44

 

26/41

 

2/26/8

 Hypertriglyceridemia

13/20

 

8/15

 

8/20

 

19/30

  

 Neutropenia

7/11

 

6/11

2/4

4/10

3/7

7/11

4/64/6

1/7/14

 Leucopoenia

13/25

1/2

7/13

1/2

6/15

1/2

16/25

2/64/4

3/16/19

 Anaemia

12/19

 

6/11

 

5/12

 

17/27

  

 Hyperglycaemia

1/2

1/2

2/4

 

3/7

 

4/6

1/64/2

1/4/25

 Hypophosphatemia

5/8

 

1/2

 

2/5

 

6/9

 

1/6/17

 Thrombocytopenia

4/7

1/2

2/4

   

4/6

1/64/2

1/4/25

 ASAT

1/2

     

1/2

  

 ALAT

2/3

1/2

    

2/3

1/64/2

1/2/50

 GGT

2/3

 

2/4

 

1/2

 

3/5

  
  1. *Genital infection (vaginal infection n = 2, testes infection n = 1)
  2. **Cysts: (ovarian, clitoris, pilonidal)
  3. ***According to the National Cancer Institute Common Terminology Criteria version 5
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172