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Table 2 Patient demographics

From: Evolution of mobility, pain/discomfort, self-care, and mental health in patients with alpha-mannosidosis: an international caregiver and patient survey

  

ERT

HSCT

UP

All

Pediatrics

< 16 years old

Adults

≥ 16 years old

< 5 years

on ERT

≥ 5 years

on ERT

All

Pediatrics

< 16 years old

Adults

≥ 16 years old

All

Pediatrics

< 16 years old

Adults

≥ 16 years old

N

26

6

20

13

13

7

4

3

18

2

16

Sex

Males

15

5

10

9

6

3

1

2

8

0

8

Females

11

1

10

4

7

4

3

1

10

2

8

Age at the time of survey

Median

21.1

12.7

29.8

18.6

29.3

13.6

12.2

20.7

29.2

11.8

30.0

Mean ± SD

24.9 ± 10.8

12.8 ± 2.4

28.5 ± 9.6

22.0 ± 10.8

27.8 ± 10.3

15.9 ± 4.9

12.4 ± 0.9

20.7 ± 3.4

26.4 ± 7.8

11.8 ± 0.1

28.3 ± 6.1

Range

10.3–45.7

10.3–15.7

16.3–45.7

10.3–42.2

13.6–45.7

11.4–24.2

11.4–13.6

17.3–24.2

11.7–38.4

11.7–11.8

17.3–38.4

Age symptoms were first noticed

Median

1.5

2.4

1.3

2.0

1.1

0.7

1.8

0.5

2.4

6.5

2.3

Mean ± SD

2.6 ± 4.5

2.6 ± 2.2

2.7 ± 5.0

2.2 ± 1.5

3.0 ± 6.3

2.4 ± 4.0

3.7 ± 5.2

0.6 ± 0.3

5.8 ± 7.2

6.5 ± 5.6

5.7 ± 7.6

Range

0.0–23.5

0.0–5.1

0.0–23.5

0.5–5.1

0.0–23.5

0.0–11.3

0.0–11.3

0.3–1.0

0.3–24.1

2.6–10.5

0.3–24.1

Age at diagnosis

Median

6.6

7.7

6.1

7.0

6.2

2.8

4.4

1.9

10.3

8.3

10.3

Mean ± SD

8.3 ± 7.2

7.3 ± 2.1

8.6 ± 8.1

8.5 ± 7.3

8.0 ± 7.3

3.8 ± 3.0

5.0 ± 3.6

2.1 ± 0.6

11.3 ± 7.0

8.3 ± 3.1

11.7 ± 7.3

Range

0.0–27.2

4.5–9.6

0.0–27.2

1.3–27.2

0.0–25.2

1.3–9.8

1.3–9.8

1.6–2.8

2.6–24.1

6.2–10.5

2.6–24.1

Age treatment started

Median

17.0

8.4

21.5

16.1

21.0

3.2

5.1

2.8

   

Mean ± SD

18.9 ± 9.7

9.2 ± 3.3

21.8 ± 9.1

19.8 ± 10.0

17.9 ± 9.7

4.0 ± 2.4

4.9 ± 3.0

2.8 ± 0.8

   

Range

5.3–37.5

5.3–14.8

5.7–37.5

7.7–37.5

5.3–34.8

1.6–7.8

1.6–7.8

2.0–3.6

   

Treatment length

Median

5.3

2.0

5.9

1.9

10.2

10.4

7.6

17.8

   

Mean ± SD

6.0 ± 4.4

3.6 ± 3.8

6.7 ± 4.4

2.2 ± 1.4

9.9 ± 2.5

12.0 ± 6.5

7.5 ± 3.1

17.9 ± 4.2

   

Range

0.3–12.5

0.5–10.3

0.3–12.5

0.3–4.7

5.8–12.5

4.3–22.2

4.3–10.4

13.8–22.2

   
  1. Treatment length varies for all patients irrespective of treatment group. Hence, data from 5 years ago to now correspond to different follow-up periods in relation to start of treatment of the patients these caregivers care for.
  2. ERT = enzyme replacement therapy; HSCT = hematopoietic stem cell transplantation; SD = standard deviation; UP = untreated patients.