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Table 2 Characteristics of deceased patients (34 patients died in total, 23 of them prior to ERT)

From: Survival and associated factors in 268 adults with Pompe disease prior to treatment with enzyme replacement therapy

 

34 (All deceased patients)

23 (died prior to ERT)

Female, n (%)

16 (47)

12 (52)

Median age at study entry, years (range)

54 (20-75)

51 (20-75)

Median age at death, years (range)

56 (23-78)

55 (23-77)

Median age at diagnosis, years (range)

42 (13-66)

42 (13-59)

Median disease duration, years (range)

14 (2-27)

16 (2-27)

Age at diagnosis in categories of 15 years, n (%)

  

   0-15 years

3 (9)

3 (13)

   16-30 years

6 (18)

3 (13)

   31-45 years

13 (38)

10 (44)

   46-60 years

10 (29)

7 (30)

   >61 years

2 (6)

0

Nationality, n (%)

  

   Netherlands

9 (27)

5 (22)

   Germany

4 (12)

4 (17)

   US

13 (38)

8 (35)

   UK

4 (12)

3 (13)

   Australia

1 (3)

0

   Canada

2 (6)

2 (9)

   Other

1 (3)

1 (4)

Disability level at study entry, n (%)

  

   No wheelchair use or respiratory support ª

4 (12)

4 (17)

   Wheelchair use

6 (18)

4 (17)

   Use of respiratory support

7 (21)

4 (17)

   Both wheelchair use and respiratory support

17 (50)

11 (48)

Median RHS score* at study entry (range)

23 (9-36) (n = 33)

22 (9-36)

  1. Continuous variables are expressed as median (range). Categorical variables are expressed as n (%). ª 'Respiratory support' includes partial and fulltime, invasive and non-invasive respiratory support. * RHS assesses the level of participation/handicap; score varies between 9 (severe participation restrictions) and 36 (no participation restrictions.