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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.