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