Table 1 Patient Characteristics of 268 untreated adult patients with Pompe disease
Female, n (%) | 141 (53) |
|---|---|
Median age at study entry, years (range) | 48 (19-79) |
Median age at diagnosis, years (range) | 38 (1-68) |
Number of patients diagnosed in age categories of 15 years, n (%) | |
<15 years | 22 (8) |
16-30 years | 59 (22) |
31-45 years | 115 (43) |
46-60 years | 61 (23) |
>61 years | 11 (4) |
Nationality, n (%) | |
Netherlands | 99 (37) |
Germany | 48 (18) |
US | 69 (26) |
UK | 20 (8) |
Australia | 13 (5) |
Canada | 9 (3) |
Other | 10 (4) |
Median disease duration at entry, years (range) | 9 (0-32) |
Median follow up time, years (range) | 3.5 (0.02-7) |
Disability level at study entry, n (%) | |
No wheelchair use or respiratory supportª | 127 (47) |
Wheelchair use | 34 (13) |
Use of respiratory support | 39 (15) |
Both wheelchair use and respiratory support | 68 (25) |
Median RHS score* at study entry (range) (n = 258) | 27 (9-36) |