Table 1 Characteristics of patients participating in the analysis of the effect of ERT on the use of a wheelchair and respiratory support, and of all patients included in the IPA/Erasmus MC Pompe survey
From: Enzyme replacement therapy reduces the risk for wheelchair dependency in adult Pompe patients
Included in analyses | All participants Survey (n = 458) | ||
|---|---|---|---|
Risk of using a wheelchair (n = 189) | Risk of using respiratory support (n = 177) | ||
Female, n (%) | 98 (52%) | 109 (62%)* | 230 (50%) |
Median age at entry into the survey, years (range) | 47 (18–75) | 46 (18–73) | 47 (2–81) |
Median age at diagnosis, years (range) | 39 (3–72) | 39 (0–72) | 38 (0–72) |
Median disease duration at entry, years (range) | 5 (0–39)* | 5 (0–31)* | 7 (0–39) |
Country of residence, n (%) | * | * | |
Netherlands | 78 (41%) | 87 (49%) | 134 (29%) |
United Kingdom | 9 (5%) | 9 (5%) | 36 (8%) |
United States | 44 (23%) | 36 (20%) | 124 (27%) |
Germany | 26 (14%) | 25 (14%) | 66 (14%) |
Other | 32 (17%) | 20 (11%) | 98 (21%) |
ERTa, n (%) | 126 (67%) | 125 (71%) | 319 (70%) |
Median age start ERT, years (range) | 48 (13–77) | 49 (13–74) | 47 (3–77) |
Median follow-up duration, years (range) | 5 (1–14) | 6 (1–14) | N.A. |
Events during follow-up, n (%) | 46 (24%) | 48 (27%) | N.A. |
Median age at event, years (range) | 52 (21–76) | 50 (24–73) | N.A. |