Table 3.
Risk of death for 283 adults with Pompe disease applying time-dependent cox regression*
| |
Model 1a† |
||
|---|---|---|---|
| Time-dependent cox regression model | HR | 95% CI | P Value |
| ERT‡ |
0.41 |
0.19 to 0.87 |
0.02 |
| Age (in quartiles) |
|
|
0.14 |
| <37 years (ref) |
1 |
|
|
| 37-48 years |
1.26 |
0.38 to 4.12 |
0.71 |
| 48-57 years |
1.42 |
0.44 to 4.61 |
0.56 |
| ≥57 years |
2.57 |
0.86 to 7.72 |
0.09 |
| Sex |
1.01 |
0.55 to 1.87 |
0.98 |
| Disease severity |
|
|
0.001 |
| No wheelchair use or respiratory support ‡ |
1 |
|
|
| Wheelchair use |
2.87 |
0.98 to 8.36 |
0.05 |
| Use of respiratory support |
2.05 |
0.62 to 6.77 |
0.24 |
| Both wheelchair use and respiratory support |
5.32 |
2.25 to 12.56 |
<0.001 |
| Country of residence |
|
|
0.13 |
| Netherlands (ref) |
1 |
|
|
| United Kingdom |
1.35 |
0.46 to 3.95 |
0.58 |
| United States |
2.14 |
1.01 to 4.55 |
0.05 |
| Germany |
0.62 |
0.19 to 1.95 |
0.41 |
| Other§ | 1.23 | 0.42 to 3.63 | 0.70 |
* ERT denotes enzyme replacement therapy and HR hazard ratio.
† Intent-to-treat approach with ERT, age categories, and disease severity as time-dependent covariates.
‡ Values for ERT were derived after adjustment for age, sex, disease severity, and country of residence.
§ Including patients from Australia and Canada.
‡Respiratory support includes partial and full-time invasive and non-invasive respiratory support.