Table 3.
Relative risk of death or invasive ventilation according to average relative dose of alglucosidase alfa
| Person Years | N Deaths | Adjusted HRa | 95% CI | p-value | |
|---|---|---|---|---|---|
| Outcome: Risk of death | |||||
| Average relative dose over time, continuous (range: > 0 to 4.0 times label dose)b | |||||
| Per 1-unit increase in average dose | 1609 | 88 | 0.40 | 0.22, 0.73 | 0.0030 |
| Categories of average dose over timeb | |||||
| Below label dose | 23 | 8 | 7.16 | 2.28, 22.48 | 0.0008 |
| Label dose | 854 | 52 | 1.00 (reference) | – | – |
| Between label and double dose | 339 | 14 | 0.87 | 0.41, 1.82 | 0.7020 |
| Double dose | 249 | 12 | 0.83 | 0.38, 1.82 | 0.6340 |
| Above double to quadruple dose | 144 | 2 | 0.10 | 0.01, 0.82 | 0.0312 |
| Outcome: Risk of death or invasive ventilation | |||||
| Average relative dose over time, continuous (range: > 0 to 4.0 times label dose)b | |||||
| Per 1-unit increase in average dose | 1237 | 97 | 0.48 | 0.28, 0.84 | 0.0100 |
| Categories of average relative dose over timeb | |||||
| Below label dose | 21 | 5 | 1.59 | 0.44, 5.73 | 0.4810 |
| Label dose | 633 | 60 | 1.00 (reference) | – | – |
| Between label and double dose | 284 | 16 | 0.70 | 0.34, 1.46 | 0.3405 |
| Double dose | 189 | 16 | 0.96 | 0.47, 2.00 | 0.9224 |
| Above double to quadruple dose | 110 | 0 | 0.00 | NE, NE | 0.9895 |
CRIM, cross-reactive immunological material; EOW, every other week; ITI, immune tolerance induction; NE, not evaluable
aModels are adjusted for age (as time scale), age at first treatment, sex, and CRIM/ITI group
bAverage dose received over time from first treatment, updated over time, measured in multiples of the label dose, ranging from > 0 to 4 times label dose. Average dose categories: 'Below label dose' is average dose < 0.95;'Label dose' is from 0.95 to < 1.05; 'Between label and double dose' is from 1.05 to < 1.75; 'Double dose' is 1.75 to < 2.25; 'Above double to quadruple dose' is 2.25 to 4.0