Cox proportional hazards regression analysis assessing the time dependence of risk factors for clinical events*
| Years on agalsidase β | |||||||||
|---|---|---|---|---|---|---|---|---|---|
| Model 1: 0–0.5 year | Model 2: >0.5–5 years | Model 3: >0–5 years | |||||||
| HR | 95% CI | p Value | HR | 95% CI | p Value | HR | 95% CI | p Value | |
| Pre-ERT event: yes (vs no) | 1.1 | 0.6 to 2.0 | 0.81 | 1.8 | 1.2 to 2.7 | <0.01 | 1.5 | 1.1 to 2.1 | 0.02 |
| Age ≥40 years at first ERT (vs age <40 years) | 4.4 | 2.2 to 8.7 | <0.01 | 2.5 | 1.7 to 3.8 | <0.01 | 2.9 | 2.1 to 4.2 | <0.01 |
| Male (vs female) | 1.9 | 1.1 to 3.4 | 0.03 | 1.5 | 1.0 to 2.1 | 0.06 | 1.6 | 1.1 to 2.2 | <0.01 |
*Three models were run to assess if the incidence of events according to the above factors was time-dependent: Model 1 examined risk factors within the first 6 months; Model 2 examined risk factors within 6 months to 5 years; and Model 3 examined risk factors for the entire analysis period of up to 5 years.
ERT, enzyme replacement therapy.