Table 7. . Sensitivity analyses estimated annualized follow-up rates of healthcare resource utilization events.
| Outcome variable | Main study sample plus male patients treated with golodirsen | Main study sample plus female patients treated with eteplirsen | Main study sample excluding those aged >28.1 years | |||
|---|---|---|---|---|---|---|
| Treated mean (SD) (n = 828) | Control mean (SD) (n = 828) | Treated mean (SD) (n = 784) | Control mean (SD) (n = 784) | Treated mean (SD) (n = 766) | Control mean (SD) (n = 766) | |
| Care-adjusted hospital encounter | 4.19 (0.43)† | 5.87 (0.67) | 4.17 (0.45)† | 6.05 (0.75) | 4.11 (0.44)† | 6.19 (0.76) |
| Care-adjusted ER | 0.63 (0.07)† | 0.88 (0.09) | 0.62 (0.07)† | 0.90 (0.09) | 0.60 (0.07)† | 0.90 (0.09) |
| Assisted ventilation | 1.80 (0.18)† | 3.64 (0.54) | 1.86 (0.19)† | 3.04 (0.53) | 1.32 (0.15)† | 2.62 (0.45) |
| Cardiac management | 1.39 (0.10)† | 1.81 (0.12) | 1.38 (0.10)† | 1.75 (0.12) | 1.38 (0.10)† | 1.76 (0.13) |
| Cough assist device | 0.70 (0.08) | 0.84 (0.12) | 0.67 (0.08) | 0.76 (0.10) | 0.68 (0.08) | 0.78 (0.10) |
| ER days | 0.36 (0.05)† | 0.51 (0.06) | 0.36 (0.04)† | 0.53 (0.06) | 0.35 (0.04)† | 0.53 (0.06) |
| Hospital days | 1.96 (0.17)† | 2.69 (0.23) | 1.89 (0.18)† | 2.85 (0.35) | 1.87 (0.18)† | 2.95 (0.36) |
| Care-adjusted ICU | 0.22 (0.04) | 0.37 (0.11) | 0.25 (0.05) | 0.27 (0.07) | 0.22 (0.04) | 0.27 (0.07) |
| ICU days | 0.20 (0.04) | 0.32 (0.08) | 0.22 (0.04) | 0.24 (0.06) | 0.19 (0.04) | 0.25 (0.06) |
| Motorized wheelchair | 0.95 (0.09) | 0.99 (0.07) | 0.95 (0.09) | 0.95 (0.07) | 0.95 (0.10) | 0.94 (0.07) |
| Pulmonary management | 1.37 (0.18)† | 2.29 (0.32) | 1.32 (0.18)† | 1.97 (0.28) | 1.09 (0.14)† | 1.95 (0.28) |
| Scoliosis | 1.37 (0.34) | 1.85 (0.61) | 0.99 (0.21) | 1.24 (0.33) | 0.99 (0.22) | 1.25 (0.34) |
| Tracheostomy | 0.85 (0.15)† | 8.13 (4.29) | 0.89 (0.17)† | 6.45 (3.45) | 0.59 (0.12)† | 4.67 (2.09) |
†
Indicates statistical significance.
ER: Emergency room; ICU: Intensive care unit; SD: Standard deviation.