Table 2.
Adherence to risdiplam among patients with SMA overall, by SMA type, and by age groups
| Adherence | Overall (N = 86) |
SMA type | Age groupa | |||||||
|---|---|---|---|---|---|---|---|---|---|---|
| Type 1 (n = 1) |
Type 2 (n = 18) |
Type 3 (n = 47) |
Type 4 (n = 20) |
0–2 years (n = 3) |
3–5 years (n = 10) |
6–12 years (n = 17) |
13–17 years (n = 15) |
≥ 18 years (n = 41) |
||
| PDC,b mean ± SD | 0.89 ± 0.18 | 0.97 ± NA | 0.89 ± 0.18 | 0.90 ± 0.19 | 0.88 ± 0.19 | 0.96 ± 0.04 | 0.92 ± 0.15 | 0.85 ± 0.24 | 0.88 ± 0.23 | 0.91 ± 0.16 |
| PDC,b median (IQR) | 0.98 (0.89, 1.00) | 0.97 (NA) |
0.98 (0.91, 1.00) |
0.98 (0.88, 1.00) | 0.96 (0.86, 0.99) | 0.97 (0.91, 1.00) | 0.98 (0.91, 0.99) | 0.97 (0.86, 1.00) | 0.97 (0.88, 1.00) | 0.98 (0.91, 0.99) |
| Adherent patients (PDC ≥ 0.80), n (%) | 72 (83.7%) | 1 (100.0%) | 15 (83.3%) | 41 (87.2%) | 15 (75.0%) | 3 (100.0%) | 9 (90.0%) | 13 (76.5%) | 12 (80.0%) | 35 (85.4%) |
IQR, interquartile range; NA, not applicable; PDC, proportion of days covered; SD, standard deviation; SMA, spinal muscular atrophy
aAge was measured at the index date
bPDC was calculated as the total number of days during which a patient had risdiplam available based on the reported days of supply of risdiplam beginning from the date of the first dispensing of risdiplam until their latest dispensing (plus days of supply), divided by the length of the adherence assessment period (i.e., study period) in days