Table 3 Treatment-emergent AEs by treatment
From: 10th European Conference on Rare Diseases & Orphan Products (ECRD 2020)
| Â | Treatment received | Â | ||||
|---|---|---|---|---|---|---|
|  | Patients receiving > 1 active therapy | Patients receiving monotherapy |  | |||
Adverse event, n (%) | Onasemnogene abeparvovec → nusinersen (n = 1) | Nusinersen → onasemnogene abeparvovec (n = 14) | Nusinersen → onasemnogene abeparvovec → nusinersen (n = 8) | Onasemnogene abeparvovec only (n = 12) | Nusinersen only (n = 4) | Total (n = 39) |
≥1 treatment-emergent AE (any grade) | 1 (100.0) | 11 (78.6) | 8 (100.0) | 10 (83.3) | 2 (50.0) | 32 (82.1) |
Serious AEs | 1 (100.0) | 4 (28.6) | 5 (62.5) | 4 (33.3) | 1 (25.0) | 15 (38.5) |
Serious and related AEsa | 0 | 3 (21.4) | 2 (25.0) | 1 (8.3) | 0 | 6 (15.4) |
AEs of special interest to onasemnogene abeparvovec | ||||||
 Hepatotoxicityb | 0 | 8 (57.1) | 6 (75.0) | 6 (50.0) | 0 | 20 (51.3) |
 Thrombocytopeniac | 0 | 3 (21.4) | 0 | 1 (8.3) | 0 | 4 (10.3) |
Cardiac AEsd | 1 (100.0) | 3 (21.4) | 3 (37.5) | 2 (16.7) | 0 | 9 (23.1) |