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Table 3 Treatment-emergent AEs by treatment

From: 10th European Conference on Rare Diseases & Orphan Products (ECRD 2020)

 Treatment received 
 Patients receiving > 1 active therapyPatients 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 AEs1 (100.0)4 (28.6)5 (62.5)4 (33.3)1 (25.0)15 (38.5)
Serious and related AEsa03 (21.4)2 (25.0)1 (8.3)06 (15.4)
AEs of special interest to onasemnogene abeparvovec
 Hepatotoxicityb08 (57.1)6 (75.0)6 (50.0)020 (51.3)
 Thrombocytopeniac03 (21.4)01 (8.3)04 (10.3)
Cardiac AEsd1 (100.0)3 (21.4)3 (37.5)2 (16.7)09 (23.1)
  1. aFor patients who received > 1 therapy, relatedness could be due to either medicinal product and was not specified by the reporter. bPrimarily transient transaminase elevations. cPrimarily isolated decrease in platelet count without clinical evidence of bleeding events. dCardiac AEs include bradycardia, troponin elevations, cardiac arrest, thrombotic microangiopathy, and hepatomegaly; at least 1 blood and lymphatic system-related AE and 1 event of hepatomegaly were misclassified as cardiac and will be corrected