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Table 1 Summary of treatment-emergent AEs in the TSP

From: Agalsidase alfa in pediatric patients with Fabry disease: a 6.5-year open-label follow-up study

Patients who experienced: TSP, N =11
n (%)
Phase 1 Phase 2 Overall
≥1 AE 11 (100) 11 (100) 11 (100)
≥1 mild AE 0 3a (27.3) 0a
≥1 moderate AE 5 (45.5) 6 (54.5) 5 (45.5)
≥1 severe or life-threatening AE 6 (54.5) 2 (18.2) 6 (54.5)
Most common AEs (≥50% of the TSP overall)  
     Cough 9 (81.8) 3 (27.3) 10 (90.9)
     Pyrexia 8 (72.7) 3 (27.3) 9 (81.8)
     Abdominal pain 7 (63.6) 2 (18.2) 8 (72.7)
     Pain in extremity 8 (72.7) 4 (36.4) 8 (72.7)
     Chest pain 6 (54.5) 3 (27.3) 7 (63.6)
     Headache 7 (63.6) 4 (36.4) 7 (63.6)
     Neuralgia 7 (63.6) 0 7 (63.6)
     Abdominal pain upper 5 (45.5) 2 (18.2) 6 (54.5)
     Diarrhea 3 (27.3) 5 (45.5) 6 (54.5)
     Dyspnea 3 (27.3) 3 (27.3) 6 (54.5)
     Nasal congestion 6 (54.5) 2 (18.2) 6 (54.5)
     Nasopharyngitis 3 (27.3) 4 (36.4) 6 (54.5)
     Vomiting 3 (27.3) 3 (27.3) 6 (54.5)
≥1 drug-related AE 8 (72.7) 2 (18.2) 8 (72.7)
≥1 infusion-related AE 5 (45.5) 2 (18.2) 6 (54.5)
≥1 SAE 1 (9.1) 1 (9.1) 2 (18.2)
≥1 drug-related SAE 0 0 0
Discontinuation due to an AE 0 0 0
Death 0 0 0
  1. aHighest AE severity was counted at the patient level and the three patients who had “mild” events in phase 2 also had a higher severity event in phase 1 (i.e., moderate, severe, or life-threatening) and are thus classified in a higher severity category in the “Overall” phases (i.e., three mild AEs in phase 2, but none overall).
  2. AE, adverse event; SAE, serious adverse event; TSP, transition study population.