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 |