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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.