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Table 3 Infusion Associated Reactions

From: Home-based enzyme replacement therapy in children and adults with Pompe disease; a prospective study

 

Total

Classic infantile

Atypical infantile

Childhood-onset

Adult

Number of IARs past 3 months, n (% of questionnaires)

 1 infusion

1 (0.2%)

0 (0.0%)

0 (0.0%)

0 (0.0%)

1 (0.3%)

 2 infusions

1 (0.2%)

1 (1.6%)

0 (0.0%)

0 (0.0%)

0 (0.0%)

 3 infusions

2 (0.5%)

2 (3.3%)

0 (0.0%)

0 (0.0%)

0 (0.0%)

 4 infusions

1 (0.2%)

1 (1.6%)

0 (0.0%)

0 (0.0%)

0 (0.0%)

 6 infusions

9 (2.1%)

5 (8.2%)

0 (0.0%)

0 (0.0%)

4 (1.3%)

 12 infusions

1 (0.2%)

0 (0.0%)

0 (0.0%)

0 (0.0%)

1 (0.3%)#

 Never

407 (96.2%)

51 (83.6%)

8 (100.0%)

44 (100.0%)

304 (98.1%)

 Missing

1 (0.2%)

1 (1.6%)

0 (0.0%)

0 (0.0%)

0 (0.0%)

Actions taken past 3 months in response to IAR, n (% of interventions)

 Infusion paused

7 (23.3%)

7 (28.0%)

N.A

N.A

0 (0.0%)

 Infusion speed adapted

4 (13.3%)

4 (16.0%)

N.A

N.A

0 (0.0%)

 Erasmus MC contacted

9 (30.0%)

7 (28.0%)

N.A

N.A

2 (40.0%)

 Medication was given

9 (30.0%)

6 (24.0%)

N.A

N.A

3 (60.0%)

 Admitted to hospital

1 (3.3%)

1 (4.0%)

N.A

N.A

0 (0.0%)

Did the IAR return after restarting the infusion*, n (% of paused infusions)

 Yes

0 (0.0%)

0 (0.0%)

N.A

N.A

N.A

 No

7 (100.0%)

7 (100.0%)

N.A

N.A

N.A

Did the infusion reaction resolve due to the actions taken**, n (% of interventions)

 Yes

11 (78.6%)

8 (88.9%)

N.A

N.A

3 (60.0%)

 No

1 (7.1%)

0 (0.0%)

N.A

N.A

1 (20.0%)

 Don’t know

1 (7.1%)

0 (0.0%)

N.A

N.A

1 (20.0%)

 Missing

1 (7.1%)

1 (11.1%)

N.A

N.A

0 (0.0%)

Actions taken to prevent IARs, n (% interventions)

 One of the infusion steps was adapted

3 (10.7%)

3 (15.0%)

N.A

N.A

0 (0.0%)

 Infusion schedule completely adapted

7 (25.0%)

4 (20.0%)

N.A

N.A

3 (37.5%)

 Premedication

10 (35.7%)

6 (30.0%)

N.A

N.A

4 (50.0%)

 Next infusion in the hospital

7 (25.0%)

7 (35.0%)

N.A

N.A

0 (0.0%)

 None

1 (3.6%)

0 (0.0%)

N.A

N.A

1 (12.5%)

  1. N.A. = Not applicable * Question only available if the patients’ infusion had been paused in response to an IAR ** Question only available if actions were taken in response to IARs # Only 6 infusions were provided to this patient in the preceding three months