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 3Â 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 3Â 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%) |