From: Home-based enzyme replacement therapy in children and adults with Pompe disease; a prospective study
 | Total | Classic infantile | Atypical infantile | Childhood-onset | Adult |
---|---|---|---|---|---|
Health complaints during and or within 48Â h after last infusion, n (% of questionnaires) | |||||
 Yes | 27 (6.4%) | 13 (21.3%) | 0 (0.0%) | 1 (2.3%) | 13 (4.2%) |
 No | 395 (93.4%) | 47 (77.0%) | 8 (100.0%) | 43 (97.7%) | 297 (95.8%) |
 Missing | 1 (0.2%) | 1 (1.6%) | 0 (0.0%) | 0 (0.0%) | 0 (0.0%) |
Health complaints during last infusion, n (% of questionnaires) | |||||
 Yes | 18 (4.3%) | 7 (11.5%) | 0 (0.0%) | 1 (2.3%) | 10 (3.2%) |
 No | 404 (95.5%) | 53 (86.9%) | 8 (100.0%) | 43 (97.7%) | 300 (96.8%) |
 Missing | 1 (0.2%) | 1 (1.6%) | 0 (0.0%) | 0 (0.0%) | 0 (0.0%) |
Health complaints within the first 24Â h after last infusion, n (% of questionnaires) | |||||
 Yes | 23 (5.4%) | 11 (18.0%) | 0 (0.0%) | 0 (0.0%) | 12 (3.9%) |
 No | 399 (94.3%) | 49 (80.3%) | 8 (100.0%) | 44 (100.0%) | 298 (96.1%) |
 Missing | 1 (0.2%) | 1 (1.6%) | 0 (0.0%) | 0 (0.0%) | 0 (0.0%) |
Health complaints within 24–48 h after last infusion, n (% of questionnaires) | |||||
 Yes | 15 (3.5%) | 7 (11.5%) | 0 (0.0%) | 0 (0.0%) | 8 (2.6%) |
 No | 407 (96.2%) | 53 (86.9%) | 8 (100.0%) | 44 (100.0%) | 302 (97.4%) |
 Missing | 1 (0.2%) | 1 (1.6%) | 0 (0.0%) | 0 (0.0%) | 0 (0.0%) |
Complaints during last infusion*, n (% of complaints within group) | |||||
 Fatigue | 12 (18.2%) | 4 (12.9%) | N.A | 1 (100.0%) | 7 (20.6%) |
 Headache | 6 (9.1%) | 2 (6.5%) | N.A | 0 (0.0%) | 4 (11.8%) |
 Generally unwell | 6 (9.1%) | 3 (9.7%) | N.A | 0 (0.0%) | 3 (8.8%) |
 Chills | 4 (6.1%) | 2 (6.5%) | N.A | 0 (0.0%) | 2 (5.9%) |
 Other | 4 (6.1%) | 1 (3.2%) | N.A | 0 (0.0%) | 3 (8.8%) |
Complaints within the first 24Â h after infusion*, n (% of complaints within group) | |||||
 Fatigue | 15 (19.0%) | 8 (19.0%) | N.A | N.A | 7 (18.9%) |
 Headache | 12 (15.2%) | 6 (14.3%) | N.A | N.A | 6 (16.2%) |
 Generally unwell | 7 (8.9%) | 5 (11.9%) | N.A | N.A | 2 (5.4%) |
 Diarrhoea | 4 (5.1%) | 2 (4.8%) | N.A | N.A | 2 (5.4%) |
Complaints within 24–48 h after infusion*, (% of complaints within group) | |||||
 Fatigue | 9 (16.4%) | 5 (16.1%) | N.A | N.A | 4 (16.7%) |
 Headache | 6 (10.9%) | 3 (9.7%) | N.A | N.A | 3 (12.5%) |
Symptoms related to infusion*, n (% of questionnaires) | |||||
 Yes | 15 (55.6%) | 9 (69.2%) | N.A | 0 (0.0%) | 6 (46.2%) |
 No | 4 (14.8%) | 3 (23.1%) | N.A | 0 (0.0%) | 1 (7.7%) |
 Maybe | 8 (29.6%) | 1 (7.7%) | N.A | 1 (100.0%) | 6 (46.2%) |
Hospitalised/emergency room/GP the week or two weeks after last infusion, n (% of questionnaires) | |||||
 No | 410 (96.9%) | 58 (95.1%) | 8 (100.0%) | 42 (95.5%) | 302 (97.4%) |
 Yes | 13 (3.1%) | 3 (4.9%) | 0 (0.0%) | 2 (4.5%) | 8 (2.6%) |
   Broken leg | 3(0.7%) | 1 (1.6%) | 0(0.0%) | 0(0.0%) | 2 (0.6%) |
   Itching skin, nausea, headache, tingling sensation mouth | 1(0.2%) | 1 (1.6%) | 0 (0.0%) | 0 (0.0%) | 0 (0.0%) |
   Pulmonary infection | 1 (0.2%) | 1(1.6%) | 0 (0.0%) | 0 (0.0%) | 0 (0.0%) |
   Burn | 2 (0.5%) | 0 (0.0%) | 0 (0.0%) | 2 (4.5%) | 0 (0.0%) |
   Inflamed intestine | 1 (0.2%) | 0 (0.0%) | 0 (0.0%) | 0 (0.0%) | 1 (0.3%) |
   Cerebral haemorrhage | 1 (0.2%) | 0 (0.0%) | 0 (0.0%) | 0 (0.0%) | 1 (0.3%) |
   Traffic accident | 1 (0.2%) | 0 (0.0%) | 0 (0.0%) | 0 (0.0%) | 1 (0.3%) |
   Kidney stones | 2 (0.5%) | 0 (0.0%) | 0 (0.0%) | 0 (0.0%) | 2 (0.3%) |
   Chest pain | 1 (0.2%) | 0 (0.0%) | 0 (0.0%) | 0 (0.0%) | 1 (0.3%) |