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Table 2 Health complaints during and after the last infusion

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 48 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 24 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 24 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%)

  1. N.A. = Not applicable GP = General Practitioner * Question was only available if the patient stated to have experienced any health complaints during and or within 48 h from the last infusion