Table 2.
Severity and frequency of abdominal and epigastric symptoms in adult patients with Pompe disease and in age- and gender-matched controls (i.e. accompanying spouses and partners) as analysed by the Gastrointestinal Symptoms Questionnaire
| Symptom | Pompe patients reporting symptoms [n (%)] | Controls reporting symptoms [n (%)] | p b | Severity Pompe patients (n = 58) | Severity Age- and gender-matched controls (n = 58)a | p a |
|---|---|---|---|---|---|---|
| Abdominal pain | ||||||
| General | 2/41 (4.5) | 2/49 (4.1) | n.s. | 0.22 ± 0.88 | 0.06 ± 0.32 | n.s. |
| Postprandial | 8/41 (20) | 8/43 (19) | n.s. | 0.34 ± 0.73 | 0.23 ± 0.52 | n.s. |
| Fasting | 2/39 (6.9) | 1/40 (2.5) | n.s. | 0.18 ± 0.72 | 0.03 ± 0.16 | n.s. |
| Persistent after stools | 3/38 (7.9) | 0/41 (0) | n.s. | 0.26 ± 0.72 | 0 | n.s. |
| Epigastric pain | ||||||
| General | 1/39 (2.6) | 1/49 (2.0) | n.s. | 0.13 ± 0.66 | 0.02 ± 0.14 | n.s. |
| During daytime | 7/39 (18) | 6/43 (14) | n.s. | 0.28 ± 0.79 | 0.19 ± 0.55 | n.s. |
| At night/asleep | 2/37 (5.4) | 2/41 (4.8) | n.s. | 0.14 ± 0.48 | 0.12 ± 0.56 | n.s. |
| Heartburn | 5/49 (10) | 14/51 (27) | 0.04 | 0.30 ± 0.71 | 0.37 ± 0.69 | n.s. |
| Regurgitation | 4/48 (8.3) | 10/50 (20) | n.s. | 0.20 ± 0.61 | 0.24 ± 0.52 | n.s. |
| Abdominal rumbling | 14/51 (27) | 23/50 (46) | n.s. | 0.84 ± 1.05 | 0.58 ± 0.70 | n.s. |
| Bloating | 17/52 (1.9) | 36/53 (68) | 0.04 | 1.32 ± 1.24 | 1.04 ± 0.91 | n.s. |
| Empty feeling | 7/48 (15) | 5/50 (10) | n.s. | 0.40 ± 0.79 | 0.16 ± 0.55 | n.s. |
| Nausea | 7/46 (15) | 3/51 (5.9) | n.s. | 0.20 ± 0.50 | 0.19 ± 0.71 | n.s. |
| Vomiting | 4/48 (8.3) | 3/51 (5.9) | n.s. | 0.06 ± 0.24 | 0.16 ± 0.70 | n.s. |
| Loss of appetite | 4/48 (8.3) | 2/51 (3.9) | n.s. | 0.23 ± 0.66 | 0.12 ± 0.71 | n.s. |
| Postprandial fullness | 8/46 (17) | 20/53 (38) | 0.03 | 0.80 ± 1.13 | 0.58 ± 0.95 | n.s. |
| Belching | 4/45 (8.9) | 18/51 (35) | 0.003 | 0.51 ± 0.76 | 0.41 ± 0.61 | n.s. |
| Flatulence | 6/43 (14) | 30/51 (59) | 0.01 | 1.07 ± 1.24 | 0.83 ± 0.79 | n.s. |
| Hematemesis | 1/44 (2.3) | 1/50 (2.0) | n.s. | 0 | 0.02 ± 0.14 | n.s. |
| Dysphagia | ||||||
| Liquid food | 7/44 (16) | 5/51 (9.8) | n.s. | 0.39 ± 0.84 | 0.21 ± 0.63 | n.s. |
| Solid food | 6/46 (13) | 6/51 | n.s. | 0.23 ± 0.67 | 0.23 ± 0.67 | n.s. |
| Stools | ||||||
| Melaena | 1/37 (2.7) | 0/41 (0) | n.s. | 0 | 0 | n.s. |
| Bloody | 1/39 (2.6) | 2/41 (4.5) | n.s. | 0.05 ± 0.22 | 0.05 + 0.22 | n.s. |
| Mucous | 2/38 (5.2) | 2/41 (4.5) | n.s. | 0.15 ± 0.57 | 0.15 + 0.57 | n.s. |
| Frequent hard | 2/39 (5.1) | 12/43 (28) | n.s. | 0.34 ± 0.62 | 0.35 + 0.65 | n.s. |
| Diarrhea | 26/41 (63) | 7/40 (18) | <0.001 | 0.93 ± 1.21 | 0.28 + 0.64 | 0.02 |
| Alternately solid or loose | 10/44 (23) | 28/48 (58) | n.s. | 0.95 ± 0.93 | 0.83 + 0.81 | n.s. |
| Constipation | 4/40 (10) | 10/41 (24) | n.s. | 0.35 ± 0.70 | 0.34 + 0.76 | n.s. |
| Frequently with pain | 1/39 (2.6) | 6/40 (15) | n.s. | 0.26 ± 0.59 | 0.25 + 0.74 | n.s. |
| Stool urgency | 23/42 (55) | 8/41 (20) | 0.0014 | 1.26 ± 1.50 | 0.29 + 0.68 | <0.01 |
| Incomplete | 6/39 (15) | 2/40 (5.0) | n.s. | 0.38 ± 0.85 | 0.08 + 0.35 | n.s. |
| Steatorrhea | 2/38 (5.2) | 1/40 (2.5) | n.s. | 0.11 ± 0.39 | 0.03 + 0.16 | n.s. |
Data are given as mean ± 1SD. Groups were compared using Mann–Whitney rank sum testa and Fisher exact testb
Frequencies were compared using Mann–Whitney rank sum test
n.s. nonsignificantly