Table 3.
Influence of severity of symptoms in PedsQL™ Family Impact Module
| Sample | Total Impact Score | Parent HRQOL | Family Functioning |
|---|---|---|---|
| Gross motor | |||
| GMFC-MLD ≤ 4 (n = 10/10/10) | 61.7 (18.6) | 63.3 (19.6) | 59.3 (19.6) |
| GMFC-MLD > 4 (n = 32/32/31) | 56.5 (17.4) | 60.6 (18.2) | 47.3 (22.7) |
| Complete immobility | |||
| GMFC-MLD < 6 (n = 30/30/30) | 61.8 (17.8) | 64.2 (18.9) | 56.0 (22.0) |
| GMFC-MLD = 6 (n = 12/12/11) | 48.6* (13.5) | 54.6 (15.8) | 37.2* (18.3) |
| Speech | |||
| VSS < 4 (n = 10/10/10) | 62.5 (16.8) | 65.0 (18.0) | 60.5 (17.8) |
| VSS = 4 (n = 32/32/31) | 56.6 (17.9) | 60.3 (18.6) | 47.9 (23.2) |
| Complete loss of communication | |||
| VSS ≤ 4 (at least ability to express emotions) (n = 34/34/34) | 61.5 (16.4) | 65.1 (17.1) | 54.3 (21.8) |
| No ability to express emotions (n = 8/8/7) | 43.1** (15.3) | 45.8** (15.9) | 34.5 (19.3) |
| Visual stimulation | |||
| Visual stimuli perceptible (n = 32/32/32) | 62.2 (16.7) | 65.8 (17.2) | 54.1 (21.5) |
| Visual stimuli not perceptible (n = 10/10/9) | 44.8** (13.9) | 47.4** (15.0) | 39.6 (23.4) |
| Independent eating | |||
| Independent eating possible (n = 8/8/8) | 66.3 (17.8) | 67.7 (18.9) | 64.5 (15.9) |
| No independent eating possible (n = 34/34/33) | 56.1 (17.3) | 60.0 (18.2) | 47.7* (22.8) |
| PEG tube | |||
| No PEG tube present (n = 23/23/23) | 62.7 (15.7) | 64.8 (17.9) | 56.4 (18.6) |
| PEG tube present (n = 19/19/18) | 52.3* (18.5) | 57.3 (18.6) | 44.0 (24.5) |
| Final stage of disease | |||
| Child alive (n = 32/32/31) | 61.7 (17.5) | 64.7 (18.2) | 55.4 (21.9) |
| Child deceased (n = 11/11/11) | 44.5** (9.8) | 49.3* (14.0) | 34.2** (14.8) |
Standard deviation presented in parentheses. Different case numbers result from the fact that one Family Functioning scale was insufficiently completed. Higher values equal better functioning/less negative impact
PedsQL™, Pediatric Quality of Life Inventory [23]; HRQOL, health-related quality of life; GMFC-MLD, Gross Motor Function Classification-MLD [16]; MLD, metachromatic leukodystrophy; VSS, The Viking Speech Scale [24]; PEG, percutaneous endoscopic gastrostomy
*P value is significant at < 0.05 level; **P value is significant at < 0.01 level. Comparison between every pair of a sub-item