Self-reported scores of questionnaires assessing quality of life, fatigue, depression, attentions deficits, and pain in association with neurodegeneration and treatment
| Self-report (scale) . | ND-LCH . | Systemic treatment∗∗ . | MS patients, treatment >48 months . | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Yes . | No . | P value# . | Yes . | No . | P value# . | Yes . | No . | P value# . | |||||||||||||
| n . | Mean (SD) . | Median (IQR) . | n . | Mean (SD) . | Median (IQR) . | n . | Mean (SD) . | Median (IQR) . | n . | Mean (SD) . | Median (IQR) . | n . | Mean (SD) . | Median (IQR) . | n . | Mean (SD) . | Median (IQR) . | ||||
| Total HRQOL score∗ (0-100) | 5 | 73.3 (21.9) | 80.4 (92.4-50.5) | 27 | 79.8 (15.2) | 81.5 (90.2-69.6) | 0.763 | 17 | 76.3 (16.8) | 80.4 (89.1-62.5) | 14 | 81.9 (16.1) | 87.5 (76.0-92.9) | 0.377 | 6 | 82.8 (20.3) | 89.7 (95.1-71.2) | 7 | 64.6 (18.0) | 67.4 (80.4-67.4) | 0.101 |
| Quality of life† (0-10) | 5 | 6.0 (2.5) | 7.0 (8.0-3.5) | 21 | 6.8 (2.5) | 7.0 (9.0-4.5) | 0.486 | 14 | 6.4 (2.7) | 6.3 (9.1-3.9) | 11 | 7.0 (2.3) | 7.0 (6.0-9.0) | 0.687 | 6 | 7.2 (1.8) | 7.5 (8.5-5.4) | 5 | 4.7 (2.9) | 4.0 (7.3-2.5) | 0.082 |
| Total fatigue score‡ (0-100) | 5 | 64.7 (27.4) | 66.7 (91.0-37.5) | 25 | 69.5 (18.5) | 69.4 (90.3-56.3) | 0.666 | 16 | 62.8 (21.8) | 57.6 (83.0-45.5) | 13 | 75.6 (15.8) | 72.2 (63.6-91.7) | 0.092 | 6 | 72.5 (25.8) | 76.4 (96.2-51.4) | 6 | 57.4 (21.5) | 52.8 (75.3-41.3) | 0.310 |
| Depression (MADRS-s)§ (0-54) | 5 | 8.4 (10.9) | 3.0 (19.0-0.5) | 21 | 10.1 (10.0) | 8.0 (14.5-2.0) | 0.569 | 14 | 12.1 (12.2) | 9.5 (19.3-1.8) | 11 | 7.0 (6.4) | 7.0 (2.0-10.0) | 0.434 | 6 | 4.5 (5.5) | 2.0 (11.3-0.0) | 5 | 23.6 (8.5) | 22.0 (31.5-16.5) | 0.004 |
| Hyperactivity (z score)|| | 5 | −0.23 (1.5) | −0.83 (1.15 to −1.32) | 27 | 0.05 (0.9) | 0.08 (0.69 to −0.68) | 0.220 | 17 | 0.16 (1.2) | 0.08 (0.88 to −0.91) | 14 | −0.19 (0.62) | −0.53 (−0.68-0.54) | 0.710 | 6 | −0.8 (0.6) | −0.99 (−0.37 to −1.23) | 7 | 0.8 (1.0) | 0.77 (1.68-0.83) | 0.005 |
| Inattention (z score)|| | 5 | −0.23 (1.5) | −0.93 (1.42 to −1.54) | 27 | 0.0 (0.9) | 0.01 (0.55 to –0.66) | 0.545 | 17 | 0.16 (1.17) | 0.33 (1.05 to −0.97) | 14 | −0.20 (0.69) | −0.12 (−0.70.0.42) | 0.377 | 6 | −0.7 (1.2) | −1.20 (0.32 to −1.50) | 7 | 0.6 (0.8) | 0.68 (1.22-0.33) | 0.073 |
| Pain¶ (0-10) | 5 | 2.8 (3.6) | 1.0 (6.5-0.0) | 16 | 3.8 (3.2) | 5.0 (6.75-0.0) | 0.719 | 12 | 4.3 (3.3) | 5.5 (7.0-0.3) | 8 | 2.1 (3.0) | 0.0 (0.0-5.0) | 0.115 | 5 | 4.0 (3.4) | 5.0 (7.0-0.5) | 4 | 4.3 (3.4) | 5.0 (7.0-0.8) | 0.905 |
| Self-report (scale) . | ND-LCH . | Systemic treatment∗∗ . | MS patients, treatment >48 months . | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Yes . | No . | P value# . | Yes . | No . | P value# . | Yes . | No . | P value# . | |||||||||||||
| n . | Mean (SD) . | Median (IQR) . | n . | Mean (SD) . | Median (IQR) . | n . | Mean (SD) . | Median (IQR) . | n . | Mean (SD) . | Median (IQR) . | n . | Mean (SD) . | Median (IQR) . | n . | Mean (SD) . | Median (IQR) . | ||||
| Total HRQOL score∗ (0-100) | 5 | 73.3 (21.9) | 80.4 (92.4-50.5) | 27 | 79.8 (15.2) | 81.5 (90.2-69.6) | 0.763 | 17 | 76.3 (16.8) | 80.4 (89.1-62.5) | 14 | 81.9 (16.1) | 87.5 (76.0-92.9) | 0.377 | 6 | 82.8 (20.3) | 89.7 (95.1-71.2) | 7 | 64.6 (18.0) | 67.4 (80.4-67.4) | 0.101 |
| Quality of life† (0-10) | 5 | 6.0 (2.5) | 7.0 (8.0-3.5) | 21 | 6.8 (2.5) | 7.0 (9.0-4.5) | 0.486 | 14 | 6.4 (2.7) | 6.3 (9.1-3.9) | 11 | 7.0 (2.3) | 7.0 (6.0-9.0) | 0.687 | 6 | 7.2 (1.8) | 7.5 (8.5-5.4) | 5 | 4.7 (2.9) | 4.0 (7.3-2.5) | 0.082 |
| Total fatigue score‡ (0-100) | 5 | 64.7 (27.4) | 66.7 (91.0-37.5) | 25 | 69.5 (18.5) | 69.4 (90.3-56.3) | 0.666 | 16 | 62.8 (21.8) | 57.6 (83.0-45.5) | 13 | 75.6 (15.8) | 72.2 (63.6-91.7) | 0.092 | 6 | 72.5 (25.8) | 76.4 (96.2-51.4) | 6 | 57.4 (21.5) | 52.8 (75.3-41.3) | 0.310 |
| Depression (MADRS-s)§ (0-54) | 5 | 8.4 (10.9) | 3.0 (19.0-0.5) | 21 | 10.1 (10.0) | 8.0 (14.5-2.0) | 0.569 | 14 | 12.1 (12.2) | 9.5 (19.3-1.8) | 11 | 7.0 (6.4) | 7.0 (2.0-10.0) | 0.434 | 6 | 4.5 (5.5) | 2.0 (11.3-0.0) | 5 | 23.6 (8.5) | 22.0 (31.5-16.5) | 0.004 |
| Hyperactivity (z score)|| | 5 | −0.23 (1.5) | −0.83 (1.15 to −1.32) | 27 | 0.05 (0.9) | 0.08 (0.69 to −0.68) | 0.220 | 17 | 0.16 (1.2) | 0.08 (0.88 to −0.91) | 14 | −0.19 (0.62) | −0.53 (−0.68-0.54) | 0.710 | 6 | −0.8 (0.6) | −0.99 (−0.37 to −1.23) | 7 | 0.8 (1.0) | 0.77 (1.68-0.83) | 0.005 |
| Inattention (z score)|| | 5 | −0.23 (1.5) | −0.93 (1.42 to −1.54) | 27 | 0.0 (0.9) | 0.01 (0.55 to –0.66) | 0.545 | 17 | 0.16 (1.17) | 0.33 (1.05 to −0.97) | 14 | −0.20 (0.69) | −0.12 (−0.70.0.42) | 0.377 | 6 | −0.7 (1.2) | −1.20 (0.32 to −1.50) | 7 | 0.6 (0.8) | 0.68 (1.22-0.33) | 0.073 |
| Pain¶ (0-10) | 5 | 2.8 (3.6) | 1.0 (6.5-0.0) | 16 | 3.8 (3.2) | 5.0 (6.75-0.0) | 0.719 | 12 | 4.3 (3.3) | 5.5 (7.0-0.3) | 8 | 2.1 (3.0) | 0.0 (0.0-5.0) | 0.115 | 5 | 4.0 (3.4) | 5.0 (7.0-0.5) | 4 | 4.3 (3.4) | 5.0 (7.0-0.8) | 0.905 |
ND-LCH, LCH-associated neurodegeneration; MS, multisystem; HRQOL, health-realted quality of life; MADRS, Montgomery Åsberg Depression Scale.
5-point Likert scale, the PedsQL Generic Core Scale 4.0, ranges from 0 (worst quality of life) to 100 (best quality of life).
Experience the last year on a 10-point scale from 0 (worst possible quality of life) to 10 (best possible quality of life).
5-point Likert scale, the PedsQL Multidimensional Fatigue Scale, ranges from 0 (most fatigue) to 100 (least fatigue).
7-point Likert scale, cut-off points are as follows: 0 to 12, no depression; 13 to 19, mild depression; 20 to 34, moderate depression; and ≥35, severe depression.
Adult ADHD self-report scales v1.1 (patients ≥18 years) and Conners 3 short form (patients <18 years) transformed to a z score, which compares each individual to the mean of the cohort.
Pain experienced the last year on a 10-point scale from 0 (no pain) to 10 (worst possible pain).
Mann-Whitney U test. P value in bold indicates statistically significant (P <.05)
One patient with only local treatment for LCH but later systemic treatment for rheumatoid arthritis is excluded.