Table 2.
Neuro-QOL Fatigue individual item responses and results from a repeated-measures proportional odds model at REGAIN week 26
| Parameter | Treatment | Observed responses for Neuro-QOL Fatigue items at REGAIN week 26 | Repeated-measures model estimate for REGAIN week 26 | ||||||
|---|---|---|---|---|---|---|---|---|---|
| 1 = Never, n (%) | 2 = Rarely, n (%) | 3 = Sometimes, n (%) | 4 = Often, n (%) | 5 = Always, n (%) | Proportional odds ratio (95% CI)b | p value for odds ratiob,c | p value for proportional odds assumptiond | ||
| I felt exhausted | ECU (na = 57) | 9 (15.8) | 14 (24.6) | 19 (33.3) | 13 (22.8) | 2 (3.5) | 2.4 (1.3, 4.4) | 0.0038 | 0.1099 |
| PLC (na = 60) | 5 (8.3) | 9 (15.0) | 25 (41.7) | 16 (26.7) | 5 (8.3) | ||||
| I felt that I had no energy | ECU (na = 57) | 9 (15.8) | 15 (26.3) | 21 (36.8) | 9 (15.8) | 3 (5.3) | 1.9 (1.1, 3.5) | 0.0322 | 0.5692 |
| PLC (na = 60) | 5 (8.3) | 10 (16.7) | 23 (38.3) | 15 (25.0) | 7 (11.7) | ||||
| I felt fatigued | ECU (na = 57) | 7 (12.3) | 16 (28.1) | 22 (38.6) | 8 (14.0) | 4 (7.0) | 1.8 (1.0, 3.4) | 0.0467 | 0.3357 |
| PLC (na = 60) | 2 (3.3) | 14 (23.3) | 24 (40.0) | 15 (25.0) | 5 (8.3) | ||||
| I was too tired to do my household chores | ECU (na = 56) | 16 (28.6) | 15 (26.8) | 13 (23.2) | 7 (12.5) | 5 (8.9) | 1.9 (1.0, 3.4) | 0.0437 | 0.6454 |
| PLC (na = 60) | 11 (18.3) | 11 (18.3) | 19 (31.7) | 17 (28.3) | 2 (3.3) | ||||
| I was too tired to leave the house | ECU (na = 57) | 22 (38.6) | 12 (21.1) | 12 (21.1) | 8 (14.0) | 3 (5.3) | 2.5 (1.4, 4.7) | 0.0033 | 0.2162 |
| PLC (na = 60) | 12 (20.0) | 11 (18.3) | 20 (33.3) | 15 (25.0) | 2 (3.3) | ||||
| I was frustrated by being too tired to do the things I wanted to do | ECU (na = 57) | 20 (35.1) | 14 (24.6) | 11 (19.3) | 8 (14.0) | 4 (7.0) | 2.5 (1.4, 4.5) | 0.0032 | 0.4059 |
| PLC (na = 60) | 14 (23.3) | 7 (11.7) | 16 (26.7) | 18 (30.0) | 5 (8.3) | ||||
| I felt tired | ECU (na = 57) | 9 (15.8) | 9 (15.8) | 22 (38.6) | 12 (21.1) | 5 (8.8) | 2.3 (1.2, 4.4) | 0.0092 | 0.3737 |
| PLC (na = 60) | 0 (0.0) | 9 (15.0) | 26 (43.3) | 17 (28.3) | 8 (13.3) | ||||
| I had to limit my social activity because I was tired | ECU (na = 57) | 15 (26.3) | 16 (28.1) | 13 (22.8) | 9 (15.8) | 4 (7.0) | 2.6 (1.4, 4.7) | 0.0026 | 0.6225 |
| PLC (na = 60) | 8 (13.3) | 11 (18.3) | 18 (30.0) | 17 (28.3) | 6 (10.0) | ||||
| I needed help doing my usual activities because of my fatigue | ECU (na = 57) | 26 (45.6) | 10 (17.5) | 10 (17.5) | 7 (12.3) | 4 (7.0) | 2.3 (1.2, 4.2) | 0.0104 | 0.5279 |
| PLC (na = 60) | 14 (23.3) | 11 (18.3) | 20 (33.3) | 12 (20.0) | 3 (5.0) | ||||
| I needed to sleep during the day | ECU (na = 57) | 14 (24.6) | 14 (24.6) | 13 (22.8) | 10 (17.5) | 6 (10.5) | 1.5 (0.8, 2.7) | 0.2164 | 0.4892 |
| PLC (na = 60) | 12 (20.0) | 7 (11.7) | 16 (26.7) | 15 (25.0) | 10 (16.7) | ||||
| I had trouble starting things because I was too tired | ECU (na = 57) | 16 (28.1) | 18 (31.6) | 13 (22.8) | 6 (10.5) | 4 (7.0) | 2.2 (1.2, 4.1) | 0.0096 | 0.1930 |
| PLC (na = 60) | 15 (25.0) | 7 (11.7) | 18 (30.0) | 13 (21.7) | 7 (11.7) | ||||
| I had trouble finishing things because I was too tired | ECU (na = 57) | 20 (35.1) | 13 (22.8) | 13 (22.8) | 8 (14.0) | 3 (5.3) | 2.5 (1.4, 4.5) | 0.0027 | 0.8333 |
| PLC (na = 60) | 12 (20.0) | 11 (18.3) | 18 (30.0) | 13 (21.7) | 6 (10.0) | ||||
| I was too tired to take a short walk | ECU (na = 57) | 19 (33.3) | 14 (24.6) | 11 (19.3) | 9 (15.8) | 4 (7.0) | 1.5 (0.8, 2.7) | 0.1896 | 0.9608 |
| PLC (na = 60) | 17 (28.3) | 10 (16.7) | 15 (25.0) | 14 (23.3) | 4 (6.7) | ||||
| I was too tired to eat | ECU (na = 57) | 30 (52.6) | 12 (21.1) | 10 (17.5) | 1 (1.8) | 4 (7.0) | 1.2 (0.6, 2.3) | 0.5365 | 0.9093 |
| PLC (na = 60) | 25 (41.7) | 17 (28.3) | 11 (18.3) | 7 (11.7) | 0 (0.0) | ||||
| I was so tired that I had to rest during the day | ECU (na = 57) | 10 (17.5) | 11 (19.3) | 13 (22.8) | 17 (29.8) | 6 (10.5) | 1.2 (0.7, 2.3) | 0.4697 | 0.4873 |
| PLC (na = 60) | 9 (15.0) | 8 (13.3) | 19 (31.7) | 14 (23.3) | 10 (16.7) | ||||
| I felt weak all over | ECU (na = 57) | 23 (40.4) | 9 (15.8) | 13 (22.8) | 7 (12.3) | 5 (8.8) | 2.5 (1.4, 4.7) | 0.0032 | 0.4309 |
| PLC (na = 60) | 12 (20.0) | 12 (20.0) | 14 (23.3) | 17 (28.3) | 5 (8.3) | ||||
| I needed help doing my usual activities because of weakness | ECU (na = 57) | 26 (45.6) | 12 (21.1) | 10 (17.5) | 6 (10.5) | 3 (5.3) | 2.3 (1.2, 4.2) | 0.0095 | 0.3340 |
| PLC (na = 60) | 14 (23.3) | 13 (21.7) | 17 (28.3) | 14 (23.3) | 2 (3.3) | ||||
| I had to limit my social activity because I was physically weak | ECU (na = 57) | 19 (33.3) | 18 (31.6) | 6 (10.5) | 10 (17.5) | 4 (7.0) | 2.9 (1.6, 5.3) | 0.0006 | 0.1119 |
| PLC (na = 60) | 10 (16.7) | 8 (13.3) | 20 (33.3) | 15 (25.0) | 7 (11.7) | ||||
| I had to force myself to get up and do things because I was physically too weak | ECU (na = 57) | 25 (43.9) | 15 (26.3) | 9 (15.8) | 5 (8.8) | 3 (5.3) | 2.1 (1.1, 4.0) | 0.0172 | 0.0927 |
| PLC (na = 60) | 19 (31.7) | 7 (11.7) | 18 (30.0) | 15 (25.0) | 1 (1.7) | ||||
The p values associated with the proportional odds assumption indicated that this assumption was upheld for each item
CI confidence interval, ECU eculizumab, Neuro-QOL Quality of Life in Neurological Disorders, PLC placebo
aNumber of patients who completed the assessment at week 26; missing scores were not imputed for patients who did not complete the assessment (ECU, n = 5 [n = 6 for ‘I was too tired to do my household chores’]; PLC, n = 3)
bProportional odds ratios and p values are based on a repeated-measures ordinal regression (implemented using the GEEORD SAS Macro). The model has the ordinal response (for each parameter) at each visit as the dependent variable with terms for treatment, visit, and treatment by visit interaction, assuming exchangeable correlation structure. A higher odds ratio indicates that eculizumab-treated patients are more likely to answer in the lower response categories than those receiving placebo at week 26 for that item
cp values are not adjusted for multiple comparisons
dA p value for proportional odds assumption < 0.05 may indicate non-proportionality