Table 3.
Absolute changes from baseline of secondary outcomes
| Variable | Start | Stop | ||||||
|---|---|---|---|---|---|---|---|---|
| Group (treatment) | Absolute changes from baseline | Comparison to A (placebo 4wks); P | Effect size (95% CI) | Group (treatment) | Absolute changes from baseline | Comparison to E (HCQ 4wks); P | Effect size (95% CI) | |
| O2-sat in room air (%) | A (placebo 4wks) | − 0.1 (2.4) | E (HCQ 12wks) | 0.3 (1.2) | ||||
| B (HCQ 4wks) | − 0.6 (3.3) | 0.757 | − 0.2 (− 1.2−0.8) | F (placebo 12wks) | − 2.4 (2.7) | 0.097 | − 1.2 (− 2.7–0.4) | |
| C (HCQ 4wks) | 1.8 (3.5) | 0.229 | 0.6 (− 0.2−1.4) | G (no med. 12wks) | − 3.7 (6.4) | 0.427 | − 0.9 (− 2.5−0.8) | |
| B + C (combined) | 1.1 (3.5) | 0.300 | 0.4 (− 0.3−1.1) | F + G combined | − 2.9 (4) | 0.071 | − 0.9 (− 2.3−0.5) | |
| Resp. rate in room air ( /min) | A (placebo 4wks) | 1.2 (5.4) | E (HCQ 12wks) | − 2.7 (1.2) | ||||
| B (HCQ 4wks) | 0.2 (0.4) | 0.543 | − 0.2 (− 1.2−0.8) | F (placebo 12wks) | − 5.4 (9.4) | 0.553 | − 0.4 (− 1.8−1.1) | |
| C (HCQ 4wks) | − 1.3 (5.6) | 0.421 | − 0.5 (− 1.2−0.3) | G (no med. 12wks) | 7.3 (5) | 0.102 | 2.8 (0.5−5) | |
| B + C (combined) | − 0.9 (4.8) | 0.289 | − 0.4 (− 1.1−0.3) | F + G combined | − 0.6 (10) | 0.589 | 0.2 (− 1.1−1.6) | |
| Quality of life (chILD specific) | A (placebo 4wks) | 6.9 (9.6) | E (HCQ 12wks) | − 5.7 (11.2) | ||||
| B (HCQ 4wks) | 0.1 (3.18) | 0.178 | − 0.8 (− 2.4−0.9) | F (placebo 12wks) | 7.5 (2.3) | 0.338 | 1.6 (− 0.6−3.9) | |
| C (HCQ 4wks) | − 3.2 (4.8) | 0.110 | − 1.3 (− 2.6–− 0.1) | G (no med. 12wks) | 2.3 (3.2) | 0.398 | 1.0 (− 1.1−3.0) | |
| B + C (combined) | − 2.4 (4.5) | 0.064 | − 1.3 (− 2.5–−0.2) | F + G combined | 4.9 (3.8) | 0.403 | 1.6 (− 0.3−3.6) | |
| Quality of life (total score) | A (placebo 4wks) | 0.4 (6.9) | E (HCQ 12wks) | 9.2 (5.4) | ||||
| B (HCQ 4wks) | 0.3 (1.2) | 0.965 | 0 (− 1.6–1.6) | F (placebo 12wks) | − 9.5 (8.8) | 0.149 | − 2.6 (− 5.2−0.1) | |
| C (HCQ 4wks) | 4.8 (4.3) | 0.374 | 0.8 (− 0.4–1.9) | G (no med. 12wks) | − 1.7 (2.3) | 0.296 | − 2.6 (− 5.3–0.0) | |
| B + C (combined) | 3.7 (4.3) | 0.333 | 0.6 (− 0.5–1.7) | F + G combined | − 5.6 (6.9) | 0.072 | − 2.3 (− 4.4– -0.1) | |
| BMI percentile | A (placebo 4wks) | 7.5 (11.7) | E (HCQ 12wks) | − 3.3 (7.9) | ||||
| B (HCQ 4wks) | − 1.8 (5.1) | 0.024 | − 0.9 (− 1.9-0) | F (placebo 12wks) | 2.2 (11.4) | 0.458 | 0.5 (− 0.9−2.0) | |
| C (HCQ 4wks) | − 1.6 (8.1) | 0.045 | − 0.9 (− 1.7–−0.1) | G (no med. 12wks) | 13.3 (20.9) | 0.422 | 1.1 (− 0.7–2.8) | |
| B + C (combined) | − 1.7 (7.1) | 0.020 | − 1.0 (− 1.7−–0.3) | F + G combined | 6.4 (15.2) | 0.213 | 0.7 (− 0.7−2.1) | |
| LDH (U/ml) | A (placebo 4wks) | − 28.2 (53.7) | E (HCQ 12wks) | 34.5 (0.7) | ||||
| B (HCQ 4wks) | 43.0 (61) | 0.063 | 1.3 (0.1−2.5) | F (placebo 12wks) | 9.3 (65.5) | 0.574 | − 0.5 (− 2.3−1.3) | |
| C (HCQ 4wks) | 11.3 (67) | 0.410 | 0.6 (− 0.3−1.6) | G (no med. 12wks) | Not done | n.appl | n.appl | |
| B + C (combined) | 21.9 (64.7) | 0.055 | 0.8 (0−1.7) | F + G combined | 9.3 (65.5) | 0.574 | − 0.5 (− 2.3−1.3) | |
| FVC (% pred) | A (placebo 4wks) | 0.0 (3.7) | E (HCQ 12wks) | 2.3 (7.6) | ||||
| B (HCQ 4wks) | 2.5 (13) | 0.730 | 0.3 (− 0.9−1.5) | F (placebo 12wks) | 0.5 (2.1) | 0.724 | − 0.3 (− 2.1−1.5) | |
| C (HCQ 4wks) | 10.6 (19.7) | 0.170 | 0.7 (− 0.3−1.8) | G (no med. 12wks) | − 1.3 (5.1) | 0.487 | − 0.6 (− 2.2−1.1) | |
| B + C (combined) | 7.9 (17.6) | 0.157 | 0.6 (− 0.3−1.5) | F + G combined | − 0.6 (3.9) | 0.583 | − 0.5 (− 2.0−0.9) | |
| FEV1 (% pred) | A (placebo 4wks) | 0.6 (4.1) | E (HCQ 12wks) | 0.0 (4.4) | ||||
| B (HCQ 4wks) | 2.0 (13.1) | 0.850 | 0.2 (− 1−1.4) | F (placebo 12wks) | − 0.5 (0.7) | 0.862 | − 0.1 (− 1.9–1.7) | |
| C (HCQ 4wks) | 9.0 (18.6) | 0.258 | 0.6 (− 0.4−1.6) | G (no med. 12wks) | − 1.0 (4.4) | 0.840 | − 0.2 (− 1.8−1.4) | |
| B + C (combined) | 6.7 (16.7) | 0.251 | 0.5 (− 0.4−1.4) | F + G combined | − 0.8 (3.1) | 0.798 | − 0.2 (− 1.7−1.2) | |
| MMEF25-75 (% pred) | A (placebo 4wks) | 3.0 (13.1) | E (HCQ 12wks) | − 8.7 (14.2) | ||||
| B (HCQ 4wks) | − 14.8 (15.2) | 0.010 | − 1.3 (− 2.6−0) | F (placebo 12wks) | − 7.0 (1.4) | 0.858 | 0.1 (− 1.6−1.9) | |
| C (HCQ 4wks) | − 2.6 (8.6) | 0.533 | − 0.5 (− 1.5−0.5) | G (no med. 12wks) | − 11.3 (7.8) | 0.661 | − 0.2 (− 1.8−1.4) | |
| B + C (combined) | − 7.0 (12.3) | 0.114 | − 0.8 (− 1.7−0.2) | F + G combined | − 9.6 (6) | 0.922 | − 0.1 (− 1.5−1.3) | |
| 6MWT distance (% pred) | A (placebo 4wks) | − 0.8 (3.6) | E (HCQ 12wks) | 0.3 (8.5) | ||||
| B (HCQ 4wks) | − 31.0 (n.appl.) | n.appl | − 8.4 (− 14– −2.7) | F (placebo 12wks) | − 0.5 (2.1) | 0.884 | − 0.1 (− 1.9−1.7) | |
| C (HCQ 4wks) | 0.8 (2.1) | 0.576 | 0.5 (− 0.9−2) | G (no med. 12wks) | 8.7 (12.6) | 0.563 | 0.8 (− 0.9−2.4) | |
| B + C (combined) | − 5.6 (14.3) | 0.501 | − 0.4 (− 1.8−0.9) | F + G combined | 5.0 (10.3) | 0.518 | 0.5 (− 1.0−1.9) | |
Data are given as mean (SD); the number of subjects in each group is indicated in Fig. 1. In START or STOP for each parameter 3 comparisons were made and thus after Bonferroni correction a P < 0.017 considered significant
6 MWT 6 min walk test, QoL Quality of life questionnaire, no med. no medication, means withdrawal of HCQ (comparable to placebo), n.a. not available, n.appl. not applicable