Table 1.
Baseline data
| Start | Stop | |||
|---|---|---|---|---|
| HCQ | Placebo | HCQ | Placebo | |
| Subjects included [n]/male | 9/2 | 17/7 | 4/2 | 5/2 |
| Age (y) | 7.8 (5.6) | 9.0 (6.8) | 9.3 (6.3) | 8.2 (9.0) |
| O2-sat, in room air (%) | 93.9 (5.8) | 92.6 (6.7) | 93.7 (5.1) | 94.8 (4.0) |
| Resp. rate in room air (/min) | 31.0 (13.6) | 33.7 (15.7) | 23.3 (3.1) | 34.4 (17.3) |
| QoL chILD specific score | 66.9 (24.5) | 71.2 (17.9) | 75.9 (19.3) | 54.9 (31.8) |
| QoL total score | 58.3 (24.7) | 65.2 (17.2) | 63.3 (14.9) | 72.1 (18.7) |
| BMI percentile | 29.2 (37.5) | 34.1 (34.4) | 20.8 (25.4) | 18.7 (15.9) |
| LDH (U/l) | 322.5 (91.2) | 351.1 (91.0) | 330.5 (136.3) | 292 (15.0) |
| FVC (% predicted) | 44.3 (21.2) | 46.7 (20.8) | 51.7 (4.0) | 76.7 (22.5) |
| FEV1 (% predicted) | 43.8 (19.7) | 44.6 (19.2) | 50.7 (3.2) | 71.0 (22.5) |
| MMEF25-75 (% predicted) | 76.7 (31.3) | 71.4 (39.0) | 78.0 (31.2) | 81.7 (30.2) |
| 6MWT distance (% predicted) | 74.2 (28.4) | 58.3 (23.1) | 58.3 (15.0) | 88.0 (19.9) |
| Stable co-medication (%) | ||||
| Prednisolon | 3 (33) | 8 (47) | 0 (0) | 1 (20) |
| Methotrexate/Azathioprine | 1 (11) | 5 (29) | 0 (0) | 1 (20) |
| Azithromycine | 2 (22) | 4 (23) | 2 (50) | 1 (20) |
| Sildenafil/Bosentan | 0 (0) | 7 (41) | 2 (50) | 2 (40) |
| HCQ whole blood level at baseline (ng/ml) | 0 (0) | 0 (0) | 432 (709) | 345 (370) |
Data are given as mean (SD) or number of subjects (%). No differences were detected between the groups
nd not done, 6MWT 6 Minute walk test