TABLE 2.
Comparability of the patient groups after the scheduled baseline WLL
| End-point | Control# | rGM-CSF# | p-value ¶ |
| Participants (n) | 9 | 9 | |
| PAP DSS, median (IQR) | 1.0 (1.0–3.5) | 1.0 (1.0, 1.0) | 0.29 |
| Spirometry and lung volumes | |||
| FEV1 (% predicted) | 77±11 | 75±18 | 0.82 |
| FVC (% predicted) | 76±12 | 78±17 | 0.76 |
| TLC (% predicted) | 73±6.0 (n=6) | 79±14 (n=8) | 0.31 |
| VC (% predicted) | 74±11 | 76±17 | 0.83 |
| Pulmonary gas exchange | |||
| DLCO (% predicted) | 52±12 | 55±14 | 0.62 |
| PaO2 (mmHg) | 72±15 | 82±11 | 0.15 |
| PA–aO2 (mmHg) | 33±15 | 26±12 | 0.28 |
| Haematology indices | |||
| WBC (×1000·mL−1) | 7.5±1.7 | 8.7±2.7 (n=8) | 0.28 |
| Platelets (×1000·mL−1) | 290±111 | 269±73 (n=8) | 0.64 |
| SF-36 general health score | 77±29 | 85±15 | 0.33 |
| Biomarkers | |||
| GM-CSF autoantibody (mg·mL−1) | 25±22 (n=8) | 52±61 | 0.23 |
| Cyfra21.1 (ng·mL−1) | 9.5±6.4 | 5.5±1.7 | 0.07 |
| CEA (ng·mL−1) | 12±9 (n=8) | 6.3±3.2 (n=8) | 0.11 |
| KL-6 (U·mL−1) | 12 142±7049 (n=8) | 8518±12 787 | 0.49 |
Enrolled patients received a scheduled, baseline WLL a median of 8 days (IQR 4–16 days) after enrolment. Data are presented as mean±sd, unless otherwise indicated. WLL: whole lung lavage; rGM-CSF: recombinant granulocyte–macrophage colony-stimulating factor; PAP: pulmonary alveolar proteinosis; DSS: disease severity score; IQR: interquartile range; FEV1: forced expiratory volume in 1 s; FVC: forced vital capacity; TLC: total lung capacity; VC: (slow) vital capacity; DLCO: diffusing capacity of the lung for carbon monoxide; PaO2: arterial oxygen tension; PA–aO2: alveolar–arterial oxygen tension difference; WBC: (total) white blood cell count; SF-36: Short Form 36-question Health Survey; Cyfra21.1: cytokeratin-19 fragment antigen; CEA: carcinoembryonic antigen; KL-6: Krebs von den Lungen. #: n<9 indicates missing data; ¶: comparison of the rGM-CSF and control groups using t-test.