TABLE 1.
Demographics and clinical characteristics of the aPAP patients and effect of the baseline WLL
| Demographic or clinical parameter | Pre-WLL visit# | Post-WLL “Baseline”# | p-value¶ |
| Participants (n) | 18 | 18 | |
| Age at diagnosis (years) | 40±12 | – | – |
| Gender (% female) | 7 (39) | – | – |
| BMI (kg·m−2) | 26±3 | – | – |
| Smoking history | |||
| Current smoker | 3 (17) | – | – |
| Ex-smoker | 2 (44) | – | – |
| Never-smoker | 7 (39) | – | – |
| Symptoms and signs | |||
| Dyspnoea | 17 (94) | – | – |
| Cough | 11 (61) | – | – |
| Fever | 0 (0) | – | – |
| Sputum production | 0 (0) | – | – |
| PAP DSS | 4.0 (2.5–5.0) | 1.0 (1.0–3.0) | <0.001 |
| Spirometry and lung volumes | |||
| FEV1 (% predicted) | 62±17 (n=14) | 76±14 | 0.015 |
| FVC (% predicted) | 61±16 (n=14) | 77±14 | 0.007 |
| FEV1/FVC (% predicted) | 98±12 (n=14) | 102±9.7 | 0.291 |
| TLC (% predicted) | 66±8 (n=10) | 76±11 (n=14) | 0.022 |
| VC (% predicted) | 62±14 (n=15) | 75±14 | 0.011 |
| Pulmonary gas exchange | |||
| DLCO (% predicted) | 41±15 (n=13) | 54±13 | 0.008 |
| PaO2 (mmHg) | 59±15 | 77±14 | <0.001 |
| PA–aO2 (mmHg) | 49±14 | 29±14 | <0.001 |
| PaCO2 (mmHg) | 34±2 | 35±3 | 0.291 |
| Radiological evaluation of the lungs | |||
| Chest CT GGO score | 4.9±0.33 (n=17) | – | – |
| Haematology indices | |||
| WBC ×1000·mL−1 | 7.0±1.7 (n=17) | 8.0±2.0 (n=17) | 0.137 |
| Platelets ×1000·mL−1 | 264±80 (n=17) | 280 ±93 (n=17) | 0.60 |
| Serum biomarkers of aPAP | |||
| GM-CSF autoantibody (mg·mL−1) | 42±54 | 38.9±47.9 (n=17) | 0.85 |
| Cyfra21.1 (ng·mL−1) | 19±15 (n=15) | 7.9±4.9 | 0.0046 |
| CEA (ng·mL−1) | 15±14 (n=15) | 9.2±7.2 (n=16) | 0.138 |
| KL-6 (U·mL−1) | 8817±6407 | 10 224±10 343 (n=17) | 0.630 |
| SF-36 component scores | |||
| General health | 35 (23–58) (n=17) | 85 (73–95) | <0.0001 |
| Health change | 0 (0–25) (n=17) | 88 (25–100) | 0.0023 |
| Physical function | 0 (0–67) (n=17) | 100 (100–100) | 0.0001 |
| Energy/fatigue | 56 (42–68) (n=17) | 78 (59–85) | 0.0098 |
| Pain | 40 (23–48) (n=17) | 75 (54–80) | <0.0001 |
| Social function | 38 (13–69) (n=17) | 81 (63–91) | 0.0006 |
| Emotional well-being | 75 (41–100) (n=17) | 100 (98–100) | 0.0008 |
| Role limitations due to physical health | 45 (50–80) (n=17) | 73 (60–80) | 0.0002 |
| Role limitations due to emotional problems | 13 (0–44) (n=17) | 100 (69–100) | <0.0001 |
Enrolled patients had moderate or severe aPAP as indicated by the results shown for all patients in both recombinant GM-CSF and control groups. The baseline visit occurred a median (interquartile range) of 8 (4–16) days after the scheduled baseline WLL. Data are presented as mean±sd, median (interquartile range) or n (%), unless otherwise indicated. Bold text indicates statistical significance. aPAP: autoimmune pulmonary alveolar proteinosis; WLL: whole lung lavage; BMI: body mass index; PAP: pulmonary alveolar proteinosis; DSS: disease severity score; 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; PaCO2: arterial carbon dioxide tension; CT: computed tomography; GGO: ground glass opacification; WBC: (total) white blood cell count; GM-CSF: granulocyte–macrophage colony-stimulating factor; Cyfra21.1: cytokeratin-19 fragment antigen; CEA: carcinoembryonic antigen; KL-6: Krebs von den Lungen; SF-36: Short Form 36-question Health Survey. #: n<18 indicates missing data; ¶: calculated by comparing corresponding data in patients before and after the baseline bilateral WLL using t-test or Mann–Whitney test.