Table 2.
Effects of Continuous Molgramostim on Primary and Selected Secondary Outcome Variables After 24 Weeks of Treatment. *
| Value at 24 Weeks |
Change from Baseline |
Estimated Difference |
|||
|---|---|---|---|---|---|
| Variable | Molgramostim (N = 47) |
Placebo (N = 47) |
Molgramostim (N = 47) |
Placebo (N = 47) |
Continuous Molgramostim vs. Placebo (95% CI) † |
| Pulmonary gas exchange | |||||
| A-aDO2,mm Hg ‡ (R-FAS) | 26.4±13.7 | 31.6±12.7 | −11.9±14.9 | −7.0±11.4 | −6.2 (−11.7 to −0.8) |
| DLCO, percentage of predicted | 63.3±22.5 | 53.6±15.5 | 11.5±17.4 | 4.0±11.0 | 7.8 (2.3 to 13.3) |
| Radiological evaluation of the lungs | |||||
| GGO score § | 7.5±3.6 | 10.0±3.5 | −3.4±3.8 | −1.2±2.6 | −2.5 (−3.7 to −1.2) |
| Functional health status | |||||
| SGRQ total score ¶ | 35.1±21.3 | 38.9±23.7 | −12.1±14.3 | −5.2±13.0 | −7.4 (−13.1 to −1.6) |
| Distance walked in six minutes, m | 450±135 | 451±145 | 38.7±95.6 | 4.3±109.0 | 24.6 (−15.3 to 64.4) |
Plus-minus values are empirical means ± SD. A-aDO2 denotes alveolar-arterial difference in oxygen concentration, CI confidence interval, DLCO diffusion capacity of the lungs for carbon monoxide, GGO ground glass opacification. See Table S3 in the Supplemental Appendix for the results of other end points.
In the revised full analysis set (R-FAS), A-aDO2 results for four patients (described in Table 1) were invalid and treated as missing data. See Table S3 in the Supplemental Appendix) and text for additional details.
Between-group differences for the change from baseline are expressed as least square means and 95% confidence intervals with use of an analysis of covariates model (all treatment groups included in the same model) with treatment, WLL within 2 months prior to Baseline (stratification) and geographic region (Japan vs other countries) as factors and Baseline values as covariates.
The GGO scores range from 0 to 15, with higher scores indicating a higher proportion of the area of the chest CT scan images corresponding to lung parenchyma affected by ground glass opacification, an indication of the abnormal accumulation of surfactant sediment in patients with aPAP.
Scores on the SGRQ range from 0 to 100, with higher scores indicating more severe effects on a patient’s functional health status.