Table 2.
Clinical Trials of Granulocyte/Macrophage Colony–Stimulating Factor Augmentation Therapy Reported in the Medical Literature
| Year | First Author | Trial Design | Subjects (n) | Drug | Route | Duration | a-aDo2 | DlCO | CT/X-Ray | 6MWT-D | TEAE | Reference |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1996 | Seymour | First in human, single site, open label | 1 | Molgramostim | s.c. | 70 d | Improved | NR | Improved | NR | None | 204 |
| 2001 | Seymour | Phase I/II, single site, open label | 14 | Molgramostim/sargramostim | s.c. | 6–12 wk | Improved | Improved | Improved | Improved | None | 165 |
| 2004 | Arai | First in human single site, open label | 1 | Molgramostim | Inhaled | 24 wk | Improved | Improved | Improved | NR | None | 211 |
| 2005 | Tazawa | Phase I/II, single site, open label | 3 | Molgramostim | Inhaled | 24 wk | Improved | NR | NR | NR | None | 210 |
| 2006 | Wylam | Retrospective, observational | 12 | Sargramostim | Inhaled | Up to 64 wk | Improved | Improved | Improved | NR | None | 213 |
| 2006 | Venkateshiah | Phase I/II, single site, open label | 25 | Sargramostim | s.c. | 24 wk | Improved | Improved | Improved | Improved | None | 208 |
| 2010 | Tazawa | Phase I/II, multisite, open label | 35 | Sargramostim | Inhaled | 24 wk | Improved | Improved | Improved | Improved | None | 215 |
| 2012 | Ohashi | Phase II, single site, open label | 6 | Sargramostim | Inhaled | 12 wk | Improved | NR | NR | NR | None | 222 |
| 2014 | Papiris | Retrospective, observational | 6 | Sargramostim | Inhaled | 14–65 mo | Improved | Improved | Improved | NR | None | 214 |
| 2016 | Campo | Phase II, single site, open label | 18 | Sargramostim* | Inhaled | 55 mo | Improved | Improved | Not significant | NR | None | 227 |
| 2019 | Tazawa | Phase III, multisite, randomized, double blind, placebo controlled | 64 | Sargramostim | Inhaled | 24 wk | Improved | Improved | Improved | Not significant | None | 224 |
| 2020 | Tian | Phase II, multisite, randomized, open label | 36 | Molgramostim | Inhaled | 24 wk | No change | Improved | No change | No change | None | 223 |
| 2020 | Trapnell | Phase II, multinational, randomized, double blind, placebo controlled | 138 | Molgramostim | Inhaled | 24 wk | Improved | Improved | Improved | Not significant | None | 225 |
Definition of abbreviations: 6MWT-D = 6-minute-walk test distance (distance covered during test); a-aDo2 = alveolar–arterial difference in oxygen concentration; CT = computed tomography (of the chest); NR = not reported; s.c. = subcutaneous; TEAE = treatment-emergent adverse events.
*
Whole-lung lavage versus whole-lung lavage plus inhaled granulocyte/macrophage colony–stimulating factor.