Table 1.
Current and potential outcome measures for use in clinical trials of new anti-inflammatory therapies in CF
| Outcome measure | Examples | Reference(s) |
|---|---|---|
| Clinical efficacy | FEV 1, rate of decline of FEV 1, rate of exacerbations | 10, 11, 13, 14, 97, 98 |
| Patient reported outcome | CF-HQR | 102 |
| BAL markers | Neutrophils, bacterial count, elastase IL-8, TNF-α, soluable ICAM-1, LB4 | 122–131 |
| EBC markers | NO, pH, MMP-9 | 132–139 |
| Sputum markers | White cell count, bacterial count, elastase IL-6, IL-8, IL-10, TNF-α | 140–143 |
| Systemic markers | CRP, NE-APC, TNF-α, IL-1, IL-2, IL-8, EGF | 104–119 |
| CT scan | CT score, airway trapping, airway wall thickening | 145–152 |
| MRI | Structural changes, lung perfusion/pulmonary blood flow, ventilation defects | 154–158 |
Note: Table does not include exhaustive list of examples for each outcome measure. For comprehensive review of outcome measures in clinical trials for CF patients/ candidate biomarkers for CF airway inflammation. See references 74 and 121.
Abbreviations: FEV 1, forced expiratory volume in 1 second; IL, interleukin; TNF, tumor necrosis factor; L, leukotriene; NO, nitric oxide; MMP, matrix metalloprotein; CRP, C-reactive protein; NE-APC, neutrophil antiproteinase complex; EGF, endothelial growth factor; CT, computed tomography; MRI, magnetic resonance imaging; CF, cystic fibrosis.