Table 2.
Recent Trials in Fibrotic Interstitial Lung Disease
| Disease | Trial/Medication | Primary End Point/Objective | Outcomes |
|---|---|---|---|
| Idiopathic pulmonary fibrosis | CAPACITY-004 (PIPF004)19 [Pirfenidone 2,403 mg/d vs 1,197 mg/d vs placebo] |
Change in FVC at 72 wk | Pirfenidone 2,403 mg/d significantly reduced decline in FVC |
| CAPACITY-006 (PIPF006)19 [Pirfenidone 2,403 mg/d vs placebo] |
Change in FVC at 72 wk | No significant difference between groups | |
| ASCEND22 [Pirfenidone 2,403 mg/d vs placebo] |
Change in FVC at 52 wk | Pirfenidone significantly reduced decline in FVC, significantly improved progression-free survival | |
| RECAP26 [Pirfenidone 2,403 mg/d] |
Long-term safety and tolerability | Pirfenidone was safe and generally well tolerated | |
| Loeh et al20 [Retrospective comparison of pirfenidone vs historical control subjects] |
Treatment-elicited changes in lung function | Reduction in annual decline in FVC after initiation of pirfenidone | |
| TOMORROW32 [Nintedanib 50 mg/d vs 50 mg bid vs 100 mg bid vs 150 mg bid vs placebo] |
Rate of decline in FVC at 12 mo | Trend toward a reduced decline in lung function and fewer acute exacerbations with 150 mg bid | |
| INPULSIS-133, 34 [Nintedanib 150 mg bid vs placebo] |
Rate of decline in FVC at 52 wk | Reduced FVC decline with nintedanib | |
| INPULSIS-233, 34 [Nintedanib 150 mg bid vs placebo] |
Rate of decline in FVC at 52 wk | Reduced FVC decline; increased time to first acute exacerbation | |
| Costabel et al.36 [Prespecified subgroup analyses of pooled data from INPULSIS-1 and -2] |
Treatment effect of nintedanib | Nintedanib had a consistent effect on slowing disease progression across several prespecified subgroups | |
| Connective tissue disease-interstitial lung disease | SLS68, 69 [Cyclophosphamide ≤ 2 mg/kg/d vs placebo] |
Percent predicted FVC at 12 mo, after adjusting for baseline FVC | Modest benefit on FVC, dyspnea, skin thickening, and quality of life with cyclophosphamide |
| EUSTAR Rituximab study76 [Retrospective comparison of rituximab with control subjects] |
Change in skin fibrosis | Rituximab improved skin fibrosis and prevented worsening of lung fibrosis | |
| LOTUSS SSc-ILD study72, 73 [Pirfenidone 2,403 mg/d] |
Evaluation of adverse events | Pirfenidone was safe and generally well tolerated | |
| Tacrolimus in polymyositis and dermatomyositis84, 85 [Retrospective comparison of tacrolimus vs conventional therapy] |
Time to relapse or death from respiratory cause or serious adverse event | Event-free survival and disease-free survival significantly longer with tacrolimus | |
| Chronic hypersensitivity pneumonitis | Keir et al60 [Rituximab, pre-to-post] |
Change in predicted percentage of FVC and Dlco | Median improvement in FVC; stable Dlco |
ASCEND = Assessment of Pirfenidone to Confirm Efficacy and Safety in Idiopathic Pulmonary Fibrosis; CAPACITY = Clinical Studies Assessing Pirfenidone in Idiopathic Pulmonary Fibrosis: Research of Efficacy and Safety Outcomes; Dlco = diffusion capacity of the lung for carbon monoxide; EUSTAR = European Scleroderma Trial and Research; LOTUSS = Safety and Tolerability of Pirfenidone in Patients With Systemic Sclerosis−Related Interstitial Lung Disease; SLS = Scleroderma Lung Study; TOMORROW = To Improve Pulmonary Fibrosis With BIBF 1120.