Summary of exploratory disease outcomes.
| Variables | Pirfenidone, 2403 mg/day | ||||
|---|---|---|---|---|---|
| 2-week Titration Group, n = 32 | 4-week Titration Group, n = 31 | No MMF, n = 23 | MMF, n = 40 | Total, n = 63 | |
| Predicted FVC, %, mean (SD) | |||||
| Baseline | 72.8 (14.39) | 79.4 (13.32) | 78.7 (13.99) | 74.5 (14.19) | 76.0 (14.15) |
| Week 16 | 71.4 (15.14) | 78.7 (13.60) | 75.5 (14.26) | 75.0 (15.13) | 75.2 (14.71) |
| Change from baseline to Week 16* | −0.6 (8.91)* | −0.6 (5.90) | −0.3 (9.50)** | 0.6 (5.82)*** | −0.6 (7.44)* |
| Predicted DLCO, %, mean (SD)† | |||||
| Baseline | 59.3 (14.36) | 60.1 (18.64) | 56.8 (14.92) | 61.4 (17.26) | 59.7 (16.47) |
| Week 16 | 60.6 (19.12)* | 63.4 (18.12) | 57.0 (14.96)** | 64.7 (19.80)*** | 62.0 (18.51) |
| Change from baseline to Week 16* | 0.7 (9.57)* | 3.2 (10.00) | −0.2 (7.12)** | 3.2 (10.85)*** | 2.0 (9.79)* |
| Mahler TDI overall score, mean (SD)†† | |||||
| Baseline | 7.7 (2.61)*** | 7.7 (3.41)*** | 8.4 (2.94)*** | 7.3 (3.01)*** | 7.7 (3.00)** |
| Change from baseline to Week 16 | 0.0 (3.16) | 2.1 (3.40) | −0.3 (3.28) | 1.8 (3.28) | 1.0 (3.41) |
| mRSS total score, mean (SD)††† | |||||
| Baseline | 9.6 (9.66) | 13.3 (9.33) | 11.1 (8.64) | 11.6 (10.22) | 11.4 (9.61) |
| Week 16 | 8.8 (9.18) | 13.6 (10.15) | 11.2 (8.94) | 11.3 (10.55) | 11.2 (9.90) |
| Change from baseline to Week 16 | −1.1 (3.95)** | 0.3 (3.40) | 0.1 (4.32) | −0.7 (3.32)** | −0.4 (3.71)** |
| HAQ-DI score, mean (SD)‡ | |||||
| Baseline | 0.55 (0.624) | 0.58 (0.854) | 0.45 (0.606) | 0.63 (0.807) | 0.57 (0.740) |
| Week 16 | 0.61 (0.719) | 0.53 (0.895) | 0.51 (0.729) | 0.61 (0.852) | 0.57 (0.805) |
| Change from baseline to Week 16 | 0.06 (0.502) | −0.05 (0.310) | 0.06 (0.503) | −0.03 (0.367) | 0.00 (0.419) |
| PtGA score, mean (SD)‡‡ | |||||
| Baseline | 35.7 (27.13) | 29.3 (28.16) | 28.8 (26.67) | 34.7 (28.24) | 32.5 (27.61) |
| Week 16 | 41.7 (25.75) | 31.5 (30.25) | 39.7 (29.36) | 35.0 (27.89) | 36.7 (28.29) |
| Change from baseline to Week 16 | 6.0 (22.25) | 2.3 (19.95) | 11.0 (19.71) | 0.3 (21.05) | 4.2 (21.06) |
↵* n = n – 3.
↵** n = n – 2.
↵*** n = n – 1.
↵† Adjusted for hemoglobin.
↵†† Measured using a scale from +9 to −9. Negative scores indicate more severe dyspnea.
↵††† Higher values indicate greater skin thickness.
↵‡ Visual analog scale scores for HAQ-DI are provided in Supplementary Table 3 (available online at jrheum.org).
↵‡‡ Measured using a visual analog scale (0–100 mm). Higher values indicate more active or severe disease. MMF: mycophenolate mofetil; FVC: forced vital capacity; TDI: Transition Dyspnea Index; mRSS: modified Rodnan skin score; HAQ-DI: Health Assessment Questionnaire–Disability Index; PtGA: patient’s global assessment of disease activity.