Baseline characteristics of study participants (n = 30)
| Demographics | |
| Female, n (%) | 15 (50.0) |
| Age, mean ± SD, y | 54.4 ± 12.2 |
| Reason for HCT | |
| Acute myelogenous leukemia, n (%) | 13 (43.3) |
| Acute lymphocytic leukemia, n (%) | 6 (20.0) |
| Myelodysplastic syndrome, n (%) | 5 (16.7) |
| Chronic lymphocytic leukemia, n (%) | 1 (3.3) |
| Chronic myelogenous leukemia, n (%) | 1 (3.3) |
| Hodgkin lymphoma, n (%) | 2 (6.7) |
| Diffuse large B-cell lymphoma, n (%) | 1 (3.3) |
| Hemoglobinopathy, n (%) | 1 (3.3) |
| BOS | |
| Diagnosed by NIH criteria/lung biopsy, n (%) | 27 (90.0) |
| Diagnosed by clinical criteria, n (%) | 3 (10.0) |
| Median time from transplant to start of trial (IQR), mo | 39.1 (25.4-79.7) |
| Median time from transplant to BOS diagnosis (IQR), mo | 16.2 (9.1-28.1) |
| Median time from BOS diagnosis to start of trial (IQR), mo | 28.5 (13.9-56.8) |
| PFTs | |
| Mean ppFVC prior to HCT (± SD) | 98.6 ± 13.8 |
| Mean ppFEV1 prior to HCT (± SD) | 99.6 ± 17.5 |
| Mean percent predicted DLCO prior to HCT (± SD) | 83.5 ± 18.5 |
| Mean ppFVC prior to STOP-BOS trial (± SD) | 72.0 ± 19.0 |
| Mean ppFEV1 prior to STOP-BOS trial (± SD) | 51.8 ± 14.2 |
| Mean percent predicted DLCO prior to STOP-BOS trial (± SD) | 68.8 ± 19.7 |
| cGVHD characteristics | |
| Overall NIH cGVHD clinical score, mean ± SD | 2.7 ± 0.7 |
| Number of affected organs, mean ± SD | 2.7 ± 1.3 |
| Skin, n (%) | 21 (70.0) |
| Ocular, n (%) | 19 (63.3) |
| Oral, n (%) | 14 (46.7) |
| GI tract, n (%) | 8 (26.7) |
| Liver, n (%) | 6 (20.0) |
| Joint, n (%) | 7 (23.3) |
| Genital tract, n (%) | 2 (6.7) |
| Concomitant medications used to treat BOS and cGVHD | |
| FAM, n (%) | 30 (100.0) |
| Prednisone, n (%) | 24 (80.0) |
| Ibrutinib, n (%) | 4 (13.3) |
| Mycophenolate mofetil, n (%) | 5 (16.7) |
| Ruxolitinib, n (%) | 3 (10.0) |
| Tacrolimus, n (%) | 13 (43.3) |
| Demographics | |
| Female, n (%) | 15 (50.0) |
| Age, mean ± SD, y | 54.4 ± 12.2 |
| Reason for HCT | |
| Acute myelogenous leukemia, n (%) | 13 (43.3) |
| Acute lymphocytic leukemia, n (%) | 6 (20.0) |
| Myelodysplastic syndrome, n (%) | 5 (16.7) |
| Chronic lymphocytic leukemia, n (%) | 1 (3.3) |
| Chronic myelogenous leukemia, n (%) | 1 (3.3) |
| Hodgkin lymphoma, n (%) | 2 (6.7) |
| Diffuse large B-cell lymphoma, n (%) | 1 (3.3) |
| Hemoglobinopathy, n (%) | 1 (3.3) |
| BOS | |
| Diagnosed by NIH criteria/lung biopsy, n (%) | 27 (90.0) |
| Diagnosed by clinical criteria, n (%) | 3 (10.0) |
| Median time from transplant to start of trial (IQR), mo | 39.1 (25.4-79.7) |
| Median time from transplant to BOS diagnosis (IQR), mo | 16.2 (9.1-28.1) |
| Median time from BOS diagnosis to start of trial (IQR), mo | 28.5 (13.9-56.8) |
| PFTs | |
| Mean ppFVC prior to HCT (± SD) | 98.6 ± 13.8 |
| Mean ppFEV1 prior to HCT (± SD) | 99.6 ± 17.5 |
| Mean percent predicted DLCO prior to HCT (± SD) | 83.5 ± 18.5 |
| Mean ppFVC prior to STOP-BOS trial (± SD) | 72.0 ± 19.0 |
| Mean ppFEV1 prior to STOP-BOS trial (± SD) | 51.8 ± 14.2 |
| Mean percent predicted DLCO prior to STOP-BOS trial (± SD) | 68.8 ± 19.7 |
| cGVHD characteristics | |
| Overall NIH cGVHD clinical score, mean ± SD | 2.7 ± 0.7 |
| Number of affected organs, mean ± SD | 2.7 ± 1.3 |
| Skin, n (%) | 21 (70.0) |
| Ocular, n (%) | 19 (63.3) |
| Oral, n (%) | 14 (46.7) |
| GI tract, n (%) | 8 (26.7) |
| Liver, n (%) | 6 (20.0) |
| Joint, n (%) | 7 (23.3) |
| Genital tract, n (%) | 2 (6.7) |
| Concomitant medications used to treat BOS and cGVHD | |
| FAM, n (%) | 30 (100.0) |
| Prednisone, n (%) | 24 (80.0) |
| Ibrutinib, n (%) | 4 (13.3) |
| Mycophenolate mofetil, n (%) | 5 (16.7) |
| Ruxolitinib, n (%) | 3 (10.0) |
| Tacrolimus, n (%) | 13 (43.3) |
DLCO, diffusing capacity of the lungs for carbon monoxide; FAM, fluticasone, azithromycin, montelukast; STOP-BOS, safety and tolerability of pirfenidone for bronchiolitis obliterans syndrome.