Characteristics of pirfenidone nonresponders and responders (n = 29)
| Characteristic . | Nonresponders (n = 17) . | Responders (n = 12) . | P value . |
|---|---|---|---|
| Demographics | |||
| Female, n (%) | 7 (41.2) | 7 (38.3) | .60 |
| Age (SD), y | 54.5 (11.4) | 55.3 (13.5) | .87 |
| Reason for HCT, n (%) | |||
| Acute lymphocytic leukemia | 4 (23.5) | 2 (16.7) | 1.00 |
| Acute myelogenous leukemia | 7 (41.2) | 5 (41.7) | 1.00 |
| Myelodysplastic syndrome | 4 (23.5) | 1 (8.3) | .57 |
| Chronic lymphocytic leukemia | 0 (0.0) | 1 (8.3) | .86 |
| Chronic myelogenous leukemia | 0 (0.0) | 1 (8.3) | .86 |
| Diffuse large B-cell lymphoma | 1 (5.9) | 0 (0.0) | 1.00 |
| Hodgkin lymphoma | 1 (5.9) | 1 (8.3) | 1.00 |
| Hemoglobinopathy | 0 (0.0) | 1 (8.3) | .859 |
| BOS characteristics, n (%) | |||
| Diagnosed by NIH criteria | 15 (88.2) | 10 (83.3) | 1.00 |
| Diagnosed by clinical criteria | 1 (5.9) | 2 (16.7) | .75 |
| Diagnosed by lung biopsy | 1 (5.9) | 0 (0.0) | 1.00 |
| Months from HCT to trial, mean (SD) | 77.3 (80.7) | 47.8 (28.7) | .24 |
| Months from BOS diagnosis to trial, mean (SD) | 37.5 (30.8) | 32.0 (25.3) | .62 |
| Months from HCT to BOS diagnosis, mean (SD) | 39.8 (58.9) | 15.8 (8.0) | .18 |
| Baseline PFTs, mean (SD) | |||
| ppFEV1 | 52.8 (14.7) | 52.4 (12.7) | .94 |
| ppFVC | 72.7 (20.1) | 72.3 (18.6) | .96 |
| Diffusion capacity for carbon monoxide percent predicted | 69.6 (20.5) | 68.9 (19.9) | .93 |
| cGVHD characteristics, n (%) | |||
| Number of affected nonpulmonary organs, mean (SD) | 2.8 (1.4) | 2.4 (1.2) | .80 |
| Skin | 13 (76.5) | 7 (58.3) | .53 |
| Ocula | 12 (70.6) | 6 (50.0) | .46 |
| Oral | 8 (47.1) | 6 (50.0) | 1.00 |
| GI tract | 6 (35.3) | 1 (8.3) | .22 |
| Liver | 3 (17.6) | 2 (16.7) | 1.00 |
| Joint | 5 (29.4) | 2 (16.7) | .35 |
| Genital tract | 1 (5.9) | 1 (8.3) | .81 |
| Concomitant medications used to treat BOS and cGVHD, n (%) | |||
| FAM | 17 (100.0) | 12 (100.0) | NA |
| Prednisone | 13 (76.5) | 10 (83.3) | 1.00 |
| Ibrutinib | 3 (17.6) | 1 (8.3) | .87 |
| Mycophenolate mofetil | 2 (11.8) | 3 (25.0) | .67 |
| Ruxolitinib | 2 (11.8) | 0 (0.0) | .63 |
| Tacrolimus | 7 (41.2) | 5 (41.7) | 1.00 |
| Sirolimus | 4 (23.5) | 2 (16.7) | 1.00 |
| Photopheresis | 0 (0) | 0 (0) | NA |
| Characteristic . | Nonresponders (n = 17) . | Responders (n = 12) . | P value . |
|---|---|---|---|
| Demographics | |||
| Female, n (%) | 7 (41.2) | 7 (38.3) | .60 |
| Age (SD), y | 54.5 (11.4) | 55.3 (13.5) | .87 |
| Reason for HCT, n (%) | |||
| Acute lymphocytic leukemia | 4 (23.5) | 2 (16.7) | 1.00 |
| Acute myelogenous leukemia | 7 (41.2) | 5 (41.7) | 1.00 |
| Myelodysplastic syndrome | 4 (23.5) | 1 (8.3) | .57 |
| Chronic lymphocytic leukemia | 0 (0.0) | 1 (8.3) | .86 |
| Chronic myelogenous leukemia | 0 (0.0) | 1 (8.3) | .86 |
| Diffuse large B-cell lymphoma | 1 (5.9) | 0 (0.0) | 1.00 |
| Hodgkin lymphoma | 1 (5.9) | 1 (8.3) | 1.00 |
| Hemoglobinopathy | 0 (0.0) | 1 (8.3) | .859 |
| BOS characteristics, n (%) | |||
| Diagnosed by NIH criteria | 15 (88.2) | 10 (83.3) | 1.00 |
| Diagnosed by clinical criteria | 1 (5.9) | 2 (16.7) | .75 |
| Diagnosed by lung biopsy | 1 (5.9) | 0 (0.0) | 1.00 |
| Months from HCT to trial, mean (SD) | 77.3 (80.7) | 47.8 (28.7) | .24 |
| Months from BOS diagnosis to trial, mean (SD) | 37.5 (30.8) | 32.0 (25.3) | .62 |
| Months from HCT to BOS diagnosis, mean (SD) | 39.8 (58.9) | 15.8 (8.0) | .18 |
| Baseline PFTs, mean (SD) | |||
| ppFEV1 | 52.8 (14.7) | 52.4 (12.7) | .94 |
| ppFVC | 72.7 (20.1) | 72.3 (18.6) | .96 |
| Diffusion capacity for carbon monoxide percent predicted | 69.6 (20.5) | 68.9 (19.9) | .93 |
| cGVHD characteristics, n (%) | |||
| Number of affected nonpulmonary organs, mean (SD) | 2.8 (1.4) | 2.4 (1.2) | .80 |
| Skin | 13 (76.5) | 7 (58.3) | .53 |
| Ocula | 12 (70.6) | 6 (50.0) | .46 |
| Oral | 8 (47.1) | 6 (50.0) | 1.00 |
| GI tract | 6 (35.3) | 1 (8.3) | .22 |
| Liver | 3 (17.6) | 2 (16.7) | 1.00 |
| Joint | 5 (29.4) | 2 (16.7) | .35 |
| Genital tract | 1 (5.9) | 1 (8.3) | .81 |
| Concomitant medications used to treat BOS and cGVHD, n (%) | |||
| FAM | 17 (100.0) | 12 (100.0) | NA |
| Prednisone | 13 (76.5) | 10 (83.3) | 1.00 |
| Ibrutinib | 3 (17.6) | 1 (8.3) | .87 |
| Mycophenolate mofetil | 2 (11.8) | 3 (25.0) | .67 |
| Ruxolitinib | 2 (11.8) | 0 (0.0) | .63 |
| Tacrolimus | 7 (41.2) | 5 (41.7) | 1.00 |
| Sirolimus | 4 (23.5) | 2 (16.7) | 1.00 |
| Photopheresis | 0 (0) | 0 (0) | NA |
NA, not available.