Patient demographics, baseline characteristics, and disease characteristics
| . | Itacitinib . | Total (N = 29) . | |
|---|---|---|---|
| First line (n = 12) . | Steroid refractory* (n = 17) . | ||
| Itacitinib dose received, n (%) | |||
| 200 mg qd | 6 (50.0) | 8 (47.1) | 14 (48.3) |
| 300 mg qd | 6 (50.0) | 9 (52.9) | 15 (51.7) |
| Age, median (range), y | 55.0 (27-71) | 49.0 (18-64) | 51.0 (18-71) |
| Sex, n (%) | |||
| Male | 6 (50.0) | 14 (82.4) | 20 (69.0) |
| Female | 6 (50.0) | 3 (17.6) | 9 (31.0) |
| Race, n (%) | |||
| White | 10 (83.3) | 14 (82.4) | 24 (82.8) |
| African American | 1 (8.3) | 1 (5.9) | 2 (6.9) |
| Asian | 1 (8.3) | 1 (5.9) | 2 (6.9) |
| Other | 0 (0) | 1 (5.9) | 1 (3.4) |
| ECOG PS, n (%) | |||
| 0 | 2 (16.7) | 0 (0) | 2 (6.9) |
| 1 | 6 (50.0) | 7 (41.2) | 13 (44.8) |
| 2 | 4 (33.3) | 9 (52.9) | 13 (44.8) |
| 3 | 0 (0) | 1 (5.9) | 1 (3.4) |
| Underlying malignancies, n (%) | |||
| Acute myeloid leukemia | 6 (50.0) | 5 (29.4) | 11 (37.9) |
| Acute lymphoblastic leukemia | 1 (8.3) | 6 (35.3) | 7 (24.1) |
| Lymphoma | 1 (8.3) | 0 (0) | 1 (3.4) |
| Myelodysplastic syndrome | 4 (33.3) | 2 (11.8) | 6 (20.7) |
| Other | 0 (0) | 4 (23.5) | 4 (13.8) |
| Donor type, n (%) | |||
| Matched related | 1 (8.3) | 5 (29.4) | 6 (20.7) |
| Matched unrelated | 3 (25.0) | 8 (47.1) | 11 (37.9) |
| Haploidentical related (nonsibling) | 1 (8.3) | 0 (0) | 1 (3.4) |
| Haploidentical unrelated | 2 (16.7) | 0 (0) | 2 (6.9) |
| Mismatched unrelated | 4 (33.3) | 4 (23.5) | 8 (27.6) |
| Other | 1 (8.3) | 0 (0) | 1 (3.4) |
| Graft type, n (%) | |||
| Peripheral blood stem cells | 8 (66.7) | 13 (76.5) | 21 (72.4) |
| Bone marrow | 3 (25.0) | 3 (17.6) | 6 (20.7) |
| Umbilical cord blood | 0 (0) | 1 (5.9) | 1 (3.4) |
| Stem cells and cord blood | 1 (8.3) | 0 (0) | 1 (3.4) |
| Conditioning regimen, n (%) | |||
| Myeloablative | 9 (75.0) | 11 (64.7) | 20 (69.0) |
| Reduced intensity | 2 (16.7) | 6 (35.3) | 8 (27.6) |
| Other | 1 (8.3) | 0 (0) | 1 (3.4) |
| History of cytopenias, n (%) | |||
| Anemia | 9 (75.0) | 6 (35.3) | 15 (51.7) |
| Thrombocytopenia | 2 (16.7) | 11 (64.7) | 13 (44.8) |
| Pancytopenia | 3 (25.0) | 5 (29.4) | 8 (27.6) |
| Febrile neutropenia | 2 (16.7) | 3 (17.6) | 5 (17.2) |
| Neutropenia | 2 (16.7) | 3 (17.6) | 5 (17.2) |
| Leukopenia | 1 (8.3) | 1 (5.9) | 2 (6.9) |
| GVHD characteristics, n (%) | |||
| Organ involvement | |||
| Lower GI | 6 (50.0) | 11 (64.7) | 17 (58.6) |
| Skin | 4 (33.3) | 9 (52.9) | 13 (44.8) |
| Upper GI | 5 (41.7) | 4 (23.5) | 9 (31.0) |
| Liver | 0 (0) | 5 (29.4) | 5 (17.2) |
| Isolated skin | 4 (33.3) | 2 (11.8) | 6 (20.7) |
| Skin plus upper GI | 0 (0) | 2 (11.8) | 2 (6.9) |
| >1 organ involved | 11 (91.7) | 15 (88.2) | 26 (89.7) |
| ≥2 organs involved | 4 (33.3) | 10 (58.8) | 14 (48.3) |
| Minnesota grade, n (%) | |||
| II to IV | 11 (91.7) | 17 (100) | 28 (96.6) |
| III/IV | 5 (41.7) | 13 (76.5) | 18 (62.1) |
| CIBMTR grade, n (%) | |||
| B to D | 11 (91.7) | 17 (100.0) | 28 (96.6) |
| C/D | 7 (58.3) | 10 (58.8) | 17 (58.6) |
| Minnesota risk score, n (%) | |||
| Standard | 7 (58.3) | 4 (23.5) | 11 (37.9) |
| High | 5 (41.7) | 13 (76.5) | 18 (62.1) |
| . | Itacitinib . | Total (N = 29) . | |
|---|---|---|---|
| First line (n = 12) . | Steroid refractory* (n = 17) . | ||
| Itacitinib dose received, n (%) | |||
| 200 mg qd | 6 (50.0) | 8 (47.1) | 14 (48.3) |
| 300 mg qd | 6 (50.0) | 9 (52.9) | 15 (51.7) |
| Age, median (range), y | 55.0 (27-71) | 49.0 (18-64) | 51.0 (18-71) |
| Sex, n (%) | |||
| Male | 6 (50.0) | 14 (82.4) | 20 (69.0) |
| Female | 6 (50.0) | 3 (17.6) | 9 (31.0) |
| Race, n (%) | |||
| White | 10 (83.3) | 14 (82.4) | 24 (82.8) |
| African American | 1 (8.3) | 1 (5.9) | 2 (6.9) |
| Asian | 1 (8.3) | 1 (5.9) | 2 (6.9) |
| Other | 0 (0) | 1 (5.9) | 1 (3.4) |
| ECOG PS, n (%) | |||
| 0 | 2 (16.7) | 0 (0) | 2 (6.9) |
| 1 | 6 (50.0) | 7 (41.2) | 13 (44.8) |
| 2 | 4 (33.3) | 9 (52.9) | 13 (44.8) |
| 3 | 0 (0) | 1 (5.9) | 1 (3.4) |
| Underlying malignancies, n (%) | |||
| Acute myeloid leukemia | 6 (50.0) | 5 (29.4) | 11 (37.9) |
| Acute lymphoblastic leukemia | 1 (8.3) | 6 (35.3) | 7 (24.1) |
| Lymphoma | 1 (8.3) | 0 (0) | 1 (3.4) |
| Myelodysplastic syndrome | 4 (33.3) | 2 (11.8) | 6 (20.7) |
| Other | 0 (0) | 4 (23.5) | 4 (13.8) |
| Donor type, n (%) | |||
| Matched related | 1 (8.3) | 5 (29.4) | 6 (20.7) |
| Matched unrelated | 3 (25.0) | 8 (47.1) | 11 (37.9) |
| Haploidentical related (nonsibling) | 1 (8.3) | 0 (0) | 1 (3.4) |
| Haploidentical unrelated | 2 (16.7) | 0 (0) | 2 (6.9) |
| Mismatched unrelated | 4 (33.3) | 4 (23.5) | 8 (27.6) |
| Other | 1 (8.3) | 0 (0) | 1 (3.4) |
| Graft type, n (%) | |||
| Peripheral blood stem cells | 8 (66.7) | 13 (76.5) | 21 (72.4) |
| Bone marrow | 3 (25.0) | 3 (17.6) | 6 (20.7) |
| Umbilical cord blood | 0 (0) | 1 (5.9) | 1 (3.4) |
| Stem cells and cord blood | 1 (8.3) | 0 (0) | 1 (3.4) |
| Conditioning regimen, n (%) | |||
| Myeloablative | 9 (75.0) | 11 (64.7) | 20 (69.0) |
| Reduced intensity | 2 (16.7) | 6 (35.3) | 8 (27.6) |
| Other | 1 (8.3) | 0 (0) | 1 (3.4) |
| History of cytopenias, n (%) | |||
| Anemia | 9 (75.0) | 6 (35.3) | 15 (51.7) |
| Thrombocytopenia | 2 (16.7) | 11 (64.7) | 13 (44.8) |
| Pancytopenia | 3 (25.0) | 5 (29.4) | 8 (27.6) |
| Febrile neutropenia | 2 (16.7) | 3 (17.6) | 5 (17.2) |
| Neutropenia | 2 (16.7) | 3 (17.6) | 5 (17.2) |
| Leukopenia | 1 (8.3) | 1 (5.9) | 2 (6.9) |
| GVHD characteristics, n (%) | |||
| Organ involvement | |||
| Lower GI | 6 (50.0) | 11 (64.7) | 17 (58.6) |
| Skin | 4 (33.3) | 9 (52.9) | 13 (44.8) |
| Upper GI | 5 (41.7) | 4 (23.5) | 9 (31.0) |
| Liver | 0 (0) | 5 (29.4) | 5 (17.2) |
| Isolated skin | 4 (33.3) | 2 (11.8) | 6 (20.7) |
| Skin plus upper GI | 0 (0) | 2 (11.8) | 2 (6.9) |
| >1 organ involved | 11 (91.7) | 15 (88.2) | 26 (89.7) |
| ≥2 organs involved | 4 (33.3) | 10 (58.8) | 14 (48.3) |
| Minnesota grade, n (%) | |||
| II to IV | 11 (91.7) | 17 (100) | 28 (96.6) |
| III/IV | 5 (41.7) | 13 (76.5) | 18 (62.1) |
| CIBMTR grade, n (%) | |||
| B to D | 11 (91.7) | 17 (100.0) | 28 (96.6) |
| C/D | 7 (58.3) | 10 (58.8) | 17 (58.6) |
| Minnesota risk score, n (%) | |||
| Standard | 7 (58.3) | 4 (23.5) | 11 (37.9) |
| High | 5 (41.7) | 13 (76.5) | 18 (62.1) |
qd, once daily.
Of 18 patients with steroid-refractory aGVHD, 1 patient randomized to 300 mg itacitinib did not receive study treatment and was excluded from the safety and efficacy analyses.