Patient characteristics
| Characteristic . | Test cohort (n = 236) . | Validation cohort 1 (n = 142) . | Validation cohort 2 (n = 129) . | P . |
|---|---|---|---|---|
| Median age, y (range) | 51 (1-73) | 49.5 (1-74) | 48 (1-74) | .840 |
| Indication for HCT, n (%) | .004 | |||
| Acute leukemia | 121 (51.3) | 73 (51.4) | 65 (50.4) | |
| MDS/MPN | 48 (20.3) | 24 (16.9) | 43 (33.2) | |
| Lymphoma | 38 (16.1) | 19 (13.4) | 6 (4.7) | |
| Other malignant | 22 (9.3) | 20 (14.1) | 9 (7.0) | |
| Nonmalignant | 7 (3.0) | 6 (4.2) | 6 (4.7) | |
| Disease risk index at HCT, n (%) | .326 | |||
| Low | 16 (6.8) | 7 (4.9) | 3 (2.3) | |
| Intermediate | 120 (50.8) | 78 (54.9) | 81 (62.8) | |
| High | 61 (25.8) | 38 (26.8) | 31 (24.0) | |
| Very high | 20 (8.5) | 7 (4.9) | 8 (6.2) | |
| Unknown | 19 (8.1) | 12 (8.5) | 6 (4.7) | |
| Donor type, n (%) | .529 | |||
| Related | 67 (28.4) | 46 (32.4) | 34 (26.4) | |
| Unrelated | 169 (71.6) | 96 (67.6) | 95 (73.6) | |
| HLA match, n (%) | .485 | |||
| Matched | 159 (67.4) | 95 (66.9) | 94 (72.9) | |
| Mismatched | 77 (32.6) | 47 (33.1) | 35 (27.1) | |
| Stem cell source, n (%) | .433 | |||
| Marrow | 36 (15.3) | 30 (21.1) | 19 (14.7) | |
| Peripheral blood | 179 (75.8) | 104 (73.2) | 101 (78.3) | |
| Cord blood | 21 (8.9) | 8 (5.7) | 9 (7.0) | |
| Conditioning regimen intensity, n (%) | .007 | |||
| Full | 194 (82.2) | 104 (73.2) | 88 (68.2) | |
| Reduced | 42 (17.8) | 38 (26.8) | 41 (31.8) | |
| Antithymocyte globulin in conditioning, n (%) | .0001 | |||
| Yes | 58 (24.6) | 30 (21.1) | 55 (42.6) | |
| No | 178 (75.4) | 112 (78.9) | 74 (57.4) | |
| GVHD prophylaxis, n (%) | .024 | |||
| CNI/MTX ± other | 144 (61.0) | 84 (59.2) | 61 (47.3) | |
| CNI/MMF ± other | 77 (32.6) | 47 (33.1) | 47 (36.4) | |
| CNI/sirolimus | 1 (0.4) | 0 (0) | 2 (1.6) | |
| Posttransplant cyclophosphamide ± other | 8 (3.4) | 9 (6.3) | 13 (10) | |
| T-cell depleted | 2 (0.9) | 0 (0) | 2 (1.6) | |
| Other | 4 (1.7) | 2 (1.4) | 4 (3.1) | |
| Onset GVHD: median day (range) | 26 (9-275) | 31 (7-204) | 26 (8-180) | .0005 |
| Onset GVHD: organ distribution, n (%) | .891 | |||
| Isolated skin | 116 (49.2) | 67 (47.2) | 68 (52.7) | |
| Isolated GI (upper and/or lower) | 63 (26.7) | 39 (27.5) | 34 (26.3) | |
| Isolated liver | 2 (0.8) | 3 (2.1) | 2 (1.6) | |
| ≥2 organs involved | 55 (23.3) | 33 (23.2) | 25 (19.4) | |
| Onset GVHD grade, n (%) | .112 | |||
| 1 | 73 (30.9) | 39 (27.5) | 51 (39.6) | |
| 2 | 105 (44.5) | 64 (45.1) | 57 (44.2) | |
| 3 | 50 (21.2) | 31 (21.8) | 14 (10.8) | |
| 4 | 8 (3.4) | 8 (5.6) | 7 (5.4) | |
| Minnesota risk score (onset), n (%) | .016 | |||
| Standard | 183 (77.5) | 107 (75.4) | 114 (88.4) | |
| High | 53 (22.5) | 35 (24.6) | 15 (11.6) | |
| Week 1 response, n (%) | .674 | |||
| CR or PR | 114 (48.3) | 62 (43.7) | 61 (47.3) | |
| Nonresponse | 122 (51.7) | 80 (56.3) | 68 (52.7) | |
| Minnesota risk score (week 1), n (%) | .045 | |||
| Standard | 187 (79.2) | 110 (77.5) | 114 (88.4) | |
| High | 49 (20.8) | 32 (22.5) | 15 (11.6) | |
| Long-term outcomes by cohort, % | ||||
| 1-year NRM | 31.3 | 28.9 | 20.5 | .084 |
| 1-year relapse rate | 20.3 | 16.3 | 8.9 | .030 |
| 1-year OS | 56.9 | 59.8 | 72.2 | .019 |
| Characteristic . | Test cohort (n = 236) . | Validation cohort 1 (n = 142) . | Validation cohort 2 (n = 129) . | P . |
|---|---|---|---|---|
| Median age, y (range) | 51 (1-73) | 49.5 (1-74) | 48 (1-74) | .840 |
| Indication for HCT, n (%) | .004 | |||
| Acute leukemia | 121 (51.3) | 73 (51.4) | 65 (50.4) | |
| MDS/MPN | 48 (20.3) | 24 (16.9) | 43 (33.2) | |
| Lymphoma | 38 (16.1) | 19 (13.4) | 6 (4.7) | |
| Other malignant | 22 (9.3) | 20 (14.1) | 9 (7.0) | |
| Nonmalignant | 7 (3.0) | 6 (4.2) | 6 (4.7) | |
| Disease risk index at HCT, n (%) | .326 | |||
| Low | 16 (6.8) | 7 (4.9) | 3 (2.3) | |
| Intermediate | 120 (50.8) | 78 (54.9) | 81 (62.8) | |
| High | 61 (25.8) | 38 (26.8) | 31 (24.0) | |
| Very high | 20 (8.5) | 7 (4.9) | 8 (6.2) | |
| Unknown | 19 (8.1) | 12 (8.5) | 6 (4.7) | |
| Donor type, n (%) | .529 | |||
| Related | 67 (28.4) | 46 (32.4) | 34 (26.4) | |
| Unrelated | 169 (71.6) | 96 (67.6) | 95 (73.6) | |
| HLA match, n (%) | .485 | |||
| Matched | 159 (67.4) | 95 (66.9) | 94 (72.9) | |
| Mismatched | 77 (32.6) | 47 (33.1) | 35 (27.1) | |
| Stem cell source, n (%) | .433 | |||
| Marrow | 36 (15.3) | 30 (21.1) | 19 (14.7) | |
| Peripheral blood | 179 (75.8) | 104 (73.2) | 101 (78.3) | |
| Cord blood | 21 (8.9) | 8 (5.7) | 9 (7.0) | |
| Conditioning regimen intensity, n (%) | .007 | |||
| Full | 194 (82.2) | 104 (73.2) | 88 (68.2) | |
| Reduced | 42 (17.8) | 38 (26.8) | 41 (31.8) | |
| Antithymocyte globulin in conditioning, n (%) | .0001 | |||
| Yes | 58 (24.6) | 30 (21.1) | 55 (42.6) | |
| No | 178 (75.4) | 112 (78.9) | 74 (57.4) | |
| GVHD prophylaxis, n (%) | .024 | |||
| CNI/MTX ± other | 144 (61.0) | 84 (59.2) | 61 (47.3) | |
| CNI/MMF ± other | 77 (32.6) | 47 (33.1) | 47 (36.4) | |
| CNI/sirolimus | 1 (0.4) | 0 (0) | 2 (1.6) | |
| Posttransplant cyclophosphamide ± other | 8 (3.4) | 9 (6.3) | 13 (10) | |
| T-cell depleted | 2 (0.9) | 0 (0) | 2 (1.6) | |
| Other | 4 (1.7) | 2 (1.4) | 4 (3.1) | |
| Onset GVHD: median day (range) | 26 (9-275) | 31 (7-204) | 26 (8-180) | .0005 |
| Onset GVHD: organ distribution, n (%) | .891 | |||
| Isolated skin | 116 (49.2) | 67 (47.2) | 68 (52.7) | |
| Isolated GI (upper and/or lower) | 63 (26.7) | 39 (27.5) | 34 (26.3) | |
| Isolated liver | 2 (0.8) | 3 (2.1) | 2 (1.6) | |
| ≥2 organs involved | 55 (23.3) | 33 (23.2) | 25 (19.4) | |
| Onset GVHD grade, n (%) | .112 | |||
| 1 | 73 (30.9) | 39 (27.5) | 51 (39.6) | |
| 2 | 105 (44.5) | 64 (45.1) | 57 (44.2) | |
| 3 | 50 (21.2) | 31 (21.8) | 14 (10.8) | |
| 4 | 8 (3.4) | 8 (5.6) | 7 (5.4) | |
| Minnesota risk score (onset), n (%) | .016 | |||
| Standard | 183 (77.5) | 107 (75.4) | 114 (88.4) | |
| High | 53 (22.5) | 35 (24.6) | 15 (11.6) | |
| Week 1 response, n (%) | .674 | |||
| CR or PR | 114 (48.3) | 62 (43.7) | 61 (47.3) | |
| Nonresponse | 122 (51.7) | 80 (56.3) | 68 (52.7) | |
| Minnesota risk score (week 1), n (%) | .045 | |||
| Standard | 187 (79.2) | 110 (77.5) | 114 (88.4) | |
| High | 49 (20.8) | 32 (22.5) | 15 (11.6) | |
| Long-term outcomes by cohort, % | ||||
| 1-year NRM | 31.3 | 28.9 | 20.5 | .084 |
| 1-year relapse rate | 20.3 | 16.3 | 8.9 | .030 |
| 1-year OS | 56.9 | 59.8 | 72.2 | .019 |
CNI, calcineurin inhibitor; GI, gastrointestinal; MMF; mycophenolic acid; MTX, methotrexate.