Patient characteristics (n = 618)
| Characteristic . | Training cohort (n = 248) . | Validation cohort (n = 367) . |
|---|---|---|
| Median age (range) | 53 y (3 mo-74 y) | 54 y (9 mo-77 y) |
| Pediatric patients (<18 y), n (%) | 23 (9) | 45 (12) |
| Indication for HCT, n (%) | ||
| Acute leukemia | 120 (48) | 197 (54) |
| MDS/MPN | 53 (21) | 103 (28) |
| Lymphoma | 32 (13) | 32 (9) |
| Other malignant | 31 (12) | 18 (5) |
| Nonmalignant | 12 (5) | 17 (5) |
| Donor type, n (%) | ||
| Related | 59 (24) | 75 (20) |
| Unrelated | 183 (74) | 261 (71) |
| Haploidentical | 6 (2) | 31 (8) |
| Cell source, n (%) | ||
| Bone marrow | 39 (16) | 72 (20) |
| Umbilical cord blood | 26 (10) | 18 (5) |
| Peripheral blood stem cells | 183 (74) | 277 (75) |
| HLA match, n (%) | ||
| Matched | 167 (67) | 266 (72) |
| Mismatched | 75 (30) | 70 (19) |
| Haploidentical | 6 (2) | 31 (8) |
| Conditioning regimen intensity, n (%) | ||
| Full | 192 (77) | 222 (60) |
| Reduced | 56 (23) | 145 (40) |
| ATG, n (%) | ||
| Yes | 77 (31) | 168 (46) |
| No | 171 (69) | 199 (54) |
| GVHD prophylaxis, n (%) | ||
| CNI/MTX ± other | 140 (56) | 188 (51) |
| CNI/MMF ± other | 93 (38) | 93 (25) |
| Cyclophosphamide based | 6 (2) | 48 (13) |
| Other | 9 (4) | 19 (5) |
| T-cell depletion | 0 (0) | 5 (1) |
| CNI+sirolimus | 0 (0) | 14 (4) |
| GVHD organ distribution at treatment initiation, n (%) | ||
| Stage 0 in all target organs* | 4 (2) | 5 (1) |
| Isolated skin | 105 (42) | 171 (47) |
| Isolated GI (upper and lower) | 78 (31) | 113 (31) |
| Isolated liver | 0 (0) | 3 (1) |
| ≥2 organs involved | 61 (25) | 75 (21) |
| GVHD grade at treatment initiation,18 n (%) | ||
| I† | 75 (30) | 112 (31) |
| II | 106 (43) | 185 (50) |
| III | 53 (21) | 57 (16) |
| IV | 14 (6) | 13 (4) |
| Onset Minnesota Risk,19 n (%) | ||
| Standard | 190 (77) | 308 (84) |
| High | 58 (23) | 59 (16) |
| Initial corticosteroid dose (methylprednisolone mg/kg) | 1.5 (0.24-2.7) | 1.0 (0.09-3.3) |
| Clinical response after 4 wk of treatment, n (%) | ||
| CR | 138 (56) | 230 (63) |
| PR | 32 (13) | 37 (10) |
| NR | 78 (31) | 100 (27) |
| Long-term outcomes, n (%) | ||
| 1-y NRM | 61 (25) | 81 (22) |
| 1-y Relapse rate | 47 (19) | 51 (14) |
| 1-y OS | 159 (64) | 260 (71) |
| Characteristic . | Training cohort (n = 248) . | Validation cohort (n = 367) . |
|---|---|---|
| Median age (range) | 53 y (3 mo-74 y) | 54 y (9 mo-77 y) |
| Pediatric patients (<18 y), n (%) | 23 (9) | 45 (12) |
| Indication for HCT, n (%) | ||
| Acute leukemia | 120 (48) | 197 (54) |
| MDS/MPN | 53 (21) | 103 (28) |
| Lymphoma | 32 (13) | 32 (9) |
| Other malignant | 31 (12) | 18 (5) |
| Nonmalignant | 12 (5) | 17 (5) |
| Donor type, n (%) | ||
| Related | 59 (24) | 75 (20) |
| Unrelated | 183 (74) | 261 (71) |
| Haploidentical | 6 (2) | 31 (8) |
| Cell source, n (%) | ||
| Bone marrow | 39 (16) | 72 (20) |
| Umbilical cord blood | 26 (10) | 18 (5) |
| Peripheral blood stem cells | 183 (74) | 277 (75) |
| HLA match, n (%) | ||
| Matched | 167 (67) | 266 (72) |
| Mismatched | 75 (30) | 70 (19) |
| Haploidentical | 6 (2) | 31 (8) |
| Conditioning regimen intensity, n (%) | ||
| Full | 192 (77) | 222 (60) |
| Reduced | 56 (23) | 145 (40) |
| ATG, n (%) | ||
| Yes | 77 (31) | 168 (46) |
| No | 171 (69) | 199 (54) |
| GVHD prophylaxis, n (%) | ||
| CNI/MTX ± other | 140 (56) | 188 (51) |
| CNI/MMF ± other | 93 (38) | 93 (25) |
| Cyclophosphamide based | 6 (2) | 48 (13) |
| Other | 9 (4) | 19 (5) |
| T-cell depletion | 0 (0) | 5 (1) |
| CNI+sirolimus | 0 (0) | 14 (4) |
| GVHD organ distribution at treatment initiation, n (%) | ||
| Stage 0 in all target organs* | 4 (2) | 5 (1) |
| Isolated skin | 105 (42) | 171 (47) |
| Isolated GI (upper and lower) | 78 (31) | 113 (31) |
| Isolated liver | 0 (0) | 3 (1) |
| ≥2 organs involved | 61 (25) | 75 (21) |
| GVHD grade at treatment initiation,18 n (%) | ||
| I† | 75 (30) | 112 (31) |
| II | 106 (43) | 185 (50) |
| III | 53 (21) | 57 (16) |
| IV | 14 (6) | 13 (4) |
| Onset Minnesota Risk,19 n (%) | ||
| Standard | 190 (77) | 308 (84) |
| High | 58 (23) | 59 (16) |
| Initial corticosteroid dose (methylprednisolone mg/kg) | 1.5 (0.24-2.7) | 1.0 (0.09-3.3) |
| Clinical response after 4 wk of treatment, n (%) | ||
| CR | 138 (56) | 230 (63) |
| PR | 32 (13) | 37 (10) |
| NR | 78 (31) | 100 (27) |
| Long-term outcomes, n (%) | ||
| 1-y NRM | 61 (25) | 81 (22) |
| 1-y Relapse rate | 47 (19) | 51 (14) |
| 1-y OS | 159 (64) | 260 (71) |
ATG, antithymocyte globulin; CNI, calcineurin inhibitor; MDS, myelodysplastic syndromes; MMF, mycophenolate mofetil; MPN, myeloproliferative neoplasms; MTX, methotrexate.
Reasons for treatment of stage 0 GVHD in all target organs include biopsy-proven GVHD without clinical symptoms.
Grade 0 and I were combined for all analyses.