Patient characteristics of the UM discovery and validation sets and FHCRC validation set
| Characteristic . | UM discovery cohort (n = 35) . | UM validation cohort (n = 109) . | P value (difference between discovery and validation) . | FHCRC validation cohort (n = 211) . | |||||||
|---|---|---|---|---|---|---|---|---|---|---|---|
| No cGVHD (n = 18) . | De novo cGVHD (n = 17) . | P value . | No cGVHD (n = 64) . | De novo cGVHD (n = 45) . | P value . | No cGVHD (n = 33) . | New-onset cGVHD (n = 86) . | Established cGVHD (n = 92) . | P value (difference between no cGVHD and new-onset cGVHD) . | ||
| Age, y | |||||||||||
| Median | 26 | 45 | .12 | 41 | 50 | .03 | .32 | 54 | 52 | 52 | .75 |
| Range | 0-66 | 19-58 | 0–67 | 10–67 | 22-72 | 19-79 | 19-74 | ||||
| Diagnosis | |||||||||||
| Malignant* | 13 (72%) | 17 (100%) | <.05 | 53 (83%) | 44 (98%) | .01 | .43 | 33 (100) | 84 (98) | 88 (96) | .38 |
| Nonmalignant† | 5 (28%) | 0 | 11 (17%) | 1 (2%) | 0 | 2 (2) | 4 (4) | ||||
| Disease status at HCT‡ | |||||||||||
| Low | 4 (31%) | 6 (35%) | .65 | 22 (42%) | 19 (43%) | .71 | — | 11 (33) | 38 (44) | 33 (36) | .45 |
| Intermediate | 7 (54%) | 6 (35%) | 17 (32%) | 11 (25%) | 12 (36) | 30 (35) | 29 (32) | ||||
| High | 2 (15%) | 5 (30%) | 14 (26%) | 14 (32%) | 10 (30) | 18 (21) | 29 (32) | ||||
| Donor type | |||||||||||
| Matched sibling | 13 (72%) | 12 (71%) | 1.0 | 40 (63%) | 25 (56%) | .55 | .81 | 18 (55) | 30 (35) | 36 (39) | .05 |
| Other | 5 (28%) | 5 (29%) | 24 (37%) | 20 (44%) | 15 (45) | 56 (65) | 56 (61) | ||||
| Source | |||||||||||
| Bone marrow | 8 (44%) | 3 (18%) | .15 | 18 (28%) | 6 (13%) | .10 | .69 | 7 (21) | 4 (5) | 9 (10) | .01 |
| Cord blood | 0 | 0 | 3 (5%) | 1 (3%) | 0 | 4 (5) | 0 | ||||
| Peripheral blood | 10 (56%) | 14 (82%) | 43 (67%) | 38 (84%) | 26 (79) | 78 (91) | 83 (90) | ||||
| Conditioning intensity | |||||||||||
| Full | 15 (83%) | 12 (71%) | .44 | 48 (75%) | 33 (73%) | 1.0 | .49 | 19 (58) | 50 (58) | 55 (60) | .96 |
| Reduced | 3 (17%) | 5 (29%) | 16 (25%) | 12 (27%) | 14 (42) | 36 (42) | 37 (40) | ||||
| aGVHD | |||||||||||
| 0 | 18 (100%) | 17 (100%) | 39 (61%) | 45 (100%) | — | — | 9 (27) | 16 (19) | 27 (29) | .30 | |
| I-IV | 0 | 0 | 25 (39%) | 0 | 24 (73) | 70 (81) | 65 (71) | ||||
| NIH global severity | |||||||||||
| Mild | — | — | — | — | 4 (9%) | — | .04 | — | 3 (3) | 10 (11) | — |
| Moderate | — | 6 (35%) | — | 25 (56%) | — | 46 (53) | 57 (62) | ||||
| Severe | — | 11 (59%) | — | 16 (35%) | — | 37 (43) | 25 (27) | ||||
| Characteristic . | UM discovery cohort (n = 35) . | UM validation cohort (n = 109) . | P value (difference between discovery and validation) . | FHCRC validation cohort (n = 211) . | |||||||
|---|---|---|---|---|---|---|---|---|---|---|---|
| No cGVHD (n = 18) . | De novo cGVHD (n = 17) . | P value . | No cGVHD (n = 64) . | De novo cGVHD (n = 45) . | P value . | No cGVHD (n = 33) . | New-onset cGVHD (n = 86) . | Established cGVHD (n = 92) . | P value (difference between no cGVHD and new-onset cGVHD) . | ||
| Age, y | |||||||||||
| Median | 26 | 45 | .12 | 41 | 50 | .03 | .32 | 54 | 52 | 52 | .75 |
| Range | 0-66 | 19-58 | 0–67 | 10–67 | 22-72 | 19-79 | 19-74 | ||||
| Diagnosis | |||||||||||
| Malignant* | 13 (72%) | 17 (100%) | <.05 | 53 (83%) | 44 (98%) | .01 | .43 | 33 (100) | 84 (98) | 88 (96) | .38 |
| Nonmalignant† | 5 (28%) | 0 | 11 (17%) | 1 (2%) | 0 | 2 (2) | 4 (4) | ||||
| Disease status at HCT‡ | |||||||||||
| Low | 4 (31%) | 6 (35%) | .65 | 22 (42%) | 19 (43%) | .71 | — | 11 (33) | 38 (44) | 33 (36) | .45 |
| Intermediate | 7 (54%) | 6 (35%) | 17 (32%) | 11 (25%) | 12 (36) | 30 (35) | 29 (32) | ||||
| High | 2 (15%) | 5 (30%) | 14 (26%) | 14 (32%) | 10 (30) | 18 (21) | 29 (32) | ||||
| Donor type | |||||||||||
| Matched sibling | 13 (72%) | 12 (71%) | 1.0 | 40 (63%) | 25 (56%) | .55 | .81 | 18 (55) | 30 (35) | 36 (39) | .05 |
| Other | 5 (28%) | 5 (29%) | 24 (37%) | 20 (44%) | 15 (45) | 56 (65) | 56 (61) | ||||
| Source | |||||||||||
| Bone marrow | 8 (44%) | 3 (18%) | .15 | 18 (28%) | 6 (13%) | .10 | .69 | 7 (21) | 4 (5) | 9 (10) | .01 |
| Cord blood | 0 | 0 | 3 (5%) | 1 (3%) | 0 | 4 (5) | 0 | ||||
| Peripheral blood | 10 (56%) | 14 (82%) | 43 (67%) | 38 (84%) | 26 (79) | 78 (91) | 83 (90) | ||||
| Conditioning intensity | |||||||||||
| Full | 15 (83%) | 12 (71%) | .44 | 48 (75%) | 33 (73%) | 1.0 | .49 | 19 (58) | 50 (58) | 55 (60) | .96 |
| Reduced | 3 (17%) | 5 (29%) | 16 (25%) | 12 (27%) | 14 (42) | 36 (42) | 37 (40) | ||||
| aGVHD | |||||||||||
| 0 | 18 (100%) | 17 (100%) | 39 (61%) | 45 (100%) | — | — | 9 (27) | 16 (19) | 27 (29) | .30 | |
| I-IV | 0 | 0 | 25 (39%) | 0 | 24 (73) | 70 (81) | 65 (71) | ||||
| NIH global severity | |||||||||||
| Mild | — | — | — | — | 4 (9%) | — | .04 | — | 3 (3) | 10 (11) | — |
| Moderate | — | 6 (35%) | — | 25 (56%) | — | 46 (53) | 57 (62) | ||||
| Severe | — | 11 (59%) | — | 16 (35%) | — | 37 (43) | 25 (27) | ||||
Malignant diseases included acute lymphoblastic leukemia, acute myelogenous leukemia, chronic lymphocytic leukemia, chronic myelogenous leukemia, chronic myelomonocytic leukemia, Hodgkin lymphoma, juvenile myelomonocytic leukemia, Kostmann syndrome, non-Hodgkin lymphoma, multiple myeloma, myelodysplastic syndrome, myeloproliferative disorder, paroxysmal nocturnal hematuria, and prolymphocytic leukemia.
Nonmalignant disease included malignant infantile osteopetrosis, severe aplastic anemia, sickle cell anemia, thalassemia, severe combined immunodeficiency disorder, X-linked lymphoproliferative disorder.
Low-, intermediate-, or high-risk disease status according to Center for International Blood and Marrow Transplant Research guidance.