Baseline characteristics
| Characteristic (overall percentages of entire evaluable cohort; n = 243) . | No L-aGVHD or cGVHD (n = 132) . | L-aGVHD (n = 60) . | cGVHD (n = 51) . |
|---|---|---|---|
| Diagnoses | |||
| Malignant (n = 168; 69.1%) | 78 (59) | 49 (81.7) | 41 (80.4) |
| ALL, n | 33 | 23 | 18 |
| MDS/AML, n | 37 | 21 | 15 |
| Mixed lineage acute leukemia, n | 0 | 0 | 1 |
| NHL, n | 4 | 3 | 3 |
| JMML, n | 3 | 2 | 1 |
| CML, n | 1 | 0 | 3 |
| Nonmalignant (n = 75; 30.9%) | 54 (41) | 11 (18.3) | 10 (19.6) |
| Sickle cell anemia, n | 12 | 4 | 4 |
| Severe aplastic anemia, n | 12 | 1 | 1 |
| Thalassemia, n | 4 | 1 | 0 |
| Primary immunodeficiency disorder, n* | 12 | 2 | 1 |
| Inherited marrow failure syndrome, n† | 5 | 1 | 2 |
| Other nonmalignant disorder, n‡ | 9 | 2 | 2 |
| Sex | |||
| Male (55.6%) | 72 (54.5) | 30 (50) | 33 (64.7) |
| Female (44.4%) | 60 (45.5) | 30 (50) | 18 (35.3) |
| Age at transplant | |||
| Median (IQR) (range), y | 9.3 (4.6-12.9) (0.2-17.9) | 7.7 (3.1-12.2) (0.9-18) | 11.9 (7.8-14.6) (2-18) |
| Donor and HLA match | |||
| HLA-matched sibling donor (8/8) | 51 (38.6) | 13 (21.7) | 8 (15.7) |
| HLA-matched nonsibling family donor | 3 (2.3) | 3 (5) | 0 (0) |
| Haploidentical family donor (with PTCy) | 2 (1.5) | 1 (1.7) | 1 (2) |
| HLA-matched unrelated donor (8/8) | 48 (36.4) | 21 (35) | 22 (43.1) |
| HLA-mismatched unrelated donor (≤7/8) | 9 (6.8) | 9 (15) | 12 (23.5) |
| HLA-matched cord blood (6/6) | 9 (6.8) | 4 (6.6) | 1 (2) |
| HLA-mismatched cord blood (≤5/6) | 9 (6.8) | 7 (11.7) | 6 (11.7) |
| Double cord blood | 1 (0.8) | 2 (3.3) | 1 (2) |
| Stem cell source | |||
| Bone marrow (62.6%) | 94 (71.2) | 33 (55) | 25 (49.0) |
| PBSC (20.6%) | 19 (14.4) | 13 (21.7) | 18 (35.3) |
| Cord blood (15.2%) | 18 (13.6) | 12 (20) | 7 (13.7) |
| Double cord blood (1.6%) | 1 (0.8) | 2 (3.3) | 1 (2.0) |
| Conditioning regimen | |||
| Myeloablative (86.8%) | 111 (84.1) | 55 (91.7) | 45 (88.2) |
| TBI 1200-1320 cGY/CY ± other, n | 30 | 23 | 19 |
| Bu + CY ± Mel, n | 27 | 17 | 14 |
| Bu + Flu ± Mel, n | 44 | 10 | 11 |
| Bu + Flu + 200-400 cGY TBI, n | 6 | 4 | 1 |
| TBI 1200 cGY + other, n | 3 | 0 | 0 |
| Treosulfan + Flu + 200 cGY TBI, n | 1 | 1 | 0 |
| Reduced intensity (13.2%) | 21 (15.9) | 5 (8.3) | 6 (11.8) |
| Cy/Flu + TBI 200-400 cGY, n | 7 | 2 | 0 |
| Cy ± Flu, n | 4 | 0 | 2 |
| TBI 300 cGY (+ serotherapy), n | 3 | 0 | 0 |
| Flu/Mel (± serotherapy ± TT), n | 3 | 2 | 2 |
| Other, n | 4 | 1 | 2 |
| Serotherapy | |||
| Rabbit ATG (31.3%) | 43 (32.5) | 20 (33.3) | 13 (25.5) |
| Horse ATG (13.1%) | 17 (12.9) | 4 (6.7) | 11 (21.6) |
| Alemtuzumab (10.7%) | 20 (15.2) | 4 (6.7) | 2 (3.9) |
| None (44.9%) | 52 (39.4) | 32 (53.3) | 25 (49) |
| GVHD prophylaxis | |||
| CNI + MTx (66.3%) | 80 (60.6) | 39 (65) | 42 (82.4) |
| CNI + MMF (22.2%) | 35 (26.5) | 11 (18.3) | 8 (15.7) |
| PTCy + CNI + MMF (3.3%) | 5 (3.8) | 2 (3.3) | 1 (1.9) |
| CNI + steroid (2.1%) | 1 (0.8) | 4 (6.7) | 0 |
| Other (6.1%) | 11 (8.3) | 4 (6.7) | 0 |
| History of aGVHD (maximal grade) | |||
| None (44%) | 87 (65.9) | 12 (20) | 8 (15.7) |
| Grade 1 (19.4%) | 19 (14.4) | 14 (23.3) | 14 (27.4) |
| Grade 2 (20.2%) | 17 (12.9) | 17 (28.3) | 15 (29.4) |
| Grade 3 (12.3%) | 7 (5.3) | 12 (20) | 11 (21.6) |
| Grade 4 (4.1%) | 2 (1.5) | 5 (8.4) | 3 (5.9) |
| Characteristic (overall percentages of entire evaluable cohort; n = 243) . | No L-aGVHD or cGVHD (n = 132) . | L-aGVHD (n = 60) . | cGVHD (n = 51) . |
|---|---|---|---|
| Diagnoses | |||
| Malignant (n = 168; 69.1%) | 78 (59) | 49 (81.7) | 41 (80.4) |
| ALL, n | 33 | 23 | 18 |
| MDS/AML, n | 37 | 21 | 15 |
| Mixed lineage acute leukemia, n | 0 | 0 | 1 |
| NHL, n | 4 | 3 | 3 |
| JMML, n | 3 | 2 | 1 |
| CML, n | 1 | 0 | 3 |
| Nonmalignant (n = 75; 30.9%) | 54 (41) | 11 (18.3) | 10 (19.6) |
| Sickle cell anemia, n | 12 | 4 | 4 |
| Severe aplastic anemia, n | 12 | 1 | 1 |
| Thalassemia, n | 4 | 1 | 0 |
| Primary immunodeficiency disorder, n* | 12 | 2 | 1 |
| Inherited marrow failure syndrome, n† | 5 | 1 | 2 |
| Other nonmalignant disorder, n‡ | 9 | 2 | 2 |
| Sex | |||
| Male (55.6%) | 72 (54.5) | 30 (50) | 33 (64.7) |
| Female (44.4%) | 60 (45.5) | 30 (50) | 18 (35.3) |
| Age at transplant | |||
| Median (IQR) (range), y | 9.3 (4.6-12.9) (0.2-17.9) | 7.7 (3.1-12.2) (0.9-18) | 11.9 (7.8-14.6) (2-18) |
| Donor and HLA match | |||
| HLA-matched sibling donor (8/8) | 51 (38.6) | 13 (21.7) | 8 (15.7) |
| HLA-matched nonsibling family donor | 3 (2.3) | 3 (5) | 0 (0) |
| Haploidentical family donor (with PTCy) | 2 (1.5) | 1 (1.7) | 1 (2) |
| HLA-matched unrelated donor (8/8) | 48 (36.4) | 21 (35) | 22 (43.1) |
| HLA-mismatched unrelated donor (≤7/8) | 9 (6.8) | 9 (15) | 12 (23.5) |
| HLA-matched cord blood (6/6) | 9 (6.8) | 4 (6.6) | 1 (2) |
| HLA-mismatched cord blood (≤5/6) | 9 (6.8) | 7 (11.7) | 6 (11.7) |
| Double cord blood | 1 (0.8) | 2 (3.3) | 1 (2) |
| Stem cell source | |||
| Bone marrow (62.6%) | 94 (71.2) | 33 (55) | 25 (49.0) |
| PBSC (20.6%) | 19 (14.4) | 13 (21.7) | 18 (35.3) |
| Cord blood (15.2%) | 18 (13.6) | 12 (20) | 7 (13.7) |
| Double cord blood (1.6%) | 1 (0.8) | 2 (3.3) | 1 (2.0) |
| Conditioning regimen | |||
| Myeloablative (86.8%) | 111 (84.1) | 55 (91.7) | 45 (88.2) |
| TBI 1200-1320 cGY/CY ± other, n | 30 | 23 | 19 |
| Bu + CY ± Mel, n | 27 | 17 | 14 |
| Bu + Flu ± Mel, n | 44 | 10 | 11 |
| Bu + Flu + 200-400 cGY TBI, n | 6 | 4 | 1 |
| TBI 1200 cGY + other, n | 3 | 0 | 0 |
| Treosulfan + Flu + 200 cGY TBI, n | 1 | 1 | 0 |
| Reduced intensity (13.2%) | 21 (15.9) | 5 (8.3) | 6 (11.8) |
| Cy/Flu + TBI 200-400 cGY, n | 7 | 2 | 0 |
| Cy ± Flu, n | 4 | 0 | 2 |
| TBI 300 cGY (+ serotherapy), n | 3 | 0 | 0 |
| Flu/Mel (± serotherapy ± TT), n | 3 | 2 | 2 |
| Other, n | 4 | 1 | 2 |
| Serotherapy | |||
| Rabbit ATG (31.3%) | 43 (32.5) | 20 (33.3) | 13 (25.5) |
| Horse ATG (13.1%) | 17 (12.9) | 4 (6.7) | 11 (21.6) |
| Alemtuzumab (10.7%) | 20 (15.2) | 4 (6.7) | 2 (3.9) |
| None (44.9%) | 52 (39.4) | 32 (53.3) | 25 (49) |
| GVHD prophylaxis | |||
| CNI + MTx (66.3%) | 80 (60.6) | 39 (65) | 42 (82.4) |
| CNI + MMF (22.2%) | 35 (26.5) | 11 (18.3) | 8 (15.7) |
| PTCy + CNI + MMF (3.3%) | 5 (3.8) | 2 (3.3) | 1 (1.9) |
| CNI + steroid (2.1%) | 1 (0.8) | 4 (6.7) | 0 |
| Other (6.1%) | 11 (8.3) | 4 (6.7) | 0 |
| History of aGVHD (maximal grade) | |||
| None (44%) | 87 (65.9) | 12 (20) | 8 (15.7) |
| Grade 1 (19.4%) | 19 (14.4) | 14 (23.3) | 14 (27.4) |
| Grade 2 (20.2%) | 17 (12.9) | 17 (28.3) | 15 (29.4) |
| Grade 3 (12.3%) | 7 (5.3) | 12 (20) | 11 (21.6) |
| Grade 4 (4.1%) | 2 (1.5) | 5 (8.4) | 3 (5.9) |
Unless otherwise noted, data are n (%).
ALL, acute lymphoblastic leukemia; AML, acute myeloid leukemia; ATG, anti-thymocyte globulin; Bu, busulfan; CML, chronic myelogenous leukemia; CNI, calcineurin inhibitor; Cy, cyclophosphamide; Flu, fludarabine; IQR, interquartile range; JMML, juvenile myelomonocytic leukemia; Mel, melphalan; MDS, myelodysplastic syndrome; MMF, mycophenolate mofetil; MTx, methotrexate; NHL, non-Hodgkin lymphoma; PBSC, peripheral blood stem cells; PTCy, posttransplant cyclophosphamide; TBI, total body irradiation; TT, thiotepa; ±, with or without.
Primary immunodeficiency disorders included chronic granulomatous disease (n = 6), severe combined immune deficiency (n = 6), leukocyte adhesion defect (n = 1), dedicator of cytokinesis 8 (DOCK8) (n = 1), and GATA2 (n = 1).
Inherited bone marrow failure syndromes included Fanconi anemia (n = 3), Diamond-Blackfan anemia (n = 2), dyskeratosis congenita (n = 1), severe congenital neutropenia (n = 1), and Shwachman-Diamond syndrome (n = 1).
Other nonmalignant disorders included Hurler syndrome (n = 5), hemophagocytic lymphohistiocytosis/X-linked lymphoproliferative syndrome (n = 2), Glanzmann’s thrombasthenia (n = 2), cerebral adrenoleukodystrophy (n = 2), osteopetrosis (n = 1), and chronic active Epstein-Barr virus infection (n = 1).