Patient baseline disease and transplant characteristics
. | ED1 n = 26 . | ED2 n = 13 . | ED1 + ED2 n = 39 . | AD n = 16 . | All patients N = 59 . |
---|---|---|---|---|---|
Median (min-max) | |||||
Age at CALD dx, y | 7 (0-14) | 7 (5-11) | 7 (0-14) | 8 (6-12) | 7 (0-14) |
Age at first allo-HSCT, y | 7 (2-14) | 8 (5-11) | 8 (2-14) | 9 (6-13) | 8 (2-14) |
Time from ALD dx to CALD dx, mo | 30.8 (0-125) | 0.2 (0-111.2) | 9.8 (0-125) | 0.03 (0-48.2) | 0.8 (0-125) |
Time from ALD dx to transplant, mo | 45.0 (2.8-152.2) | 4.4 (0.8-113.2) | 25.3 (0.8-152.2) | 3.5 (1.1-99.5) | 9.4 (0.8-152.2) |
Time from CALD dx to transplant, mo | 3.6 (1.3-76.3) | 3.0 (0.6-78) | 3.6 (0.6-78) | 3.4 (1.1-51.4) | 3.5 (0.6-78) |
Length of follow-up, mo | 25.3 (0.9-49.2) | 31.2 (0.9-48.1) | 25.8 (0.9-48.1) | 8.9 (2.1-48.6) | 23.0 (0.9-49.5) |
n (%) | |||||
Baseline NFS 0 1 >1-≤4 >4 Missing | 26 (100) 0 0 0 0 | 12 (92.3) 1 (7.7) 0 0 0 | 38 (97.4) 1 (2.6) 0 0 0 | 5 (31.3) 5 (31.3) 4 (25.0) 1 (6.3) 1 (6.3) | 43 (72,9) 7 (11.9) 4 (6.8) 1 (1.7) 4 (6.8) |
Baseline Loes score 0 0.5-4 >4-9 >9 Missing | 4 (15.4) 22 (84.6) 0 0 0 | 0 0 13 (100) 0 0 | 4 (10.3) 22 (56.4) 13 (33.3) 0 0 | 0 0 1 (6.3) 13 (81.3) 2 (12.5) | 6 (10.2) 22 (37.3) 15 (25.4) 13 (22.0) 7 (11.9) |
Baseline GdE status + – Missing | 16 (61.5) 8 (30.8) 2 (7.7) | 11 (84.6) 2 (15.4) 0 | 27 (69.2) 10 (25.6) 2 (5.1) | 12 (75.0) 2 (12.5) 2 (12.5) | 39 (66.1) 13 (22.0) 7 (11.9) |
Conditioning regimen Bu/Cy Bu/Flu Missing | 10 (38.5) 15 (57.7) 1 (3.8) | 6 (46.2) 7 (53.8) 0 | 16 (41.0) 22 (56.4) 1 (2.6) | 6 (37.5) 9 (56.3) 1 (6.3) | 25 (42.4) 32 (54.2) 2 (3.4) |
Donor source MRD (BM+PB+UCB) UR (BM+PB) UR (UCB) Haploidentical | 7 (26.9) 9 (4.6) 10 (38.5) 0 (0) | 4 (30.8) 5 (38.5) 4 (30.8) 0 (0) | 11 (28.2) 14 (35.9) 14 (35.9) 0 (0) | 1 (6.3) 4 (25.0) 6 (37.5) 5 (31.3) | 12 (20.3) 19 (32.2) 23 (39.0) 0 (0) |
Donor match Matched Haplo+mismatched | 20 (76.9) 6 (23.1) | 9 (69.2) 4 (30.8) | 29 (74.4) 10 (25.6) | 6 (37.5) 10 (62.5) | 36 (61.0) 23 (39.0) |
HLA match type Matched 10/10 8/8 6/6 Mismatched 9/10 5/6 4/6 Haploidentical | 8 (30.8) 6 (23.1) 6 (23.1) 1 (3.8) 3 (11.5) 2 (7.7) 0 | 4 (30.8) 4 (30.8) 1 (7.7) 1 (7.7) 2 (15.4) 1 (7.7) 0 | 12 (30.8) 10 (25.6) 7 (17.9) 2 (5.1) 5 (12.8) 3 (7.7) 0 | 3 (18.8) 1 (6.3) 2 (12.5) 1 (6.3) 1 (6.3) 3 (18.8) 5 (31.3) | 16 (27.1) 11 (18.6) 9 (15.3) 3 (5.1) 7 (11.9) 8 (13.6) 5 (8.5) |
. | ED1 n = 26 . | ED2 n = 13 . | ED1 + ED2 n = 39 . | AD n = 16 . | All patients N = 59 . |
---|---|---|---|---|---|
Median (min-max) | |||||
Age at CALD dx, y | 7 (0-14) | 7 (5-11) | 7 (0-14) | 8 (6-12) | 7 (0-14) |
Age at first allo-HSCT, y | 7 (2-14) | 8 (5-11) | 8 (2-14) | 9 (6-13) | 8 (2-14) |
Time from ALD dx to CALD dx, mo | 30.8 (0-125) | 0.2 (0-111.2) | 9.8 (0-125) | 0.03 (0-48.2) | 0.8 (0-125) |
Time from ALD dx to transplant, mo | 45.0 (2.8-152.2) | 4.4 (0.8-113.2) | 25.3 (0.8-152.2) | 3.5 (1.1-99.5) | 9.4 (0.8-152.2) |
Time from CALD dx to transplant, mo | 3.6 (1.3-76.3) | 3.0 (0.6-78) | 3.6 (0.6-78) | 3.4 (1.1-51.4) | 3.5 (0.6-78) |
Length of follow-up, mo | 25.3 (0.9-49.2) | 31.2 (0.9-48.1) | 25.8 (0.9-48.1) | 8.9 (2.1-48.6) | 23.0 (0.9-49.5) |
n (%) | |||||
Baseline NFS 0 1 >1-≤4 >4 Missing | 26 (100) 0 0 0 0 | 12 (92.3) 1 (7.7) 0 0 0 | 38 (97.4) 1 (2.6) 0 0 0 | 5 (31.3) 5 (31.3) 4 (25.0) 1 (6.3) 1 (6.3) | 43 (72,9) 7 (11.9) 4 (6.8) 1 (1.7) 4 (6.8) |
Baseline Loes score 0 0.5-4 >4-9 >9 Missing | 4 (15.4) 22 (84.6) 0 0 0 | 0 0 13 (100) 0 0 | 4 (10.3) 22 (56.4) 13 (33.3) 0 0 | 0 0 1 (6.3) 13 (81.3) 2 (12.5) | 6 (10.2) 22 (37.3) 15 (25.4) 13 (22.0) 7 (11.9) |
Baseline GdE status + – Missing | 16 (61.5) 8 (30.8) 2 (7.7) | 11 (84.6) 2 (15.4) 0 | 27 (69.2) 10 (25.6) 2 (5.1) | 12 (75.0) 2 (12.5) 2 (12.5) | 39 (66.1) 13 (22.0) 7 (11.9) |
Conditioning regimen Bu/Cy Bu/Flu Missing | 10 (38.5) 15 (57.7) 1 (3.8) | 6 (46.2) 7 (53.8) 0 | 16 (41.0) 22 (56.4) 1 (2.6) | 6 (37.5) 9 (56.3) 1 (6.3) | 25 (42.4) 32 (54.2) 2 (3.4) |
Donor source MRD (BM+PB+UCB) UR (BM+PB) UR (UCB) Haploidentical | 7 (26.9) 9 (4.6) 10 (38.5) 0 (0) | 4 (30.8) 5 (38.5) 4 (30.8) 0 (0) | 11 (28.2) 14 (35.9) 14 (35.9) 0 (0) | 1 (6.3) 4 (25.0) 6 (37.5) 5 (31.3) | 12 (20.3) 19 (32.2) 23 (39.0) 0 (0) |
Donor match Matched Haplo+mismatched | 20 (76.9) 6 (23.1) | 9 (69.2) 4 (30.8) | 29 (74.4) 10 (25.6) | 6 (37.5) 10 (62.5) | 36 (61.0) 23 (39.0) |
HLA match type Matched 10/10 8/8 6/6 Mismatched 9/10 5/6 4/6 Haploidentical | 8 (30.8) 6 (23.1) 6 (23.1) 1 (3.8) 3 (11.5) 2 (7.7) 0 | 4 (30.8) 4 (30.8) 1 (7.7) 1 (7.7) 2 (15.4) 1 (7.7) 0 | 12 (30.8) 10 (25.6) 7 (17.9) 2 (5.1) 5 (12.8) 3 (7.7) 0 | 3 (18.8) 1 (6.3) 2 (12.5) 1 (6.3) 1 (6.3) 3 (18.8) 5 (31.3) | 16 (27.1) 11 (18.6) 9 (15.3) 3 (5.1) 7 (11.9) 8 (13.6) 5 (8.5) |
dx, diagnosis; UR, unrelated donor.