Table 1.

Patient baseline disease and transplant characteristics

ED1 n = 26ED2 n = 13ED1 + ED2 n = 39AD n = 16All 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 = 26ED2 n = 13ED1 + ED2 n = 39AD n = 16All 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.