Supplementary Information
Table 1. Univariate study of risk factors for EFS
Patients
Events
Patient age
≤ 8.5 years / > 8.5 years
0.992
41 / 40
15 / 15
Patient gender
Male / Female
0.674
51 / 30
20 / 10
Risk status1
Standard / High
0.016
51 / 30
14 / 16
HLA match (8/8)2
Match / Mismatch
0.339
57 / 24
19 / 11
Conditioning regimen
TBI based / Non-TBI based
0.191
27 / 54
13 / 17
GVHD prophylaxis3
CS + MTX (4) / CS + MTX (0-3)
0.739
53 / 28
19 / 11
Donor-recipient gender
Female to Male / Others
0.029
13 / 68
9 / 21
CD34+ dose
0.008
Lower (≤ 10.0 x 106/kg)
40
21
Higher (> 10.0 x 106/kg)
41
9
CD3+ dose
1
P value
0.458
Lower (≤ 60.0 x 107/kg)
37
16
Higher (> 60.0 x 107/kg)
44
14
Standard risk patients included acute leukemia in first CR, chronic myeloid leukemia in first
chronic phase, and refractory anemia.
High risk patients included acute leukemia in second CR
or greater, or without complete remission, myelodysplastic syndrome with excess of blasts,
juvenile myelomonocytic leukemia, and secondary leukemia.
2
3
HLA match at HLA-A, B, C, DRB1 alleles
Cyclosporine and all 4 doses of mini-methotrexate vs. cyclosporine and 3 or fewer doses
Abbreviations: TBI, total body irradiation; CS, cyclosporine; MTX, methotrexate.
Table 2. Univariate study of risk factors for overall chronic GVHD
Patients
Events
Patient age
≤ 8.5 years / > 8.5 years
0.449
41 / 40
13 / 16
Patient gender
Male / Female
0.196
51 / 30
16 / 13
Risk status1
Standard / High
0.178
51 / 30
21 / 8
HLA match (8/8)2
Match / Mismatch
0.454
57 / 24
19 / 10
Conditioning regimen
TBI based / Non-TBI based
0.064
27 / 54
6 / 23
GVHD prophylaxis3
CS + MTX (4) / CS + MTX (0-3)
0.669
53 / 28
18 / 11
Donor-recipient gender
Female to Male / Others
0.554
13 / 68
6 / 23
Previous acute GVHD
No / Yes
0.232
39 / 42
11 / 18
CD34+ dose
0.519
Lower (≤ 10.0 x 106/kg)
40
13
Higher (> 10.0 x 106/kg)
41
16
CD3+ dose
1
P value
0.255
Lower (≤ 60.0 x 107/kg)
37
11
Higher (> 60.0 x 107/kg)
44
18
Standard risk patients included acute leukemia in first CR, chronic myeloid leukemia in first
chronic phase, and refractory anemia.
High risk patients included acute leukemia in second CR
or greater, or without complete remission, myelodysplastic syndrome with excess of blasts,
juvenile myelomonocytic leukemia, and secondary leukemia.
2
3
HLA match at HLA-A, B, C, DRB1 alleles
Cyclosporine and all 4 doses of mini-methotrexate vs. cyclosporine and 3 or fewer doses
Abbreviations: TBI, total body irradiation; CS, cyclosporine; MTX, methotrexate
Table 3. Univariate study of risk factors for extensive chronic GVHD
Patients
Events
Patient age
≤ 8.5 years / > 8.5 years
0.332
41 / 40
8 / 12
Patient gender
Male / Female
0.122
51 / 30
10 / 10
Risk status1
Standard / High
0.205
51 / 30
15 / 5
HLA match (8/8)2
Match / Mismatch
0.239
57 / 24
12 / 8
Conditioning regimen
TBI based / Non-TBI based
0.130
27 / 54
4 / 16
GVHD prophylaxis3
CS + MTX (4) / CS + MTX (0-3)
0.947
53 / 28
13 / 7
Donor-recipient gender
Female to Male / Others
0.755
13 / 68
4 / 16
Previous acute GVHD
No / Yes
0.803
39 / 42
9 / 11
CD34+ dose
0.356
Lower (≤ 10.0 x 106/kg)
40
12
Higher (> 10.0 x 106/kg)
41
8
CD3+ dose
1
P value
0.860
Lower (≤ 60.0 x 107/kg)
37
9
Higher (> 60.0 x 107/kg)
44
11
Standard risk patients included acute leukemia in first CR, chronic myeloid leukemia in first
chronic phase, and refractory anemia.
High risk patients included acute leukemia in second CR
or greater, or without complete remission, myelodysplastic syndrome with excess of blasts,
juvenile myelomonocytic leukemia, and secondary leukemia.
2
3
HLA match at HLA-A, B, C, DRB1 alleles
Cyclosporine and all 4 doses of mini-methotrexate vs. cyclosporine and 3 or fewer doses
Abbreviations: TBI, total body irradiation; CS, cyclosporine; MTX, methotrexate
Table 4. Rates of acute and chronic GVHD for the younger subcohort, analyzed according to the median CD34+ and CD3+ cell dose for the subcohort.
Patient
Median
age
≤ 8.5
CD34+
years
dose/kg
CD3+
dose/kg
N
Acute GVHD II-IV
Overall Chronic GVHD
Extensive Chronic GVHD
Rate %
P value
Rate %
P value
Rate %
P value
0.818
26.3±10.5
0.461
21.1±9.7
0.896
≤ 15 x 106
19
52.6±11.9
> 15 x 106
22
45.5±11.0
≤ 110 x 107
19
47.4±11.9
> 110 x 107
22
50.0±11.0
36.4±10.6
0.665
31.6±11.1
31.8±10.3
18.2±8.4
0.937
21.1±9.7
18.2±8.5
0.880