Table 3.

Univariate analysis of 5-year event-free survival probability (EFS), cumulative incidence of relapse (RI), and cumulative incidence of transplantation-related mortality (TRM)

EFS
RI
TRM
No. of patients
Probability
95% CI
Cumulative incidence
95% CI
Cumulative incidence
95% CI
Overall probability or incidence   100   52%   42-62   35%   27-46   13   8-22  
Patient sex        
Male   67   61%   49-73   30%   20-44   9%   4-19  
Female   33   33%   17-49   45%   31-66   21%   11-41  
p  —   .0021   —   .012   —   .065   —  
Donor sex        
Male   50   53%   38-67   41%   30-59   6%   2-18  
Female   46   53%   39-68   25%   15-42   22%   13-38  
Missing   4   —   —   —   —   —   —  
p  —   NS   —   NS   —   .035   —  
Age at diagnosis        
Less than 1 y   38   65%   46-78   17%   8-35   21%   12-40  
1-2 y   18   67%   45-88   22%   9-53   11%   3-41  
2-3 y   9   47%   10-84   53%   27-100   0%   —  
3-4 y   18   50%   27-73   44%   27-75   6%   1-37  
4 y or more   17   16%   0-34   73%   53-98   12%   3-43  
p  —   .013   —   .0003   —   NS   —  
Less than 2 y   56   64%   51-76   18%   10-32   18%   10-32  
2-4 y   27   50%   31-70   46%   30-70   4%   1-25  
4 y   17   16%   0-34   73%   53-98   12%   3-43  
p  —   .0020   —   .0001   —   NS   —  
Interval between diagnosis and HSCT        
Less than 3 mo   24   56%   35-77   31%   17-59   13%   4-36  
3-6 mo   29   54%   35-73   39%   24-62   7%   2-28  
6-9 mo   25   47%   27-67   37%   22-62   16%   7-40  
9 mo or more   22   50%   29-71   32%   17-59   18%   7-44  
p  —   NS   —   NS   —   NS   —  
Age at HSCT        
Less than 1 y   17   64%   41-87   12%   3-45   24%   10-55  
1-2 y   27   66%   48-84   19%   9-42   15%   6-37  
2-3 y   13   62%   35-88   23%   9-62   15%   4-55  
3-4 y   18   55%   32-78   45%   27-75   0%   —  
4 y or more   25   23%   6-40   65%   48-87   12%   4-35  
p  —   .014   —   .0008   —   NS   —  
Less than 2 y   44   65%   51-79   16%   8-32   18%   10-34  
2-4 y   31   57%   39-75   37%   23-59   6%   2-25  
4 y or more   25   23%   6-40   65%   48-87   12%   4-35  
p  —   .0022   —   .0008   —   NS   —  
Donor age        
Less than 10 y   31   60%   42-78   34%   20-56   6%   2-25  
10-20 y   9   33%   3-64   33%   13-84   33%   13-84  
20 y or more   39   47%   31-63   37%   25-57   16%   8-33  
Missing   21   —   —   —   —   —   —  
p  —   NS   —   NS   —   NS   —  
Leukocyte count at diagnosis, × 109/L        
Less than 20   25   51%   31-71   45%   29-70   4%   1-27  
20-39   35   49%   32-67   30%   18-51   20%   10-39  
40-59   17   40%   17-64   36%   19-69   24%   10-55  
60-79   11   64%   35-92   36%   17-79   0%   —  
80 or more   9   63%   30-97   37%   15-91   0%   —  
Missing   3   —   —   —   —   —   —  
p  —   NS   —   NS   —   .09   —  
Monocyte count at diagnosis, × 109/L        
1-2   13   54%   27-80   31%   14-70   15%   4-55  
2-3   15   51%   25-78   42%   23-78   7%   1-44  
3-5   16   56%   32-81   31%   15-65   13%   3-46  
5-10   25   38%   19-58   37%   22-62   25%   12-50  
10 or more   25   62%   43-82   34%   19-59   4%   1-27  
Missing   6   —   —   —   —   —   —  
p  —   NS   —   NS   —   NS   —  
Platelet count at diagnosis, × 109/L        
Less than 50   38   44%   28-60   42%   29-62   13%   6-30  
50-100   27   48%   28-69   37%   22-63   15%   6-37  
100 or more   32   62%   45-79   29%   17-50   9%   3-28  
Missing   3   —   —   —   —   —   —  
p  —   NS   —   NS   —   NS   —  
Karyotype        
Normal   66   49%   36-61   37%   27-52   14%   8-25  
Monosomy 7   20   68%   46-89   22%   9-54   10%   3-37  
Other   13   46%   19-73   46%   26-83   8%   1-51  
Missing   1   —   —   —   —   —   —  
p  —   NS   —   NS   —   NS   —  
HbF %        
Less than 5%   26   61%   42-80   20%   9-43   20%   9-43  
5%-9%   14   43%   17-69   57%   36-90   0%   —  
10%-39%   23   51%   29-72   32%   17-59   17%   7-42  
40% or more   14   19%   0-41   81%   62-100   0%   —  
Missing   23   —   —   —   —   —   —  
p  —   NS   —   .0083   —   NS   —  
Less than 40%   63   53%   41-66   33%   23-47   14   8-26  
40% or more   14   19%   0-41   81%   62-100   0   —  
Missing   23   —   —   —   —   —   —  
p  —   .07   —   .004   —   NS   —  
Clinical evidence of NF1        
No   78   55%   43-66   34%   24-47   12%   6-22  
Yes   14   36%   11-61   50%   30-84   14%   4-52  
Missing   8   —   —   —   —   —   —  
p  —   NS   —   NS   —   NS   —  
Leukocyte count at HSCT, × 109/L        
Less than 10   46   53%   38-68   32%   20-49   15%   8-30  
10-19   20   47%   25-70   37%   20-67   16%   6-45  
20-39   17   45%   20-69   43%   25-77   12%   3-43  
40 or more   9   44%   12-77   56%   31-100   0%   —  
Missing   8   —   —   —   —   —   —  
p  —   NS   —   NS   —   —   —  
Bone marrow blast percentage at HSCT        
Less than 5%   30   63%   46-81   20%   10-41   17%   7-37  
5%-19%   47   52%   37-67   35%   24-52   13%   6-27  
20% or more   10   0%   —   90%   73-100   10%   2-64  
Missing   13   —   —   —   —   —   —  
p  —   .10   —   .017   —   NS   —  
Spleen size at HSCT        
Less than 5 cm   34   61%   44-78   24%   13-44   15%   7-33  
5 cm or more   36   44%   26-62   45%   30-67   11%   4-28  
Splenectomized   24   48%   28-69   39%   23-65   13%   4-36  
Missing   6   —   —   —   —   —   —  
p  —   NS   —   NS   —   NS   —  
Pre-HSCT treatment        
None or low dose   84   52%   41-63   35%   26-47   13%   8-23  
AML-like   16   50%   26-75   38%   20-71   13%   3-46  
p  —   NS   —   NS   —   NS   —  
Donor        
Matched family donor   48   55%   41-70   35%   23-52   10%   5-24  
Unrelated donor   52   49%   35-63   36%   24-52   16%   8-30  
p  —   NS   —   NS   —   NS   —  
Stem cell source        
Bone marrow   79   51%   40-62   35%   26-47   14%   8-24  
Peripheral blood   14   55%   28-82   36%   18-72   9%   1-59  
Cord blood   7   54%   14-93   32%   10-100   14%   2-88  
p  —   NS   —   NS   —   NS   —  
Stem cell dose, BM only        
Less than 5 × 108/kg   22   58%   37-79   33%   18-61   9%   2-34  
5 × 108/kg or more   46   54%   39-68   31%   20-48   15%   8-30  
Missing   11   —   —   —   —   —   —  
p  —   NS   —   NS   —   NS   —  
HCMV serology        
Negative/Negative   41   49%   33-65   39%   26-58   13%   6-29  
Other   53   54%   40-67   33%   22-49   13%   7-26  
Missing   6   —   —   —   —   —   —  
p  —   NS   —   NS   —   NS   —  
Busulfan PK study        
Yes   25   60%   40-79   32%   18-57   8%   2-30  
No   75   49%   37-61   36%   27-59   15%   9-269  
p  —   NS   —   NS   —   NS   —  
GVHD prophylaxis        
All transplantations         
Monotherapy   35   59%   42-75   33%   20-54   9%   3-25  
Combination treatment   14   36%   11-61   36%   18-72   29%   12-65  
Serotherapy   51   52%   37-66   36%   25-53   12%   6-25  
p  —   NS   —   NS   —   NS   —  
Sibling donor transplantations         
Monotherapy   35   59%   42-75   33%   20-54   9%   3-25  
Combination treatment   8   38%   4-71   38%   11-92   25%   8-83  
Serotherapy   5   60%   17-100   40%   14-100   0%   —  
p  —   NS   —   NS   —   —   —  
Unrelated donor transplantations         
Monotherapy   0   —   —   —   —   —   —  
Combination treatment   6   33%   0-71   33%   11-100   33%   11-100  
Serotherapy   46   51%   36-66   36%   24-53   13%   6-28  
p  —   NS   —   NS   —   0.08   —  
Acute GVHD occurrence        
Grades 0-I   60   48%   35-61   37%   26-52   15%   8-28  
Grades II-IV   40   57%   42-73   33%   21-51   10%   4-25  
p  —   NS   —   NS   —   .083   —  
Chronic GVHD        
Absent   73   59%   48-71   35%   26-48   6%   2-15  
Present   13   67%   41-94   25%   9-67   8%   1-51  
Not evaluable   14   —   —   —   —   —   —  
p
 		 —
 		 NS
 		 —
 		 NS
 		 —
 		 NS
 		 —
 
EFS
RI
TRM
No. of patients
Probability
95% CI
Cumulative incidence
95% CI
Cumulative incidence
95% CI
Overall probability or incidence   100   52%   42-62   35%   27-46   13   8-22  
Patient sex        
Male   67   61%   49-73   30%   20-44   9%   4-19  
Female   33   33%   17-49   45%   31-66   21%   11-41  
p  —   .0021   —   .012   —   .065   —  
Donor sex        
Male   50   53%   38-67   41%   30-59   6%   2-18  
Female   46   53%   39-68   25%   15-42   22%   13-38  
Missing   4   —   —   —   —   —   —  
p  —   NS   —   NS   —   .035   —  
Age at diagnosis        
Less than 1 y   38   65%   46-78   17%   8-35   21%   12-40  
1-2 y   18   67%   45-88   22%   9-53   11%   3-41  
2-3 y   9   47%   10-84   53%   27-100   0%   —  
3-4 y   18   50%   27-73   44%   27-75   6%   1-37  
4 y or more   17   16%   0-34   73%   53-98   12%   3-43  
p  —   .013   —   .0003   —   NS   —  
Less than 2 y   56   64%   51-76   18%   10-32   18%   10-32  
2-4 y   27   50%   31-70   46%   30-70   4%   1-25  
4 y   17   16%   0-34   73%   53-98   12%   3-43  
p  —   .0020   —   .0001   —   NS   —  
Interval between diagnosis and HSCT        
Less than 3 mo   24   56%   35-77   31%   17-59   13%   4-36  
3-6 mo   29   54%   35-73   39%   24-62   7%   2-28  
6-9 mo   25   47%   27-67   37%   22-62   16%   7-40  
9 mo or more   22   50%   29-71   32%   17-59   18%   7-44  
p  —   NS   —   NS   —   NS   —  
Age at HSCT        
Less than 1 y   17   64%   41-87   12%   3-45   24%   10-55  
1-2 y   27   66%   48-84   19%   9-42   15%   6-37  
2-3 y   13   62%   35-88   23%   9-62   15%   4-55  
3-4 y   18   55%   32-78   45%   27-75   0%   —  
4 y or more   25   23%   6-40   65%   48-87   12%   4-35  
p  —   .014   —   .0008   —   NS   —  
Less than 2 y   44   65%   51-79   16%   8-32   18%   10-34  
2-4 y   31   57%   39-75   37%   23-59   6%   2-25  
4 y or more   25   23%   6-40   65%   48-87   12%   4-35  
p  —   .0022   —   .0008   —   NS   —  
Donor age        
Less than 10 y   31   60%   42-78   34%   20-56   6%   2-25  
10-20 y   9   33%   3-64   33%   13-84   33%   13-84  
20 y or more   39   47%   31-63   37%   25-57   16%   8-33  
Missing   21   —   —   —   —   —   —  
p  —   NS   —   NS   —   NS   —  
Leukocyte count at diagnosis, × 109/L        
Less than 20   25   51%   31-71   45%   29-70   4%   1-27  
20-39   35   49%   32-67   30%   18-51   20%   10-39  
40-59   17   40%   17-64   36%   19-69   24%   10-55  
60-79   11   64%   35-92   36%   17-79   0%   —  
80 or more   9   63%   30-97   37%   15-91   0%   —  
Missing   3   —   —   —   —   —   —  
p  —   NS   —   NS   —   .09   —  
Monocyte count at diagnosis, × 109/L        
1-2   13   54%   27-80   31%   14-70   15%   4-55  
2-3   15   51%   25-78   42%   23-78   7%   1-44  
3-5   16   56%   32-81   31%   15-65   13%   3-46  
5-10   25   38%   19-58   37%   22-62   25%   12-50  
10 or more   25   62%   43-82   34%   19-59   4%   1-27  
Missing   6   —   —   —   —   —   —  
p  —   NS   —   NS   —   NS   —  
Platelet count at diagnosis, × 109/L        
Less than 50   38   44%   28-60   42%   29-62   13%   6-30  
50-100   27   48%   28-69   37%   22-63   15%   6-37  
100 or more   32   62%   45-79   29%   17-50   9%   3-28  
Missing   3   —   —   —   —   —   —  
p  —   NS   —   NS   —   NS   —  
Karyotype        
Normal   66   49%   36-61   37%   27-52   14%   8-25  
Monosomy 7   20   68%   46-89   22%   9-54   10%   3-37  
Other   13   46%   19-73   46%   26-83   8%   1-51  
Missing   1   —   —   —   —   —   —  
p  —   NS   —   NS   —   NS   —  
HbF %        
Less than 5%   26   61%   42-80   20%   9-43   20%   9-43  
5%-9%   14   43%   17-69   57%   36-90   0%   —  
10%-39%   23   51%   29-72   32%   17-59   17%   7-42  
40% or more   14   19%   0-41   81%   62-100   0%   —  
Missing   23   —   —   —   —   —   —  
p  —   NS   —   .0083   —   NS   —  
Less than 40%   63   53%   41-66   33%   23-47   14   8-26  
40% or more   14   19%   0-41   81%   62-100   0   —  
Missing   23   —   —   —   —   —   —  
p  —   .07   —   .004   —   NS   —  
Clinical evidence of NF1        
No   78   55%   43-66   34%   24-47   12%   6-22  
Yes   14   36%   11-61   50%   30-84   14%   4-52  
Missing   8   —   —   —   —   —   —  
p  —   NS   —   NS   —   NS   —  
Leukocyte count at HSCT, × 109/L        
Less than 10   46   53%   38-68   32%   20-49   15%   8-30  
10-19   20   47%   25-70   37%   20-67   16%   6-45  
20-39   17   45%   20-69   43%   25-77   12%   3-43  
40 or more   9   44%   12-77   56%   31-100   0%   —  
Missing   8   —   —   —   —   —   —  
p  —   NS   —   NS   —   —   —  
Bone marrow blast percentage at HSCT        
Less than 5%   30   63%   46-81   20%   10-41   17%   7-37  
5%-19%   47   52%   37-67   35%   24-52   13%   6-27  
20% or more   10   0%   —   90%   73-100   10%   2-64  
Missing   13   —   —   —   —   —   —  
p  —   .10   —   .017   —   NS   —  
Spleen size at HSCT        
Less than 5 cm   34   61%   44-78   24%   13-44   15%   7-33  
5 cm or more   36   44%   26-62   45%   30-67   11%   4-28  
Splenectomized   24   48%   28-69   39%   23-65   13%   4-36  
Missing   6   —   —   —   —   —   —  
p  —   NS   —   NS   —   NS   —  
Pre-HSCT treatment        
None or low dose   84   52%   41-63   35%   26-47   13%   8-23  
AML-like   16   50%   26-75   38%   20-71   13%   3-46  
p  —   NS   —   NS   —   NS   —  
Donor        
Matched family donor   48   55%   41-70   35%   23-52   10%   5-24  
Unrelated donor   52   49%   35-63   36%   24-52   16%   8-30  
p  —   NS   —   NS   —   NS   —  
Stem cell source        
Bone marrow   79   51%   40-62   35%   26-47   14%   8-24  
Peripheral blood   14   55%   28-82   36%   18-72   9%   1-59  
Cord blood   7   54%   14-93   32%   10-100   14%   2-88  
p  —   NS   —   NS   —   NS   —  
Stem cell dose, BM only        
Less than 5 × 108/kg   22   58%   37-79   33%   18-61   9%   2-34  
5 × 108/kg or more   46   54%   39-68   31%   20-48   15%   8-30  
Missing   11   —   —   —   —   —   —  
p  —   NS   —   NS   —   NS   —  
HCMV serology        
Negative/Negative   41   49%   33-65   39%   26-58   13%   6-29  
Other   53   54%   40-67   33%   22-49   13%   7-26  
Missing   6   —   —   —   —   —   —  
p  —   NS   —   NS   —   NS   —  
Busulfan PK study        
Yes   25   60%   40-79   32%   18-57   8%   2-30  
No   75   49%   37-61   36%   27-59   15%   9-269  
p  —   NS   —   NS   —   NS   —  
GVHD prophylaxis        
All transplantations         
Monotherapy   35   59%   42-75   33%   20-54   9%   3-25  
Combination treatment   14   36%   11-61   36%   18-72   29%   12-65  
Serotherapy   51   52%   37-66   36%   25-53   12%   6-25  
p  —   NS   —   NS   —   NS   —  
Sibling donor transplantations         
Monotherapy   35   59%   42-75   33%   20-54   9%   3-25  
Combination treatment   8   38%   4-71   38%   11-92   25%   8-83  
Serotherapy   5   60%   17-100   40%   14-100   0%   —  
p  —   NS   —   NS   —   —   —  
Unrelated donor transplantations         
Monotherapy   0   —   —   —   —   —   —  
Combination treatment   6   33%   0-71   33%   11-100   33%   11-100  
Serotherapy   46   51%   36-66   36%   24-53   13%   6-28  
p  —   NS   —   NS   —   0.08   —  
Acute GVHD occurrence        
Grades 0-I   60   48%   35-61   37%   26-52   15%   8-28  
Grades II-IV   40   57%   42-73   33%   21-51   10%   4-25  
p  —   NS   —   NS   —   .083   —  
Chronic GVHD        
Absent   73   59%   48-71   35%   26-48   6%   2-15  
Present   13   67%   41-94   25%   9-67   8%   1-51  
Not evaluable   14   —   —   —   —   —   —  
p
 		 —
 		 NS
 		 —
 		 NS
 		 —
 		 NS
 		 —
 

GVHD prophylaxis was defined as follows: monotherapy, a single drug used (cyclosporin-A or methotrexate; the single patient who did not receive any GVHD prophylaxis was included in this group); combination treatment: more than one drug used (cyclosporin-A + methotrexate or steroids) without the addition of antilymphocyte globulin or monoclonal antibodies; serotherapy: any drug combination plus the addition of antilymphocyte globulin or monoclonal antibodies (Campath-1G).

—indicates that no value is available.

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