Table 1.

Intensive immunosuppression (ATG plus cyclosporine) for severe aplastic anemia


Study

N

Median age, y

Response, %

Relapse, %

Clonal evolution, %

Survival, %
German68   84   32   65   19   8   58 at 11 y  
EGMBT69   100   16   77   12   11   87 at 5 y  
NIH70   122   35   61   35   11   55 at 7 y  
Japan*71   119   9   68   22   6   88 at 3 y  
NIH72 
 
104
 
30
 
62
 
37
 
9
 
80 at 4 y
 

Study

N

Median age, y

Response, %

Relapse, %

Clonal evolution, %

Survival, %
German68   84   32   65   19   8   58 at 11 y  
EGMBT69   100   16   77   12   11   87 at 5 y  
NIH70   122   35   61   35   11   55 at 7 y  
Japan*71   119   9   68   22   6   88 at 3 y  
NIH72 
 
104
 
30
 
62
 
37
 
9
 
80 at 4 y
 

Only studies of more than 20 enrolled patients are tabulated. Responses to immunosuppressive therapy are usually partial; blood counts may not become normal but transfusions are no longer required and the neutrophil count is adequate to prevent infection. Relapse is usually responsive to further immunosuppressive therapies. Clonal evolution is to dysplastic bone marrow changes and/or cytogenetic abnormalities. For details, see “Immunosuppression”

*

With androgens and ± G-CSF

With mycophenolate mofetil

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