Table 1

Results of recent clinical trials for pediatric AML

StudyYears of enrollmentEligible age, yNo. of patientsCR rate,* %OutcomeKey points
St Jude AML02 2002-2008 ≤ 21 216 94 3-y EFS: 63% Risk-adapted therapy was based on molecular genetics and MRD 
     3-y OS: 71% High-dose cytarabine during induction was not beneficial 
AML-BFM 9811,26  1998-2003 < 18 473 88 5-y EFS: 49% G-CSF did not decrease the incidence of infection or infection-related mortality 
     5-y OS: 62% Shorter, intensive consolidation cycles did not improve outcome 
MRC AML1212  1995-2002 < 16 529 92 10-y EFS: 54% No differences in EFS or OS rates between mitoxantrone- and daunorubicin-based inductions 
     10-y OS: 64% No difference in EFS or OS rates between 4 and 5 courses of therapy 
NOPHO-AML 200413  2004-2009 ≤ 18 151 92 3-y EFS: 57% Intensity of therapy based on early response to therapy 
     3-y OS: 69%  
AML9910  2000-2002 ≤ 18 240 95 5-y EFS: 62% Intensive use of high-dose cytarabine 
     5-y OS: 76% Six courses of therapy 
COG AAML03P179  2003-2005 ≤ 21 350 87 3-y EFS: 53% Safe to add gemtuzumab ozogamicin to first and fourth courses of therapy 
     3-y OS: 66%  
StudyYears of enrollmentEligible age, yNo. of patientsCR rate,* %OutcomeKey points
St Jude AML02 2002-2008 ≤ 21 216 94 3-y EFS: 63% Risk-adapted therapy was based on molecular genetics and MRD 
     3-y OS: 71% High-dose cytarabine during induction was not beneficial 
AML-BFM 9811,26  1998-2003 < 18 473 88 5-y EFS: 49% G-CSF did not decrease the incidence of infection or infection-related mortality 
     5-y OS: 62% Shorter, intensive consolidation cycles did not improve outcome 
MRC AML1212  1995-2002 < 16 529 92 10-y EFS: 54% No differences in EFS or OS rates between mitoxantrone- and daunorubicin-based inductions 
     10-y OS: 64% No difference in EFS or OS rates between 4 and 5 courses of therapy 
NOPHO-AML 200413  2004-2009 ≤ 18 151 92 3-y EFS: 57% Intensity of therapy based on early response to therapy 
     3-y OS: 69%  
AML9910  2000-2002 ≤ 18 240 95 5-y EFS: 62% Intensive use of high-dose cytarabine 
     5-y OS: 76% Six courses of therapy 
COG AAML03P179  2003-2005 ≤ 21 350 87 3-y EFS: 53% Safe to add gemtuzumab ozogamicin to first and fourth courses of therapy 
     3-y OS: 66%  

CR indicates complete remission.

*

CR rate after 2 courses of induction therapy.

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