Table 1

Clinical and genomic characteristics of pediatric AML patients with or without CSF3R mutations

No mutation (N = 768)CSF3R mutation (N = 19)No mutation vs CSF3R mutation
CharacteristicsN(%)N(%)P
Gender      
 Male 389 51 15 79 .015 
 Female 379 49 21 — 
Age (y)      
 Median (range) 10.08 (0.02-29.84) 10.87 (2.38-22.77) .133 
 0-2 y 183 24 .059 
 3-10 y 229 30 47 .100 
 +11 y 356 46 47 .930 
FAB      
 M0 19 1.000 
 M1 84 13 13 1.000 
 M2 164 25 47 .153 
 M4 166 25 27 1.000 
 M5 150 22 .034 
 M6 12 1.000 
 M7 33 1.000 
 Other 39 13 .260 
 Unknown 101 — — — 
Cytogenetics      
 Normal 176 24 16 .588 
 t(8;21) 108 15 26 .174 
 inv(16) 102 14 .495 
 t(9;11)/11q23 153 21 .034 
 t(6;9) 12 1.000 
 Monosomy 7 12 11 .042 
 Del(7q) 1.000 
 −5/5q- 1.000 
 +8 41 .618 
 Other 117 16 42 .005 
 Missing 34 — — — 
FLT3/ITD-positive 118 16 26 .209 
FLT3/ITD point mutation 52 .632 
CEBPA mutation 44 47 < .001 
NPM mutation 63 .392 
Risk group      
 Standard 353 47 11 .001 
 Low 293 39 12 63 .055 
 High 98 13 26 .161 
CR1 response      
 CR 577 76 14 74 .789 
 Not in CR 170 22 26 .780 
 Death 12 1.000 
 Unevaluable — — — 
MRD1      
 No 443 71 10 71 1.000 
 Yes 177 29 29 — 
CR2 response      
 CR 665 88 16 89 1.000 
 Not in CR 74 10 11 .695 
 Death 15 1.000 
Unevaluable 13 — — — 
5-y OS from study entry 44 67 ± 15 89 ± 21 .146 
5-y EFS from study entry 768 49 ± 4 19 44 ± 23 .900 
5yr DFS from end of course 1 (CR patients only) 577 55 ± 4 14 36 ± 26 .250 
5-y RR from end of course 1 (CR patients only) 577 40 ± 4 14 64 ± 27 .111 
WBC × 103 µL: median (range) 29.8 (0.2-610) 39 (0.9-439.2) .359 
BM blasts %: median (range) 70.0 (0-100) 50.5 (23-99) .040 
Platelet count (1000/µL): median (range) 48.0 (1-1177) 34 (8-405) .240 
Hemoglobin: median (range) 8.1 (1.8-17.0) 8.9 (4.2-12.3) .347 
No mutation (N = 768)CSF3R mutation (N = 19)No mutation vs CSF3R mutation
CharacteristicsN(%)N(%)P
Gender      
 Male 389 51 15 79 .015 
 Female 379 49 21 — 
Age (y)      
 Median (range) 10.08 (0.02-29.84) 10.87 (2.38-22.77) .133 
 0-2 y 183 24 .059 
 3-10 y 229 30 47 .100 
 +11 y 356 46 47 .930 
FAB      
 M0 19 1.000 
 M1 84 13 13 1.000 
 M2 164 25 47 .153 
 M4 166 25 27 1.000 
 M5 150 22 .034 
 M6 12 1.000 
 M7 33 1.000 
 Other 39 13 .260 
 Unknown 101 — — — 
Cytogenetics      
 Normal 176 24 16 .588 
 t(8;21) 108 15 26 .174 
 inv(16) 102 14 .495 
 t(9;11)/11q23 153 21 .034 
 t(6;9) 12 1.000 
 Monosomy 7 12 11 .042 
 Del(7q) 1.000 
 −5/5q- 1.000 
 +8 41 .618 
 Other 117 16 42 .005 
 Missing 34 — — — 
FLT3/ITD-positive 118 16 26 .209 
FLT3/ITD point mutation 52 .632 
CEBPA mutation 44 47 < .001 
NPM mutation 63 .392 
Risk group      
 Standard 353 47 11 .001 
 Low 293 39 12 63 .055 
 High 98 13 26 .161 
CR1 response      
 CR 577 76 14 74 .789 
 Not in CR 170 22 26 .780 
 Death 12 1.000 
 Unevaluable — — — 
MRD1      
 No 443 71 10 71 1.000 
 Yes 177 29 29 — 
CR2 response      
 CR 665 88 16 89 1.000 
 Not in CR 74 10 11 .695 
 Death 15 1.000 
Unevaluable 13 — — — 
5-y OS from study entry 44 67 ± 15 89 ± 21 .146 
5-y EFS from study entry 768 49 ± 4 19 44 ± 23 .900 
5yr DFS from end of course 1 (CR patients only) 577 55 ± 4 14 36 ± 26 .250 
5-y RR from end of course 1 (CR patients only) 577 40 ± 4 14 64 ± 27 .111 
WBC × 103 µL: median (range) 29.8 (0.2-610) 39 (0.9-439.2) .359 
BM blasts %: median (range) 70.0 (0-100) 50.5 (23-99) .040 
Platelet count (1000/µL): median (range) 48.0 (1-1177) 34 (8-405) .240 
Hemoglobin: median (range) 8.1 (1.8-17.0) 8.9 (4.2-12.3) .347 

The data from AAML0531, AAML03P1, and CCG-2961 were acquired from the TARGET project, current as of June 30, 2015. The χ2 test was used to test the significance of observed differences in patients’ characteristics, and Fisher’s exact test was used when data were sparse. Median differences were compared by the Mann–Whitney or Wilcoxon signed-rank test as appropriate. A P value < .05 was considered statistically significant. The Kaplan–Meier method was used to estimate OS (defined as time from study entry to death) and EFS (time from study entry until failure to achieve CR during induction, relapse, or death). RR was calculated by cumulative incidence methods defined as time from the end of induction I for patients in CR to relapse or death, where deaths without a relapse were considered competing events. Patients who withdrew from therapy due to relapse, persistent central nervous system disease, or refractory disease with >20% BM blasts by the end of induction I were defined as induction I failures. The significance of predictor variables was tested with the log-rank statistic for OS, EFS, and with Gray’s statistic for RR. All estimates are reported with 2× the Greenwood standard errors. Children lost to follow-up were censored at their date of last known contact.

BM, bone marrow; CR, complete remission; EFS, event-free survival; FAB, French-American-British; FLT3, FMS-like tyrosine kinase; ITD, internal tandem duplications; MRD, minimal residual disease; NPM, nucleophosmin; OS, overall survival; RR, relapse risk.

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