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. Author manuscript; available in PMC: 2016 Feb 25.
Published in final edited form as: JAMA. 2015 Aug 25;314(8):811–822. doi: 10.1001/jama.2015.9643

Table.

Characteristics for the 71 Adult Patients With Acute Myeloid Leukemiaa

Refractory Group R6–12 Group LFR Group P Valueb Additional Cases for
Clearance Assessment
Patients in cohort, No. 34 12 25 25
Age at study entry, mean (95% CI), y 51.6 (46.4–56.9) 48.7 (39.0–58.3) 50.6 (45.4–55.8) .82 57.4 (53.1–61.8)
Male sex, No. (%) 15 (44.1) 5 (41.7) 14 (56.0) .59 12 (48.0)
Bone marrow blasts at diagnosis, mean (95% CI), % 72.5 (65.8–79.2) 63.1 (50.2–76.0) 75.4 (69.5–81.3) .15 56.4 (47.5–65.4)
White cell count at diagnosis, mean (95% CI), per mm3 52 600 (37 400–67 700) 75 900 (27 600–124 200) 44 800 (17 700–71 900) .18 33 940 (21 040–46 830)
  Median (IQR) 47 100 (12 000–80 500) 51 900 (15 400–118 200) 19 800 (5 000–59 300) 21 700 (7500–21 700)
Event-free survival, median (95% CI), mo 3.0 (2.2–3.7) 8.2 (6.4–10.2) 20.6 (16.6–40.4) <.001 10.7 (9.5–17.3)
Overall survival, median (95% CI), mo 7.5 (5.7–9.3) 11.7 (7.9–16.4) 42.3 (24.8-NE) <.001 20.4 (15.1–22.2)
Normal cytogenetic profile, No. (%) 13 (38.2) 8 (66.7) 20 (80.0) .02 12 (48.0)
Cytogenetic risk group, No. (%)
  Favorable 0 0 0 6 (24.0)
  Intermediate 19 (55.9) 10 (83.3) 23 (92.0) 14 (56.0)
  Unfavorable 13 (38.2) 2 (16.7) 2 (8.0) 5 (20.0)
  Missing data 2 (5.9) 0 0 0
Total coding mutations, mean (95% CI)c 21.8 (17.7–25.9) 20.8 (14.6–27.1) 16.0 (12.1–19.9) .06 11.8 (9.7–13.9)
  Median (IQR) 20.0 (14.0–27.0) 19.5 (15.0–25.5) 14.0 (9.0–20.0) 12.0 (8.5–14.0)
Common AML somatic mutations, No. (%)
  NPM1 7 (20.6) 6 (50.0) 15 (60.0) .005 8 (32.0)
  DNMT3A 8 (23.5) 5 (41.7) 10 (40.0) .31 5 (20.0)
  FLT3 13 (38.2) 6 (50.0) 10 (40.0) .77 10 (40.0)
  TP53 7 (20.6) 2 (16.7) 1 (4.0) .19 1 (4.0)
  IDH1 and/or IDH2 10 (29.4) 1 (8.3) 10 (40.0) .13 4 (16.0)
  KRAS and/or NRAS 6 (17.7) 2 (16.7) 5 (20) >.99 11 (44.0)
  WT1 6 (17.7) 3 (25) 1 (4.0) .16 1 (4.0)
  RUNX1 5 (14.7) 0 (0.0) 0 (0.0) .08 2 (8.0)
  NF1 4 (11.8) 0 (0.0) 0 (0.0) .16 0
WGS cases, samples 29 12 17
  Total variants, mean (95% CI) 411.2 (336.2–486.3) 422.9 (306.6–539.3) 375.1 (284.6–465.6) .78
    Median (IQR) 435.0 (282.0–509.0) 414.5 (268.0–583.5) 378.0 (287.0–439.0)
  Subclones, mean (95% CI) 1.2 (0.9–1.5) 1.3 (0.6–2.1) 1.4 (0.9–1.9) .78
    Median (IQR) 1.0 (1.0–2.0) 1.0 (0.5–2.5) 1.0 (1.0–2.0)

Abbreviations: AML, acutemyeloid leukemia; IQR, interquartile range; LFR, long first remission; R6–12, relapsed between 6 and 12 months from the initiation of therapy;WGS, whole-genome sequencing.

a

Percentages may not total 100 because of rounding.

b

P values are based on a comparison of the 3 different outcome groups.

c

Coding variants for the 71 refractory, R6–12, or LFR cases are based on the number of coding variants from the WGS or exome sequencing data; for the 25 additional cases for clearance assessment, this number is based on the total number of variants identified by exome sequencing. Common AML somatic mutations are the numbers of cases with any somatic mutation in DNMT3A, TP53 (including deletions), WT1, RUNX1, or NF1, and canonical mutations in IDH1, IDH2, KRAS, NRAS, or FLT3.