Table 1.
Results of miRNA profiles within clinical trials including patients with AML.
| References | Cell source | Training cohort (n°) | Validation cohort (n°) | Dysregulated miRNAs | Prognostic or Risk-group correlation | Multivariable analysis |
Parameters included at the multivariable analysis |
|---|---|---|---|---|---|---|---|
| Havelange et al, 2014 | Pre-treatment BM | 54 adult patients with de novo AML | 183 elderly patients (≥60 yrs) | miR-10 family | Higher miR-10a and miR-10b levels associated with the odds of achieving CR | Yes | miR-10a and -10b levels, NPM1 mutations, age, unfavorable cytogenetic, CN-AML (training cohort). miR-10a and -10b, NPM1 mutations, BAALC expression (validation cohort) |
| Li et al, 2013 | pre-treatment BM and PB | 81 patients AML-M2 (AML/ETO+ and AML/ETO-) | 89 patients with(8;21) AML | miR-193a | overexpression of miR-193a: better EFS and OS | No | NA |
| Marcucci et al, 2013 | pre-treatment BM and PB | 363 patients with de novo NC-AML | Not included | miR-155 | High miR-155levels were associated with WBC counts, percentage of blasts, FLT3-ITD, RUNX1 mutated, WT1 mutated, high ERG and BAALC High miR-155 had lower odds of achieving CR, shorter DFS and OS |
Yes | Age, race, WBC count, miR-155, NPM1status, FLT3-ITD, BAALC and ERG expression |
| Blum et al, 2010 | pre-treatment BM | 53 previously untreated AML patients ≥60 yrs | Not included | miR-29b | High pre-treatment miR-29b had better response to decitabine | No | NA |
| Schwind et al, 2010 | pre-treatment BM | 187 adult patients with CN-AML < 60 yrs | 122 patients with CN-AML, ≥60 yrs | miR-181a | Higher miR-181a associated with better CR, longer OS and a trend in longer DFS | Yes | Age, WBC count, miR-181a, ERG and BAALCexpressione, CEBPA and NPM1mutation, WT1, FLT3-ITD. |
| Whitman et al, 2010 | pre-treatment BM or PB | 243 adult patients with CN-AML | not included | miR-155, miR-144 miR-451 | Overexpression of miR-155 and underexpression of miR-144 and miR-451 associated with FLT3-ITD | ||
| Garzon et al, 2008 | pre-treatment BM | 85 adult patients with AML | not included | miR-10a, miR-10b, miR-204, miR-155 |
miR-10a, miR-10b, let-7, miR-29 family were upregulated in NPMc+ AML miR-155 was upregulated in FLT3-ITD AML |
No | NA |
| Marcucci et al, 2008 | pre-treatment BM | 64 patients with high molecular risk CN-AML < 60 yrs | 55 patients with high molecular risk CN-AML | miR-181 family, miR-124, miR-128, miR-194, miR-219, miR-220a, miR-320 |
miR-181 family was inversely associated with EFS (death, lack of CR, or relapse) miR-124, miR-128, miR 194, miR-219, miR-220a, miR-320 were positively with EFS |
Yes | MicroRNA summary value, ratio of FLT3-ITD to FLT3 (high vs low) and WBC count (log), age, sex, race, Hb level, platelet count, percentage of blasts, the presence or absence of extramedullary involvement, NPM1 mutation, and BAALC and ERG expression |
Annotations: CN-AML, cytogenetics normal, acute myeloid leukemia; yrs, years; EFS, event free survival; OS, overall survival, WBC, white blood cell; FLT3-ITD, fms-like tyrosine receptor internal tandem duplications; WT1, Wilm’s tumor gene; BAALC, brain and acute leukemia cytoplasmic gene; CR, complete response; DFS, disease free survival; NPM1, nuclephosmin; CEBPA, CCAAT/enhancer binding protein-alpha gene; NPMc+, nucleophosmin cytoplasmic positive.