Sialidase inhibition to increase platelet counts: A new treatment option for thrombocytopenia.

Finally, we did not detect a specific gene expression signature for GATA2 mutated vs. non-mutated cases. Overall, CR was achieved in 113 patients (88%), of whom 36 relapsed (estimated CIR at 3 and 5 years, 39%). Neither age nor karyotype or gene mutations (including NPM1, FLT3, of GATA2 and type CEBPA mutation) significantly influenced CIR in multivariate analysis. Five-year OS was estimated at 58% in the whole cohort, with longer OS in cases with normal karyotype (P5 0.05) and CEBPA biallelic mutations (dm vs. sm, P5 0.03). In multivariate analysis, younger age (P5 0.020) and normal karyotype (P5 0.029) remained the only factors significantly associated with better overall survival. Of note, CEBPA bi-allelic mutations occur more frequently in younger patients. More specifically, the presence of GATA2 mutations did not affect OS in CEBPA mutated AML in general and not within CEBPA dm mutated AML neither. In conclusion, we confirmed a strong association of GATA2 mutations with CEPBA biallelic-mutations, which was not exclusive. However, we confirmed no prognostic impact of GATA2 mutations (n5 25) compared to wild-type (n5 62) in our cohort of CEBPA dm AML [8,9] (22 vs. 58 and 10 vs. 16) with a trend similar of better outcome in CEBPA dm with GATA2 mutations compared to wild-type [5,6] (18 vs. 62). Furthermore, our study provided first evidence that homozygous CEPBA mutations have a similar gene expression signature as CEPBA dm and thus may be considered as equivalent. Acknowledgments. Authors acknowledge the contribution of Nicolas Duployez PharmD, Olivier Nibourel PharmD PhD, Nathalie Helevaut, C eline Rodriguez, Karine Celli-Lebras, C eline Berthon MD PhD, Guy Leverger MD, Nicolas Boissel MD PhD, Christine Terr e PharmD, Xavier Thomas MD, and Aline Renneville PharmD PhD.