MDS-MSCs that fail to respond to hypomethylating therapy are associated with patients with rapid adverse disease transformation and this further suggests that MDS-MSCs may be an integral part of disease progression and have prognostic value as well as potential as a therapeutic target.
This study included patients with IPSS lower-risk MDS from the DRAMA (An Observational Study for Dacogen Long-Term Treatment in Patients With Myelodysplastic Syndrome; NCT01400633) and DIVA (A Study for Dacogen Treatment in Patients With Myelodysplastic Syndrome; NCT01041846) studies, which were prospective observational studies on the efficacy and safety of decitabine treatment in patients with MDS.
MDS patients with high (≥median) TCF4 mRNA expression had higher hemoglobin (Hb) levels than MDS patients with low TCF4 expression (mean 9.0 vs. 8.55 g/dL, p = 0.02).
<b>Objectives:</b> To evaluate the value of Wilms' tumor 1 mRNA (<i>WT1</i>) expression in the differential diagnosis of childhood myelodysplastic syndrome (MDS) and aplastic anemia (AA).
Note that 100 mg daily was selected as the RP2D for glasdegib in combination with standard chemotherapies in the absence of an estimated MTD in this setting.<b>Conclusions:</b> Treatment with glasdegib in combination with standard chemotherapy was generally well-tolerated and consistent with prior findings, warranting further evaluation of glasdegib-based combinations in patients with AML or high-risk MDS.<i></i>.
We hypothesized that vaccinating against NY-ESO-1 in patients with MDS receiving decitabine would capitalize upon induced NY-ESO-1 expression in malignant myeloid cells to provoke an NY-ESO-1-specific MDS-directed cytotoxic T-cell immune response.<b>Experimental Design:</b> In a phase I study, 9 patients with MDS received an HLA-unrestricted NY-ESO-1 vaccine (CDX-1401 + poly-ICLC) in a nonoverlapping schedule every four weeks with standard-dose decitabine.<b>Results:</b> Analysis of samples serially obtained from the 7 patients who reached the end of the study demonstrated induction of <i>NY-ESO-1</i> expression in 7 of 7 patients and NY-ESO-1-specific CD4<sup>+</sup> and CD8<sup>+</sup> T-lymphocyte responses in 6 of 7 and 4 of 7 of the vaccinated patients, respectively.
We hypothesized that vaccinating against NY-ESO-1 in patients with MDS receiving decitabine would capitalize upon induced NY-ESO-1 expression in malignant myeloid cells to provoke an NY-ESO-1-specific MDS-directed cytotoxic T-cell immune response.<b>Experimental Design:</b> In a phase I study, 9 patients with MDS received an HLA-unrestricted NY-ESO-1 vaccine (CDX-1401 + poly-ICLC) in a nonoverlapping schedule every four weeks with standard-dose decitabine.<b>Results:</b> Analysis of samples serially obtained from the 7 patients who reached the end of the study demonstrated induction of <i>NY-ESO-1</i> expression in 7 of 7 patients and NY-ESO-1-specific CD4<sup>+</sup> and CD8<sup>+</sup> T-lymphocyte responses in 6 of 7 and 4 of 7 of the vaccinated patients, respectively.
A randomForest analysis of the gene expression array data from 55 MDS patients (GSE4619) demonstrated a significant (p = 0.0007) correlation (Pearson r =-0.4068) between GPI-anchor biosynthesis gene expression and genomic instability, in which PIGN, a gene participating in GPI-AP biosynthesis, was ranked as the third most important in predicting risk of MDS progression.