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Project description:Lower-risk (LR) myelodysplastic syndromes (MDS) are heterogeneous hematopoietic stem and progenitor disorders caused by early accumulation of somatic mutations in epigenetic factors and non-epigenetic factors that may produce convergent DNA methylation patterns driving specific gene expression profiles. The integration of genomic, epigenomic and transcriptomic profiling has the potential to spotlight distinct LR-MDS categories on the basis of pathophysiological mechanisms.
2025-01-29 | GSE220518 | GEO
Project description:Lower-risk (LR) myelodysplastic syndromes (MDS) are heterogeneous hematopoietic stem and progenitor disorders caused by early accumulation of somatic mutations in epigenetic factors and non-epigenetic factors that may produce convergent DNA methylation patterns driving specific gene expression profiles. The integration of genomic, epigenomic and transcriptomic profiling has the potential to spotlight distinct LR-MDS categories on the basis of pathophysiological mechanisms.
2025-01-29 | GSE256394 | GEO
Project description:Lower-risk myelodysplastic syndromes are defined using prognostic scoring systems that incorporate data on bone marrow blast percentage, degree and numbers of cytopenias, and cytogenetic abnormalities. Increasingly, these are incorporating molecular abnormalities to further refine risk. Therapy is geared toward predominating cytopenias, with erythropoiesis-stimulating agents luspatercept and lenalidomide used to ameliorate anemia, romiplostim and eltrombopag tackling thrombocytopenia, and hypomethylating agents and antithymocyte globulin palliating pancytopenia. Newer agents on the horizon are abrogating the downstream sequelae of specific molecular mutations. One challenge for the future is in further modifying response criteria to align with improvements that are clinically meaningful to patients.
| S-EPMC6913460 | biostudies-literature
Project description:Myelodysplastic syndromes (MDS) are a heterogeneous group of hematopoietic stem cell disorders characterized by ineffective hematopoiesis with abnormal blood cell development (dysplasia) leading to cytopenias and an increased risk for progression to acute myeloid leukemia (AML). Patients with MDS can generally be classified as lower- (LR-MDS) or higher-risk (HR-MDS). As treatment goals for patients with LR-MDS and those with HR-MDS differ significantly, appropriate diagnosis, classification, and follow-up are critical for correct disease management. In this review, we focus on the diagnosis, prognosis, and treatment options, as well as the prediction of the disease course and monitoring of treatment response in patients with LR-MDS. We discuss how next-generation sequencing, increasing knowledge on mechanisms of MDS pathogenesis, and novel therapies may change the current treatment landscape in LR-MDS and why structured assessments of responses, toxicities, and patient-reported outcomes should be incorporated into routine clinical practice.
| S-EPMC9751093 | biostudies-literature
Project description:Myelodysplastic syndrome is one of the main hematological malignancies that threaten the health of the elderly. However, biomarkers which predict the progression and prognosis of MDS are still controversial and puzzling. FOXO1 gene plays an important role in a variety of intracellular functions, including tumor suppression and cellular immune regulation. However, there is no research report on the correlation between FOXO1 and the clinical features of MDS including immune environment. In this study, we observed that FOXO1 expression is associated with neutrophil count, blasts, chromosome and different MDS scoring systems. FOXO1 expression is closely related to MDS cell immune polarization, and the increase expression of FOXO1 is significantly related to the amplification of immune cell polarization ratio. In addition, FOXO1 expression is associated with progression-free survival and overall survival in MDS patients. Moreover, in a multivariate model FOXO1 low-expression was an independent predictor of poor survival in MDS. In summary, FOXO1 may play a candidate tumor suppressor in MDS, and FOXO1 is a useful independent prognostic predictor in MDS, and it may provide a candidate target therapy in future.
| S-EPMC9600488 | biostudies-literature
Project description: Not available
| S-EPMC7932918 | biostudies-literature
Project description:Myelodysplastic syndromes (MDS) are a heterogeneous group of clonal hematological disorders. Treatment options are classified and defined by prognostic risk based on the International Prognostic Scoring System (IPSS) and, more recently, the revised IPSS (IPSS-R). The treatment goal for lower-risk MDS is to correct cytopenias or their consequences, with the goal of maintaining or improving quality of life. Erythropoiesis-stimulating agents (ESAs) play an important role in treating anemia. Individuals with MDS who have a 5q deletion are particularly sensitive to treatment with lenalidomide; however, this comprises the minority of patients with MDS. Luspatercept was recently approved in the United States and the European Union for the treatment of ESA-refractory MDS with ring sideroblasts. Research into new treatment options, especially after ESA failure, is needed. In this review, we will focus on the current therapeutic options for MDS-related anemia.
| S-EPMC9713208 | biostudies-literature
Project description:Patients with myelodysplastic syndromes (MDS) are at risk of early death from cardiovascular complications due to the link between clonal hematopoiesis and endothelial dysfunction. EASIX (Endothelial Activation and Stress Index) has been established to predict endothelial complications after allogeneic transplantation. We investigated the impact of EASIX measured at first diagnosis on survival of patients with lower- and higher-risk MDS (no allogeneic transplantation) in two independent institutions: n = 192 (training cohort) and n = 333 (validation cohort). Serum markers of endothelial cell distress were measured and correlated to EASIX. While no effects of EASIX on survival were observed in higher-risk patients, EASIX was associated with shorter survival in patients with lower-risk MDS in both cohorts (univariate: Cohort I: hazard ratio (HR): 1.46; 95% confidence interval (CI) 1.24-1.71; p-value < 0.001/Cohort II: HR 1.31 [1.17-1.48]; p-value < 0.001). Multivariate Cox regression analysis and prediction error analyses confirmed that EASIX remained a significant predictor of survival after adjustment for age, sex, cytogenetic abnormalities and bone marrow blasts in lower-risk patients. The model of the training cohort could be validated. Serum levels of Angiopioetin-2 correlated significantly with EASIX. We introduce EASIX as an easily accessible and independent predictor for survival in patients with lower-risk MDS.
| S-EPMC6848148 | biostudies-literature
Project description:Supplemental Digital Content is available in the text.
| S-EPMC7000478 | biostudies-literature
Project description:Myelodysplastic syndromes (MDS) are a spectrum of clonal stem-cell disorders characterized clinically by bone-marrow failure. Resultant cytopenias are responsible for significant mortality and decreased quality of life in patients with MDS. In patients with low-risk MDS (LR-MDS), anemia is the most common cytopenia and erythropoiesis-stimulating agents (ESA) are usually used as first-line therapy. Those patients who become refractory to ESA have a poor survival. Available treatment options such as lenalidomide, hypomethylating agents, and immunosuppressive therapy can provide some hematologic response among selected subsets of patients, however durable responses are limited, and these agents can carry significant adverse effects. Chronic transfusions help to alleviate symptoms of anemia but still carry risks associated with transfusion and iron overload. Luspatercept, recently approved for those LR-MDS with ring sideroblasts refractory to ESA, was found to have an improvement in transfusion independence with a well-tolerated safety profile. While anemia is the most common cytopenia, thrombocytopenia and neutropenia management is challenging and the co-occurrence of these cytopenias with anemia may dictate the choice of therapy. In this article, we review LR-MDS and discuss the optimal use of current treatment options and explore new therapeutic options on the horizon.
| S-EPMC7812395 | biostudies-literature