Myelodysplastic Syndromes (MDS)
Retuning the immune system in myelodysplastic syndromes: from immunomodulatory approaches to vaccination strategies and non myeloablative hemopoietic cell transplant.
Several findings suggest that the pathogenesis and clinical history of myelodysplastic syndromes (MDS) is influenced by a variety of immune pathways and mechanisms. Coherently several therapeutic approaches based on the idea of modulating the immune system have been exploited in this clinical setting. The present review will first consider more consolidated strategies such as antithymocyte globulin and immunomodulatory (IMiDs) analogues. Less explored approaches, such as anti tumor necrosis factor antibodies, cyclosporin and bortezomib will be also evaluated.
Prognostic value and clinical feature of SF3B1 mutations in myelodysplastic syndromes: A meta-analysis.
SF3B1 gene mutations are the most frequent mutations found in myelodysplastic syndromes (MDS), and the prognostic implication of these mutations remains controversial. We conducted a meta-analysis of studies assessing the prognostic impact and clinical feature of SF3B1 mutations in MDS patients. The overall hazard ratio for overall survival (OS) was 0.90 (95% confidence interval 0.60-1.35, P = 0.61) in MDS patients with SF3B1 mutations compared to those without. Lower leukemia-free survival was associated with SF3B1 mutations.
The central role of inflammatory signaling in the pathogenesis of myelodysplastic syndromes
In cancer biology, tumor-promoting inflammation and an inflammatory microenvironment play a vital role in disease pathogenesis. In the past decade, aberrant innate immune activation and proinflammatory signaling within the malignant clone and the bone marrow (BM) microenvironment were identified as key pathogenic drivers of myelodysplastic syndromes (MDS). In particular, S100A9-mediated NOD-like receptor protein 3 (NLRP3) inflammasome activation directs an inflammatory, lytic form of cell death termed pyroptosis that underlies many of the hallmark features of the disease.
Rethinking clinical trial endpoints in myelodysplastic syndromes.
The myelodysplastic syndromes (MDS) are a heterogeneous collection of clonal, hematopoietic disorders primarily affecting an older population, making successful drug development a complicated process. A sole focus on response rate in clinical trials is likely not clinically meaningful if not accompanied by substantive response duration, improvement in quality of life, and ideally prolongation of survival. The process of receiving a new therapy should not be more burdensome than the MDS sequela it is intended to ameliorate.
A Phase II Study to Determine the Safety and Efficacy of the Oral Inhibitor of Indoleamine 2,3-Dioxygenase (IDO) Enzyme INCB024360 in Patients with Myelodysplastic Syndromes
BACKGROUND:
INCB024360 is an oral inhibitor of the enzyme indoleamine 2,3-dioxygenase (IDO), which catalyzes the degradation of tryptophan to kynurenine. Preclinical data suggest that IDO1 inhibition by INCB024360 will increase T cell proliferation, and decrease T regulatory cells and myeloid derived suppressor cells suppressive activity.
Fatigue, symptom burden, and health-related quality of life in patients with myelodysplastic syndrome, aplastic anemia, and paroxysmal nocturnal hemoglobinuria.
BACKGROUND:
Fatigue is distressing and affects quality of life (QoL) among patients with myelodysplastic syndrome (MDS), aplastic anemia (AA), and paroxysmal nocturnal hemoglobinuria (PNH).
Patient Trusts Her Instincts in Finding the Right Treatment
I never thought I would be diagnosed with cancer. I taught exercise classes, walked every day, ate a Mediterranean diet, and paid attention to products I used on my skin. I became a certified health coach and taught healthy living classes.