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Optimal red cell transfusion support in myelodysplastic syndromes (MDS) has not been tested and established. The aim of this study was to demonstrate feasibility of recruitment and follow-up in an outpatient setting with an exploratory assessment of quality of life (QoL) outcomes (EORTC QLQ-C30 and EQ-5D-5L). We randomised MDS patients to standardised transfusion algorithms comparing current restrictive transfusion thresholds (80 g/l, to maintain haemoglobin 85-100 g/l) with liberal thresholds (105 g/l, maintaining 110-125 g/l). The primary outcomes were measures of compliance to transfusion thresholds. Altogether 38 patients were randomised (n = 20 restrictive; n = 18 liberal) from 12 participating sites in UK, Australia and New Zealand. The compliance proportion for the intention-to-treat population was 86% (95% confidence interval 75-94%) and 99% (95-100%) for restrictive and liberal arms respectively. Mean pre-transfusion haemoglobin concentrations for restrictive and liberal arms were 80 g/l (SD6) and 97 g/l (SD7). The total number of red cell units transfused on study was 82 in the restrictive and 192 in the liberal arm. In an exploratory analysis, the five main QoL domains were improved for participants in the liberal compared to restrictive arm. Our findings support the feasibility and need for a definitive trial to evaluate the effect of different red cell transfusion thresholds on patient-centred outcomes., (© 2020 British Society for Haematology and John Wiley & Sons Ltd.)

Background: Myelodysplastic Syndromes (MDS) are a group of clonal hematopoietic stem cell disorders with significant heterogeneity in their clinical presentation and the prognosis of the patients. Several attempts have been made to incorporate flow cytometry (FC) findings into the diagnostic and/or prognostic criteria of dysplasia, but bone marrow (BM) aspirate morphology evaluation remains the gold-standard for diagnosis. The purpose of this study was to provide a diagnostic tool for MDS that relies on BM immunophenotyping and objectifies the interpretation of FC analysis and to validate its capacity to discriminate MDS from other causes of cytopenias., Methods: To that purpose, a mathematical formula was developed which incorporates granulocytic maturation markers and the percentage of selected myeloid populations and translates them into a single parameter that quantifies the maturation and differentiation defects of BM granulocytes, named Dysmyelopoiesis Index (DMI). Bone marrow samples from 84 MDS patients and 47 non-MDS cytopenic patients were analyzed with FC and DMI was calculated for every patient., Results: DMI detected clonal dysplasia with 84.5% sensitivity and 93.6% specificity, identified as MDS 77.2% of low grade patients and revealed multilineage dysplasia for a number of RA and RARS cases. It discriminated prognostic subgroups of MDS patients (P< .005) and negatively correlated with IPSS (r= - .472, P= .000), WPSS (r= - .481, P= .000) and IPSS-R (r= -.395, P= .000)., Conclusions: DMI represents an accurate quantification of dysmyelopoiesis and an effective stand-alone diagnostic test for MDS, facilitating FC analysis and daily clinical practice.

Descriptive epidemiology of the myelodysplastic syndromes (MDS) is always interesting and may reveal time-dependent and geographical variations, as well as occupational exposure. Epidemiological data in Greece are not available by now. We have collected and analyzed medical records of all patients with a documented diagnosis of MDS, performed by an expert hematologist and/or hematopathologist, in the geographical area of Western Greece, during the 20-year period, defined between 1990 and 2009. We have then calculated and described demographic and clinical features of the diagnosed MDS patient population, and assessed the incidence and prevalence rates of MDS in Western Greece, during the above-mentioned period. A total of 855 patients with newly diagnosed MDS have been identified. Refractory anemia was the most common subtype in both FAB and WHO classification systems and in both genders. Del-5q and RARS were more commonly encountered among females, and the dysplastic subtype of chronic myelomonocytic leukemia among males. Trisomy 8 was the most common single cytogenetic abnormality. The crude mean annual incidence rate of MDS was 6.0 per 100,000 inhabitants aged ≥15 years old (all subtypes according to FAB), and it was 4.8 per 100,000 when CMML and RAEB-T were excluded. Crude incidence rate was higher in rural than in urban areas, but this finding was not confirmed after age standardization. Age-standardized mean annual incidence rate in men was 7.9/100,000 and in women 3.4/100,000. A continuously increasing incidence rate of MDS has been observed throughout the study period.

Myelodysplastic syndromes (MDS) are clonal stem cell disorders, characterized by ineffective and dysplastic hematopoiesis. MDS patients have a defective immune response manifested by increased susceptibility to bacterial infections, autoimmune phenomena, and high incidence of lymphoid malignancies. Presently, we investigated the phenotype and function of monocyte-derived dendritic cells (MoDC) in 23 MDS patients and 15 controls at different stages of differentiation using the maturation stimuli tumor necrosis factor-alpha (TNF-alpha) and LPS. Monocytes from MDS patients showed low potential to differentiate into dendritic cells (DC), as determined by low cell yield and CD1a expression. MDS-MoDCs exhibited low expression of mannose receptor and reduced endocytic capacity. MDS-MoDCs showed a diminished response to TNF-alpha with low CD83, CD80, and CD54 expression and allostimulatory capacity. In patients with 5q syndrome, monocytes and MoDCs were positive for the 5q deletion, suggesting their origin from the malignant clone. Our data indicate that MoDCs in MDS display quantitative and functional abnormalities that may contribute to the defective immune response of these patients.