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Article
Open AccessPractical recommendations on the use of lenalidomide in the management of myelodysplastic syndromes
Lenalidomide, an oral immunomodulatory agent, has received approval in the USA from the Food and Drug Administration (FDA) for the management of myelodysplastic syndromes (MDS) classified by the International ...
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Article
Open AccessValuation of transfusion-free living in MDS: results of health utility interviews with patients
This study measured how myelodysplastic syndrome (MDS) patients value transfusion independence (TI), reduced transfusions (RT) and transfusion-dependence (TD) using health utility assessment methodology.
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Article
Open AccessA European survey on the detection and management of iron overload in transfusion-dependent patients with MDS
To better understand the detection and management of iron overload in transfusion-dependent patients with myelodysplastic syndromes (MDS), a 15-min web- or paper-based survey was conducted among 338 European p...
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Article
Open AccessLenalidomide as a disease-modifying agent in patients with del(5q) myelodysplastic syndromes: linking mechanism of action to clinical outcomes
Deletion of the long arm of chromosome 5, del(5q), is the most prevalent cytogenetic abnormality in patients with myelodysplastic syndromes (MDS). In isolation, it is traditionally associated with favorable pr...
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Article
Erratum to: Flt3 receptor inhibition reduces constitutive NFκB activation in high-risk myelodysplastic syndrome and acute myeloid leukemia
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Article
Open AccessGEP analysis validates high risk MDS and acute myeloid leukemia post MDS mice models and highlights novel dysregulated pathways
In spite of the recent discovery of genetic mutations in most myelodysplasic (MDS) patients, the pathophysiology of these disorders still remains poorly understood, and only few in vivo models are available to...
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Article
Open AccessMutation allele burden remains unchanged in chronic myelomonocytic leukaemia responding to hypomethylating agents
The cytidine analogues azacytidine and 5-aza-2’-deoxycytidine (decitabine) are commonly used to treat myelodysplastic syndromes, with or without a myeloproliferative component. It remains unclear whether the r...
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Article
Open AccessClinical characteristics and outcomes according to age in lenalidomide-treated patients with RBC transfusion-dependent lower-risk MDS and del(5q)
Particularly since the advent of lenalidomide, lower-risk myelodysplastic syndromes (MDS) patients with del(5q) have been the focus of many studies; however, the impact of age on disease characteristics and re...
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Article
Open AccessCytomorphology review of 100 newly diagnosed lower-risk MDS patients in the European LeukemiaNet MDS (EUMDS) registry reveals a high inter-observer concordance
The European LeukemiaNet MDS (EUMDS) registry is collecting data of myelodysplastic syndrome (MDS) patients belonging to the IPSS low or intermediate-1 category, newly diagnosed by local cytologists. The diagn...
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Open AccessValidation of a post-hypomethylating agent failure prognostic model in myelodysplastic syndromes patients treated in a randomized controlled phase III trial of rigosertib vs. best supportive care
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Article
Open AccessDual origin of relapses in retinoic-acid resistant acute promyelocytic leukemia
Retinoic acid (RA) and arsenic target the t(15;17)(q24;q21) PML/RARA driver of acute promyelocytic leukemia (APL), their combination now curing over 95% patients. We report exome sequencing of 64 matched sampl...
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Article
Open AccessHealth-related quality of life in lower-risk MDS patients compared with age- and sex-matched reference populations: a European LeukemiaNet study
In myelodysplastic syndromes (MDS), health-related quality of life (HRQoL) represents a relevant patient-reported outcome, which is essential in individualized therapy planning. Prospective data on HRQoL in lo...
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Open AccessThe impact of lenalidomide exposure on response and outcomes in patients with lower-risk myelodysplastic syndromes and del(5q)
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Article
Open AccessMulticenter validation of the flow measurement of classical monocyte fraction for chronic myelomonocytic leukemia diagnosis
Peripheral blood monocytes include three subsets defined by CD14 and CD16 surface markers. An increase in the CD14++CD16− classical monocyte fraction ≥ 94% of the total monocytes was proposed to rapidly and effic...
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Article
Open AccessA phase 3 randomized, placebo-controlled study assessing the efficacy and safety of epoetin-α in anemic patients with low-risk MDS
Erythropoiesis-stimulating agents are first choice for treating anemia in low-risk MDS. This double-blind, placebo-controlled study assessed the efficacy and safety of epoetin-α in IPSS low- or intermediate-1 ...
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Article
Open AccessTP53 mutation status divides myelodysplastic syndromes with complex karyotypes into distinct prognostic subgroups
Risk stratification is critical in the care of patients with myelodysplastic syndromes (MDS). Approximately 10% have a complex karyotype (CK), defined as more than two cytogenetic abnormalities, which is a hig...
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Article
Open AccessThe complex genetic landscape of familial MDS and AML reveals pathogenic germline variants
The inclusion of familial myeloid malignancies as a separate disease entity in the revised WHO classification has renewed efforts to improve the recognition and management of this group of at risk individuals....
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Open AccessSingle agent talacotuzumab demonstrates limited efficacy but considerable toxicity in elderly high-risk MDS or AML patients failing hypomethylating agents
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Article
Open AccessOutcome of older (≥70 years) APL patients frontline treated with or without arsenic trioxide—an International Collaborative Study
Data on outcome in older (≥70 years) patients with acute promyelocytic leukemia after treatment with arsenic trioxide (ATO) compared with standard chemotherapy (CTX) is scarce. We evaluated 433 patients (media...
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Author Correction: Implications of TP53 allelic state for genome stability, clinical presentation and outcomes in myelodysplastic syndromes
A Correction to this paper has been published: https://doi.org/10.1038/s41591-021-01253-5.