Search Results - "Galactéros, Frederic"
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1
Eculizumab salvage therapy for delayed hemolysis transfusion reaction in sickle cell disease patients
Published in Blood (25-02-2016)Get full text
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2
Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency
Published in The New England journal of medicine (05-09-2019)“…In this study, approximately half the patients with red-cell pyruvate kinase deficiency who were treated with mitapivat had an improvement in their hemoglobin…”
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3
Incidence and predictive score for delayed hemolytic transfusion reaction in adult patients with sickle cell disease
Published in American journal of hematology (01-12-2017)“…Delayed hemolytic transfusion reaction (DHTR) is a life‐threatening complication of transfusion in sickle cell disease (SCD). The frequency of DHTR is…”
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4
Complement activation in sickle cell disease: Dependence on cell density, hemolysis and modulation by hydroxyurea therapy
Published in American journal of hematology (01-05-2020)“…The complement system is an innate immune defense cascade that can cause tissue damage when inappropriately activated. Evidence for complement over activation…”
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Epidemiology and disease burden of sickle cell disease in France: A descriptive study based on a French nationwide claim database
Published in PloS one (09-07-2021)“…Context Sickle cell disease (SCD) is a severe hematological disorder. The most common acute complication of SCD is vaso-occlusive crisis (VOC), but SCD is a…”
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6
Clinical and biological features in PIEZO1 -hereditary xerocytosis and Gardos channelopathy: a retrospective series of 126 patients
Published in Haematologica (Roma) (01-08-2019)“…We describe the clinical, hematologic and genetic characteristics of a retrospective series of 126 subjects from 64 families with hereditary xerocytosis…”
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Clinical management of adult sickle-cell disease
Published in Current opinion in hematology (01-05-2012)“…PURPOSE OF REVIEWThis review provides an overview of the clinical management of sickle-cell disease (SCD) with recently published findings. RECENT…”
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Sotatercept, a novel transforming growth factor β ligand trap, improves anemia in β-thalassemia: a phase II, open-label, dose-finding study
Published in Haematologica (Roma) (01-03-2019)“…β-thalassemia, a hereditary blood disorder caused by defective synthesis of hemoglobin β globin chains, leads to ineffective erythropoiesis and chronic anemia…”
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Variant-aware saturating mutagenesis using multiple Cas9 nucleases identifies regulatory elements at trait-associated loci
Published in Nature genetics (01-04-2017)“…Stuart Orkin, Daniel Bauer and colleagues present DNA Striker , a computational tool to design variant-aware saturating-mutagenesis screens with multiple…”
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10
Mitapivat versus Placebo for Pyruvate Kinase Deficiency
Published in The New England journal of medicine (14-04-2022)“…Pyruvate kinase deficiency, the most common genetic lesion in the glycolytic pathway, leads to chronic hemolytic anemia. Mitapivat, an oral agent, can activate…”
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11
Clinical phenotypes and outcomes of precapillary pulmonary hypertension of sickle cell disease
Published in The European respiratory journal (01-12-2019)“…Precapillary pulmonary hypertension (PH) is a devastating complication of sickle cell disease (SCD). Little is known about the influence of the SCD genotype on…”
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12
Whole‐blood CCR7 expression and chemoattraction in red blood cell alloimmunization
Published in British journal of haematology (01-07-2021)Get full text
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Real‐Life experience with hydroxyurea in patients with sickle cell disease: Results from the prospective ESCORT‐HU cohort study
Published in American journal of hematology (01-10-2021)“…Several controlled studies have evidenced good efficacy and short‐term and mid‐term safety profiles for hydroxyurea (HU), which has become the cornerstone for…”
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14
Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia
Published in Nature (London) (16-09-2010)“…Gene therapy success Blood disorders caused by abnormal β-globin — β-thalassaemia and sickle cell disease — are the most prevalent inherited disorders…”
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15
How to implement endurance exercise training in sickle cell disease
Published in Haematologica (Roma) (01-05-2021)Get full text
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A Hemodynamic Study of Pulmonary Hypertension in Sickle Cell Disease
Published in The New England journal of medicine (07-07-2011)“…Patients with sickle cell disease underwent ECG with assessment of tricuspid valve regurgitant jet velocity (TRJV) to screen for pulmonary hypertension,…”
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A rare G6PD variant (c.383T > G; p.128Leu > Arg) with a molecular pathophysiological mechanism similar to that of G6PD A − (68Val > Met, 126Asn > Asp)
Published in Blood cells, molecules, & diseases (01-11-2009)“…We report the second documented observation of a rare class-III variant, we named G6PD Pyrgos, [c.383 T > G, p.128Leu > Arg] found in a Greek family. A…”
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Fetal Hemoglobin Decrease During Voxelotor Treatment
Published in European journal of haematology (21-10-2024)“…Voxelotor modifies hemoglobin-oxygen affinity improving anemia and reducing hemolysis in sickle cell patients. However, the impact of Voxelotor on fetal…”
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Determination of hydroxyurea in human plasma by HPLC-UV using derivatization with xanthydrol
Published in Journal of chromatography. B, Analytical technologies in the biomedical and life sciences (01-10-2017)“…[Display omitted] •Hydroxyurea is a challenging compound for chromatographic separation and detection.•A simple method for hydroxyurea determination in plasma…”
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20
Cardiac diastolic maladaptation is associated with the severity of exercise intolerance in sickle cell anemia patients
Published in Scientific reports (15-05-2024)“…This pilot study focusing on Sickle Cell Anemia (SCA) patients offers a comprehensive and integrative evaluation of respiratory, cardiovascular, hemodynamic,…”
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