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American College of Rheumatology Guidance Statement for Diagnosis and Management of VEXAS Developed by the International VEXAS Working Group Expert Panel
Arthritis &Rheumatology, EarlyView.Objective
Vacuoles E1 enzyme X‐linked autoinflammatory somatic syndrome (VEXAS) is a recently identified rare genetic disorder associated with somatic mutations in the UBA1 gene. VEXAS presents with a combination of inflammatory and hematologic manifestations, leading to increased morbidity and mortality.Arsene Mekinian, Sophie Georgin‐Lavialle, Marcela A. Ferrada, Sinisa Savic, Matthew J. Koster, Olivier Kosmider, Thibault Comont, Mael Heilblig, Juan I. Arostegui, Annmarie Bosco, Rim Bourguiba, Katherine R. Calvo, Catherine Cargo, Chiara Cattaneo, François Chasset, Henrique Coelho, Corrado Campochiaro, Francesca Crisafulli, Stephanie Ducharme‐Benard, Raquel Faria, Franco Franceschini, Micol Frassi, Emma M. Groarke, Carmelo Gurnari, Yervand Hakobyan, Yvan Jamilloux, Ciprian Jurcut, Yohei Kirino, Austin Kulasekararaj, Hiroyoshi Kunimoto, Lauren M. Madigan, Heřman F. Mann, Chiara Marvisi, Marcin Milchert, Sara Morais, Katja Sockel, Francesco Muratore, Hideaki Nakajima, Mrinal M. Patnaik, Luísa Regadas, Marie Robin, Abraham Rutgers, Carlo Salvarani, Anthony M. Sammel, Joerg Seebach, Pierre Sujobert, Alessandro Tomelleri, Geoffrey Urbanski, Frédéric Vandergheynst, Romana Vieira, David S. Viswanatha, Ewa Więsik‐Szewczyk, Elisa Diral, Benjamin Terrier, Bhavisha A. Patel, Pierre Fenaux, Peter C. Grayson, David B. Beck, on behalf of the International VEXAS working group, and with endorsement of EuroBloodNet, the European Reference Network in Rare Hematological Diseases, Heřman Mann, Benjamin Terrier, François Chasset, Sophie Georgin Lavialle, Alessandro Tomelleri, Campochiaro Corrado, Carlo Salvarani, Francesca Crisafulli, Franco Franceschini, Micol Frassi, Yohei Kirino, Ewa Więsik‐Szewczyk, Marcin Milchert, Raquel Faria, Ciprian Jurcut, Joerg Seebach, Sinisa Savic, David Beck, Lauren Madigan, Matthew Koster, Patnaik Mrinal, Olivier Kosmider, Pierre Sujobert, Juan I. Arostegui, Catherine Cargo, David Viswanatha, Yervand Hagopian, Mael Heilblig, Pierre Fenaux, Thibault Comont, Bruno Alessandro, Chiara Cattaneo, Elisa Diral, Sara Morais, Austin Kulasekarara, Emma Groarke, Katherine Calvo, Patel Bhavisha, Anthony Sammel, Arsene Mekinian, Benjamin Terrier, Marie Robin, Sophie Georgin Lavialle, Katja Sockel, Yvan Jamilloux, Carmelo Gurnari, Henrique Coelho, Romana Vieira, Rim Bourguiba, Marcela Ferrada, Peter Grayson +111 morewiley +1 more sourceTherapy-related myeloid neoplasms following treatment with radioiodine
Haematologica, 2012 Background Few data are available on therapy-related myelodysplastic syndromes and acute myeloid leukemia developing after radioiodine treatment.Design and Methods We retrospectively analyzed 39 patients with myeloid neoplasms following radioiodine ...Thomas Schroeder, Andrea Kuendgen, Sabine Kayser, Nicolaus Kröger, Friederike Braulke, Uwe Platzbecker, Viola Klärner, Fabian Zohren, Detlef Haase, Michael Stadler, Richard Schlenk, Akos G. Czibere, Ingmar Bruns, Roland Fenk, Norbert Gattermann, Rainer Haas, Guido Kobbe, Ulrich Germing +17 moredoaj +1 more sourceValue of infliximab (Remicade) in patients with low-risk myelodysplastic syndrome. Final results of a randomized phase II trial (EORTC trial 06023) of the EORTC Leukemia Group [PDF]
, 2011 peer reviewedTumor-necrosis factor alpha activity has been correlated to ineffective erythropoiesis in lower risk myelodysplastic syndromes. Infliximab (Remicade) is an anti-tumor-necrosis factor alpha chimeric antibody that is used in the treatment of ...Amadori, Sergio, Baron, Frédéric, De Witte, Theo, Delforge, Michel, Denzlinger, Claudio, Indrak, Karel, Muus, Petra, Ossenkoppele, Geert, Selleslag, Dominik, Suciu, Stefan, Thyss, Antoine, Zwierzina, Heinz +11 morecore +2 more sourcesComparative Efficacy and Safety of Anakinra and Canakinumab in Patients With VEXAS Syndrome: An International Multicenter Study
Arthritis &Rheumatology, EarlyView.Objective
The aim of this study was to compare differences in clinical response, drug survival, and adverse event rates between anakinra and canakinumab in VEXAS (vacuoles, E1 enzyme, X‐linked, autoinflammatory, somatic) syndrome. Methods
This multicenter international study includes patients with VEXAS from France, Israel, and Italy treated with ...Tali Eviatar, Dafne Capelusnik, Corrado Campochiaro, Valentin Lacombe, Vincent Jachiet, Michael Zisapel, Iftach Sagy, Oshrat E. Tayer‐Shifman, David Ozeri, Shaye Kivity, Alessandro Tomelleri, Benjamin Terrier, Hagit Peleg, Thibault Comont, Karim Sacre, Pascal Woaye‐Hune, Laurent Arnaud, Estibaliz Lazaro, Vincent Grobost, Francois Lifermann, Maxime Samson, Samuel Ardois, Alice Garnier, Alexandre Maria, Alain Cantagrel, Aurore Meyer, Jean‐David Bouaziz, Mael Heiblig, Lorenzo Dagna, Elisa Diral, Olivier Kosmider, Ori Elkayam, Jérôme Hadjadj, Sophie Georgin‐Lavialle, Olivier Fain, Arsene Mekinian +35 morewiley +1 more sourceTargeted re-sequencing analysis of 25 genes commonly mutated in myeloid disorders in del(5q) myelodysplastic syndromes
Haematologica, 2013 Interstitial deletion of chromosome 5q is the most common chromosomal abnormality in myelodysplastic syndromes. The catalogue of genes involved in the molecular pathogenesis of myelodysplastic syndromes is rapidly expanding and next-generation sequencing Marta Fernandez-Mercado, Adam Burns, Andrea Pellagatti, Aristoteles Giagounidis, Ulrich Germing, Xabier Agirre, Felipe Prosper, Carlo Aul, Sally Killick, James S. Wainscoat, Anna Schuh, Jacqueline Boultwood +11 moredoaj +1 more sourcePreleukemia: one name, many meanings. [PDF]
, 2017 Definition of preleukemia has evolved. It was first used to describe the myelodysplastic syndrome (MDS) with a propensity to progress to acute myeloid leukemia (AML). Individuals with germline mutations of either RUNX1, CEBPA, or GATA2 can also be called Koeffler, HP, Leong, Gcore A clinical-molecular update on azanucleoside-based therapy for the treatment of hematologic cancers [PDF]
, 2016 The azanucleosides azacitidine and decitabine are currently used for the treatment of acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) in patients not only eligible for intensive chemotherapy but are also being explored in other ...Buschbeck, Marcus, Diesch, Jeannine, Garz, Anne-Kathrin, Götze, Katharina S., Palau, Anna, Zwick, Anabel +5 morecore +2 more sourcesTelomere Length of Peripheral Blood Leukocytes Predicts Disease Severity and Worse Survival in Systemic Sclerosis
Arthritis &Rheumatology, EarlyView.Objective
Peripheral blood leukocyte telomere length (PBL‐TL) shortening is associated with systemic sclerosis–related interstitial lung disease (SSc‐ILD). However, its association with other organ involvement, disease severity, and survival remains unclear.Monica M. Yang, Shuo Liu, Michael Wax II, Seoyeon Lee, Sarah French, Paul J. Wolters, Francesco Boin +6 morewiley +1 more sourceTransforming growth factor β1-mediated functional inhibition of mesenchymal stromal cells in myelodysplastic syndromes and acute myeloid leukemia
Haematologica, 2018 Mesenchymal stromal cells are involved in the pathogenesis of myelodysplastic syndromes and acute myeloid leukemia, but the underlying mechanisms are incompletely understood.Stefanie Geyh, Manuel Rodríguez-Paredes, Paul Jäger, Annemarie Koch, Felix Bormann, Julian Gutekunst, Christoph Zilkens, Ulrich Germing, Guido Kobbe, Frank Lyko, Rainer Haas, Thomas Schroeder +11 moredoaj +1 more sourceThe myelodysplastic syndromes [PDF]
Clinical & Laboratory Haematology, 2008 Clinicomorphological features of myelodysplastic syndromes, Antonio Pagliuca and Ghulam J. Mufti FAB and MIC classification of myelodysplastic syndromes - points of controversy, John M. Bennett incidence and epidemiology of the myelodysplastic syndromes, R. A. Cartwright pathogenesis and evolution of refractory anaemia, A. Jacobs and D.openaire +2 more sources