Results 41 to 50 of about 3,120 (153)

Increased intervals in enzyme replacement therapy for stable type 1 Gaucher disease: A non‐inferiority sequential trial emulation

Journal of Internal Medicine, EarlyView.
Abstract Objective To compare the efficacy and safety of extended interval (Q3–4W) enzyme replacement therapy (ERT) versus standard biweekly (Q2W) ERT in clinically stable type 1 Gaucher disease (GD) patients. Methods We emulated a target trial with a sequential trial design, using data from the French Gaucher Disease Registry.
Maxime Beydon, Jérôme Stirnemann, Karima Yousfi, Samira Zebiche, Dalil Hamroun, Anaïs Brassier, Samia Pichard, Laure Swiader, Thierry Billette de Villemeur, Bénédicte Héron, Florence Dalbies, Bérengère Cador, Anne‐Sophie Guemann, Francis Gaches, Bénédicte Hivert, Vanessa Leguy‐Seguin, Agathe Masseau, Yves‐Marie Pers, Magali Pettazzoni, Soumeya Bekri, Catherine Caillaud, Edouard Le Guillou, Marie Szymanowski, Leonardo Astudillo, Wladimir Mauhin, Yann Nadjar, Christine Serratrice, Marc G. Berger, Fabrice Camou, Nadia Belmatoug, Yann Nguyen, the French Evaluation of Gaucher Disease Treatment Committee   +31 more
wiley   +1 more source

Acquired von Willebrand syndrome and lymphoproliferative disorders: A case report

Clinical Case Reports, 2020
Acquired von Willebrand syndrome is a rare bleeding disorder often secondary to an underlying lymphoproliferative disorder. We report a case in whom response of both the acquired von Willebrand syndrome and smoldering multiple myeloma persist 14 months ...
Christophe Nicol, Leela Raj, Gaëlle Guillerm, Francis Couturaud, Jean‐Richard Eveillard, Brigitte Pan‐Petesch   +5 more
doaj   +1 more source

American College of Rheumatology Guidance Statement for Diagnosis and Management of VEXAS Developed by the International VEXAS Working Group Expert Panel

Arthritis &Rheumatology, Volume 78, Issue 3, Page 509-522, March 2026.
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 Heiblig, 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 more
wiley   +1 more source

Comparative Efficacy and Safety of Anakinra and Canakinumab in Patients With VEXAS Syndrome: An International Multicenter Study

Arthritis &Rheumatology, Volume 78, Issue 2, Page 475-482, February 2026.
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 more
wiley   +1 more source

Une polyradiculonévrite inflammatoire démyélinisante chronique paranéoplasique secondaire à un lymphome natural killer nasal

The Pan African Medical Journal, 2020
Nous rapportons un cas unique d´une polyradiculonévrite inflammatoire démyélinisante chronique paranéoplasique secondaire à un lymphome non hodgkinien T de type natural killer nasal.
Selim Jennane, Nawal Hasnaoui, Mahtat El Mehdi, Hicham El Maaroufi, Nezha Messaoudi, Mohamed Mikdame, Kamal Doghmi   +6 more
doaj   +1 more source

Long‐term outcome after allogeneic stem cell transplantation for GATA2 deficiency: An analysis of 67 adults and children from France and Belgium

British Journal of Haematology, Volume 208, Issue 1, Page 250-258, January 2026.
The overall survival of patients receiving an allogeneic haematopoietic stem cell transplant (HSCT) for GATA2 deficiency was significantly better if they were transplanted recently, with a bone marrow or cord blood graft and if the transplant was performed before the onset of excess blast. Excess blast before HSCT was the only factor associated with an
Flore Sicre de Fontbrune, Florian Chevillon, Mony Fahd, Kristell Desseaux, Xavier Poiré, Edouard Forcade, Arthur Sterin, Bénédicte Neven, Virginie Gandemer, Sylvain Thepot, Alice Garnier, Bruno Lioure, Ambroise Marcais, Stephanie Nguyen‐Quoc, Suzanne Tavitian, Laure Vincent, Jean Donadieu, Matthieu Resche Riggon, Sylvie Chevret, Marlene Pasquet, Regis Peffault de Latour   +20 more
wiley   +1 more source

Perceived disabling physical pain and suicidal ideation in aging people living with HIV cured of hepatitis C: A multi‐center survey in France (ANRS CO13 HEPAVIH)

HIV Medicine, Volume 27, Issue 1, Page 106-119, January 2026.
Abstract Introduction Suicidal ideation (SI) is highly prevalent among people living with HIV (PWH) and those with chronic hepatitis C virus (HCV) infection. Individuals with long‐term HIV–HCV co‐infection face specific health challenges, including heightened physical pain.
Tangui Barré, Clémence Ramier, Camelia Protopopescu, Philippe Sogni, Karine Ory, Tounes Saidi, Sophie Abgrall, Sylvie Brégigeon‐Ronot, Patrizia Carrieri, Fabienne Marcellin, the ANRS CO13 HEPAVIH Study Group, D. Salmon, L. Wittkop, P. Sogni, P. Carrieri, B. Spire, K. Ory, P. Trimoulet, J. Izopet, L. Serfaty, L. Alric, M. A. Valantin, G. Pialoux, J. Chas, K. Barange, A. Naqvi, E. Rosenthal, A. Bicart‐See, O. Bouchaud, A. Gervais, C. Lascoux‐Combe, C. Goujard, K. Lacombe, C. Duvivier, D. Neau, P. Morlat, F. Bani‐Sadr, F. Boufassa, C. Solas, H. Fontaine, L. Piroth, A. Simon, D. Zucman, F. Boué, P. Miailhes, E. Billaud, H. Aumaître, D. Rey, G. Peytavin, O. Zaeglel‐Faucher, D. Salmon, R. Usubillaga, P. Sogni, B. Bienvenu, O. Launay‐Puybasset, C. Bernasconi, A. Brunet, B. Silbermann, H. Boucher, F. Rollot, O. Zak Dit Zbar, S. Pol, C. Le Jeunne‐Ballif, P. Louergue, H. Mehawej, L. Merine‐Belardi, F. Almasi, N. Benammar, L. Alagna, S. Chaussade, L. Guillevin, B. Terris, C. Norgeux, C. Katlama, M. A. Valantin, H. Stitou, F. Caby, L. Paris, L. Schneider, R. Tubiana, A. Simon, L. Roudière, O. Benveniste, G. Breton, M. Bonmarchand, M. Kirstetter, F. Charlotte, F. Capron, V. Calvez, V. Thibaut, C. Soulie, B. Abdi, S. Lambert‐Niclot, S. Bregigeon‐Ronot, O. Zaegel‐Faucher, H. Laroche, A. Menard, E. Gaubert‐Marechal, S. Thirée, C. Tamalet, C. Hakimi, J. C. Poluzzi, C. Solas, G. Pialoux, J. Chas, L. Lassel, T. Lyavanc, S. Le Nagat, L. Slama, A. Canesti, P. Callard, B. Bachour, L. Alric, N. Sigur, D. Bonnet, M. Guivarch, K. Barange, J. M. Peron, P. Massip, L. Cuzin, M. Obada, E. Bonnet, M. Alvarez, E. Labau, M. Mularczyk, N. Bourdoncle, J. Penecy, L. Porte, F. Busato, S. Khatibi, J. Bernard, J. Selves, F. Larroquette, V. Ferrer, J. Chiabrandon, E. Rosenthal, C. Pradier, V. Mondain, P. Dellamonica, P. M. Roger, S. Ferrando, C. Ceppi, A. Naqvi, V. Rio, B. Dunais, B. Prouvost, F. De Salvadore‐Guillouet, I. Prebost, J. Durant, P. Pugliese, J. F. Michels, M. C. Saint‐Paul, C. Partouche, K. Nerini, O. Bouchaud, S. Abgrall, I. Zamord, F. Bidegain, M. Azghay, C. Rouyer, M. Tatay, F. Mechai, A. Martin, Y. Baazia, V. Iwaka‐Bande, A. Gerber, A. Bicart‐See, D. Garipuy, M. J. Ferro‐Collados, F. Gaches, F. Abravanel, F. Larroquette, J. Chiabrandon, A. Gervais, Y. Yazdanpanah, D. Henin, M. Bertine, M. Onambele, G. Peytavin, C. Lascoux‐Combe, T. Kandel, C. Pintado, J. M. Molina, C. Gatey, S. Gallien, W. Rozenbaum, M. Lafaurie, A. L. Munier, D. Ponscarme, M. Previllon, N. Colin De Verdière, M. G. Tateo, P. Palmer, A. Gabassi, R. Amarsy, M. C. Mazeron, K. Lacombe, J. Krause, D. Wendum, D. Fofana, C. Goujard, Y. Quertainmont, E. Teicher, L. Escaut, O. Derradji, C. Pallier, F. Veillet, L. Mouna, O. Lortholary, C. Duvivier, C. Rouzaud, M. Shoai Tehrani, S. Boucly, G. Obenga, J. P. Viard, E. Gardiennet, A. Mélard, D. Neau, A. Ochoa, E. Blanchard, C. Cazanave, M. Dupon, H. Dutronc, F. Dauchy, H. Wille, P. Bioulac‐Sage, P. Trimoulet, P. Morlat, D. Lacoste, F. Bonnet, N. Bernard, M. Hessamfar, F. Paccalin, C. Martell, M. C. Pertusa, M. Vandenhende, P. Mercié, T. Pistone, M. C. Receveur, M. Méchain, P. Duffau, C. Rivoisy, I. Faure, D. Malvy, J. Roger‐Schmeltz, D. Dondia, P. Bioulac‐Sage, P. Trimoulet, J. L. Pellegrin, E. Lazzaro, P. Bioulac‐Sage, P. Trimoulet, D. Zucman, C. Majerholc, J. E. Kahn, S. Hilaire, M. Brollo, F. Boué, S. Abgrall, J. Polo Devoto, I. Kansau, V. Chambrin, C. Pignon, C. Gatey, M. Favier, C. Deback, C. Vauloup, C. Pailler, F. Veillet, L. Mouna, J. L. Lopez Zaragoza, Y. Lévy, J. D. Lelièvre, A. S. Lascaux, S. Scerra, M. Bouvier‐Alias, E. Billaud, F. Raffi, C. Allavena, V. Reliquet, D. Boutoille, C. Biron, M. Lefebvre, N. Hall, S. Bouchez, C. Bernaud, O. Mounoury, A. Rodallec, L. Le Guen, P. Miailhes, D. Peyramond, C. Chidiac, F. Ader, F. Biron, A. Boibieux, L. Cotte, T. Ferry, T. Perpoint, M. Amiri, F. Valour, J. Koffi, F. Zoulim, F. Bailly, P. Lack, M. Maynard, S. Radenne, C. Augustin‐Normand, E. Vassel, T. T. Le‐Thi, L. Piroth, P. Chavanet, M. Duong Van Huyen, M. Buisson, A. Waldner‐Combernoux, S. Mahy, A. Fillion, H. Aumaître, M. Jean, A. Eden, M. Medus, M. Saada, S. Gaba, F. Bani‐Sadr, D. Lambert, Y. Nguyen, J. L. Berger, C. Rouger, V. Brodard, D Rey, M. Partasani, C. Cheneau, M. Priester, C. Bernard‐Henry, E. de Mautort, A. Ertle, F. Jegou, A. Fush, P. Gantner, S. Fafi‐Kremer, L. Pinheiro, F. Roustand, I. Kmiec, L. Traore, S. Le Puil, M. G. Tateo, S. Benothame, C. Pomes, C. Louisin, M. Mole, A. Adda, P. Thibaut, S. Poirier, R. Ben Rayana, M. P. Pietri, V. Le Baut, Y. Cuvillier, M. Alabatanah, F. Barret, F. Juster, C. Chesnel, D. Beniken, M. Sebire‐Le Cam, R. Monard, F. Badji, S. Caldato, T. Rojas Rojas, A. S. Ritleng, A. Ivanova, L. Chalal, Z. Julia, M. Cavellec, C. Chevalier, E. Paredes‐Manyari, S. Breau, N. Boughdiri, P. Fischer, C. Charles, S. Ogoudjobi, C. Brochier, V. Thoirain‐Galvan, L. Traore, S. Chaussee, S. Benothmane, A. Sondro, O. Camou, V. Godard, M. Kai, C. Gilbert, M. Pailler, L. Merchadou, L. Esterle, K. Ory, S. Gillet‐Rousseau, C. Khan, L. Abbad, M. Chalouni, V. Conte, F. Marcellin, R. Knight, M. Mora, C. Protopopescu, P. Roux, T. Barré   +402 more
wiley   +1 more source

Short‐ and long‐term outcome of neonates with thrombocytopenia from maternal immune thrombocytopenia

British Journal of Haematology, EarlyView.
Boris Sorin, Billal Azzouguen, Valentine Loustau, Emmanuelle Boutin, Helene Henique, Julie Graveleau, Odile Souchaud‐Debouverie, Laurent Pascal, Laurent Gilardin, Nathalie Costedoat‐Chalumeau, Quitterie Reynaud, Brigitte Pan‐Petesch, Stéphane Cheze, Louis Terriou, Thibault Comont, Gaetan Sauvetre, Bruno Royer, Matthieu Mahévas, Marc Michel, Florence Canoui‐Poitrine, Bertrand Godeau, Stéphanie Guillet   +21 more
wiley   +1 more source

A novel RHCE*cE689C allele (p.Ser230Thr) in 2 donors of European ancestry with discrepant E antigen typing

Transfusion, EarlyView.
Gabriel Neto Braga, Christophe Tournamille, Cédric Le Maréchal, Raynald Flahaut, Céline Ricard, Aline Floch   +5 more
wiley   +1 more source

Outcomes after a second allogeneic haematopoietic stem cell transplant for relapsed paediatric acute myeloid leukaemia improved over time: A study from the EBMT Paediatric Diseases Working Party

British Journal of Haematology, Volume 207, Issue 6, Page 2496-2506, December 2025.
We retrospectively evaluated outcomes after a second haematopoietic stem cell transplantation (HSCT) performed between 2000 and 2022 for relapsed acute myeloid leukaemia in 345 children. We found that leukaemia‐free survival, overall survival, graft‐versus‐host disease (GVHD)/relapse‐free survival (GRFS) and relapse incidence improved over time, while ...
Nimrod Buchbinder, Victor Michel, Arnaud Dalissier, Katharina Kleinschmidt, Franco Locatelli, Alexei Maschan, Robert Wynn, Franca Fagioli, Marco Zecca, Charlotte Jubert, Birgitta Versluys, Petr Sedlacek, Ludmila Zubarovskaya, Marta Gonzalez Vicent, Alessandra Biffi, Gérard Michel, Oana Mirci‐Danicar, Wolfgang Holter, Marc Ansari, Jacques‐Emmanuel Galimard, Pascale Schneider, Mouad Abouqateb, Krzysztof Kałwak   +22 more
wiley   +1 more source