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169 results on '"Labeyrie, Céline"'

1. Clinical, paraclinical and outcome features of 166 patients with acute anti-GQ1b antibody syndrome

Author

Coly, Martin, Adams, David, Attarian, Shahram, Bouhour, Françoise, Camdessanché, Jean-Philippe, Carey, Guillaume, Cauquil, Cécile, Chanson, Jean-Baptiste, Chrétien, Pascale, Créange, Alain, Delmont, Emilien, Fargeot, Guillaume, Frachet, Simon, Gendre, Thierry, Kuntzer, Thierry, Labeyrie, Céline, Maisonobe, Thierry, Michaud, Maud, Moulin, Maximilien, Nicolas, Guillaume, Noury, Jean-Baptiste, Péréon, Yann, Puma, Angela, Sole, Guilhem, Taithe, Frédéric, Tard, Céline, Théaudin, Marie, Timsit, Serge, Venditti, Laura, and Echaniz-Laguna, Andoni

Published
2024
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2. Transthyretin amyloid cardiomyopathy in France: A cross-sectional multi-centre study (333 patients)

Author

Damy, Thibaud, Donal, Erwan, Lairez, Olivier, Eicher, Jean-Christophe, Karoubi, Mounira, Trochu, Jean-Noël, Inamo, Jocelyn, Habib, Gilbert, Roubille, François, Hagège, Albert, Morio, Flore, Cariou, Eve, Adda, Jérôme, Algalarrondo, Vincent, Coste, Agathe, Bartoli, Mathilde, Rudant, Jérémie, Salvi, Lara, Francou, Bruno, Guiochon-Mantel, Anne, Adams, David, Antoine, Jean-Christophe, Attarian, Shahram, Cintas, Pascal, Morales, Raul Juntas, Lagrange, Emmeline, Magy, Laurent, Mallaret, Martial, Péréon, Yann, Petiot, Philippe, Cauquil, Cécile, Labeyrie, Céline, Echaniz-Laguna, Andoni, Sole, Guilhem, Tard, Céline, Oghina, Silvia, Charron, Philippe, and Slama, Michel

Published
2024
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4. Current practices and access to cardiac bone scans for the detection of transthyretin cardiac amyloidosis based on the results of a large national electronic survey

Author

Hyafil, Fabien, primary, Oghina, Silvia, primary, Legallois, Damien, primary, Amara, Walid, primary, Andres, Emmanuel, primary, Bardin, Thomas, primary, Fournier, Pauline, primary, Guignard, Sandra, primary, Labeyrie, Céline, primary, Toulza, Olivier, primary, Tresorier, Romain, primary, Piriou, Nicolas, primary, SABOURET, Pierre, primary, Stanbury, Trevor, primary, Canali, Giorgia, primary, DUBOIS, Margaux, primary, Noirot-Cosson, Charlotte, primary, Rudant, Jeremie, primary, Bouquillon, Benoit, primary, Sauvage, Cedric, primary, Charron, Philippe, primary, and Damy, Thibaud, primary

Published
2024
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6. Evaluating the current physicians’ knowledge and patients’ pathways for diagnosing transthyretin cardiac amyloidosis (ATTR-CM) in France: An extensive survey of diverse medical specialists.

Author

Oghina, Silvia, primary, Legallois, Damien, primary, Hyafil, Fabien, primary, Amara, Walid, primary, Andres, Emmanuel, primary, Bardin, Thomas, primary, Fournier, Pauline, primary, Guignard, Sandra, primary, Labeyrie, Céline, primary, Piriou, Nicolas, primary, SABOURET, Pierre, primary, Toulza, Olivier, primary, Tresorier, Romain, primary, Stanbury, Trevor, primary, Canali, Giorgia, primary, DUBOIS, Margaux, primary, Noirot-Cosson, Charlotte, primary, Rudant, Jeremie, primary, Bouquillon, Benoit, primary, Sauvage, Cedric, primary, Charron, Philippe, primary, and Damy, Thibaud, primary

Published
2024
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7. Nonamyloidogenic TTR gene variants c.76G>A and c.337‐18G>C are not associated with idiopathic small‐fiber neuropathy.

Author

Konecki, Céline, Francou, Bruno, Chappell, Kenneth, Augey, Lucie, Beaudonnet, Guillemette, Cauquil, Cécile, Dimitri‐Boulos, Dalia, Not, Adeline, Adam, Clovis, Poinsignon, Vianney, Verstuyft, Céline, Adams, David, Echaniz‐Laguna, Andoni, and Labeyrie, Céline

Subjects
GENETIC variation, DATABASES, IDIOPATHIC diseases, DYSAUTONOMIA, TRANSTHYRETIN
Abstract

Background and Purpose: Small‐fiber neuropathy (SFN) affects only unmyelinated and thin myelinated fibers. It may be caused by amyloidogenic mutations of the transthyretin (TTR) gene, but not all TTR gene variants are pathogenic. The nonamyloidogenic c.76G>A (rs1800458) and c.337‐18G>C (rs36204272) variants of TTR were recently reported to be associated with SFN. We investigated this putative association by analyzing TTR gene sequencing data retrospectively for two cohorts of patients, one with SFN and a control group. Methods: In this retrospective single‐center study, we analyzed the frequency of the c.76G>A and c.337‐18G>C TTR gene variants in a cohort of patients meeting a strict definition of SFN, with or without dysautonomia, a control cohort of patients investigated for nonneurological conditions, and the gnomAD international database. Results: We included 55 SFN patients in this study, 17 of whom had dysautonomia. The allelic frequencies of the two variants in our cohort of 55 SFN patients were 7.27% for c.76G>A TTR and 5.25% for c.337‐18G>C. The frequencies of both variants were statistically similar in the 337 control patients and the gnomAD database. Conclusions: The c.76G>A and c.337‐18G>C TTR gene variants are not associated with SFN. [ABSTRACT FROM AUTHOR]

Published
2024
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8. Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study

Author

Vita, Giuseppe, Rizzo, Vincenzo, Russo, Massimo, Mazzeo, Anna, Gentile, Luca, Berk, John L, Brueckner, Caitlin, Lazzari, Victoria, Wiesman, Janice, DeLong, Douglas, Victory, Jennifer, Dalton, James, May, John, Gilmore, Catherine, Attarian, Shahram, Diallo, Saran, Delmont, Emilien, Pouget, Jean, Verschueren, Annie, Grapperon, Aude-Marie, Campana-Salort, Emmanuelle, Conceição, Isabel M, Lopes, Ana, Lamas, Filipa, Neves, Carlos, Castro, Jose, Pereira, Pedro, Castro, Isabel, Franco, Ana, Santos, Miguel Oliveira, de Azevedo Coutinho, Conceição, Falcao de Campos, Catarina, Coelho, Teresa, Hipólito Reis, Antonio, Correia, Nuno, Perez, Javier M, Martins da Silva, Ana, Alves, Cristina, Cardoso, Marcio, Valdrez, Katia, Monte, Julia R, Pessoa, Bernardete, Guimaraes, Nadia, Freitas, Monica, Ramalho, Joana, Ferreira, Natalia, Kuzume, Daisuke, Tard, Celine, Waucquier, Nawal, Rougeaux, Isabelle, Brice, Sylvie, Kasprzyk, Emmanuelle, Elrezzi, Elise, Meguig, Sayah, Hachulla, Eric, Gauvain, Clement, Migaud-Chervy, Maria-Claire, Deplanque, Dominique, Jozefowicz, Elsa, Lebellec, Loic, Adams, David, Balaya-Gouraya, Line, Jehan Lacour, Nathalie, Bournane, Halima, Martin, Nathalie, Elabed, Mongia, Sacko, Niamey, Boubrit, Yasmine, Gaouar, Amina, Rakotondratafika, Fetra, Théaudin-Saliou, Marie, Cauquil-Michon, Cécile, Labeyrie, Celine, Not, Adeline, Al-Salameh, Abdallah, Lecoq, Anne-Lise, Stephant, Maeva, Echaniz-Laguna, Andoni, Becquemont, Laurent, Beaudonnet, Guillemette, Algalarrondo, Vincent, Eliahou, Ludivine, Slama, Michel S, Rousseau, Antoine, Signate, Aissatou, Berthelot, Emeline, Inamo, Jocelyn, Planté-Bordeneuve, Violaine, Vervoitte, Laetitia, Focseneanu, Cecile, Gendre, Thierry, Arrouasse, Raphaele, Ayache, Samar S., Ernande, Laura, Le Corvoisier, Philippe, Salhi, Hayet, Choumert, Ariane, Ehinger, Vincent, Ruiz, Julie, Charlin, Cyril, Megelin, Thomas, Brannagan III, Thomas H, Fayerman, Raisy, Kim, Arreum, Paras, Allan, Gonzalez, Leidy J, Tsang, Steven, Wajnsztajn, Fernanda, Shije, Jeffrey, Ulane, Christina, Kleyman, Inna, Weimer, Louis, Cioroiu, Comana, Lambrianides, Sakis, Abu-Manneh, Rana, Zamba-Papanicolaou, Eleni, Agathangelou, Petros, Leonidou, Eleni, Tada, Satoshi, Fujita, Akemi, Nagai, Masahiro, Ando, Rina, Hosokawa, Yuko, Yamanishi, Yuki, Overcash, J. Scott, Giardino, Elena, Boyer, Leslie, Dang, Lien, Le, An, Nguyen, Tyler, Giang, Lien, Sellers, Peter, Tran, Leyla, Truong, Nghi, Vinas, Maita, Hrkman, Nicole, Miller, Sarah, Nguyen, David, Smith, Ashley, Pu, Helen, Li, Steve, Vuong, Thao, Dioso, Holly, Green, Sinikka, Lee, Kia, Chu, Hanh, Waters, Michael, Coskun, Derya J, Zepeda, Karla A, O'Riordan, William, Obici, Laura, Cortese, Andrea, Lozza, Alessandro, Merlini, Giampaolo, Rosti, Vittorio, Sabatelli, Mario, Bisogni, Giulia, Bernardo, Daniela, Luigetti, Marco, Di Paolantonio, Andrea, Guglielmino, Valeria, Romano, Angela, Nienhuis, Hans, Bulthuis-Kuiper, Janita, Kristen, Arnt V, Gerk, Olga, Ulbricht, Hannah, Taylor, Lenka, Meyle, Eva, Kleinschmidt, Natalia, Meyrath, David, Noe-Schwenn, Simone, Meng, Ulrike, Bauer, Ralf, aus dem Siepen, Fabian, Hein, Selina, Takahashi, Tetsuya, Oshita, Tomohiko, Koujin, Yoko, Neshige, Shuichiro, Nezu, Tomohisa, Segawa, Akiko, Ueno, Hiroki, Morino, Hiroyuki, Campistol, Josep M, Rodas Marin, Lida Maria, Blasco Pelicano, Josep Miquel, Dávila, Lucía Galán, Palacios, Marta, Pytel Cordoba, Vanesa, Guerrero Sola, Antonio, Horga, Alejandro, García Feijoo, Julián, Perez de Isla, Leopoldo, Marques Júnior, Wilson, Moscardini, Mariana, Litcanov, Debora Cristina, Viera Lima, Ana Flavia, Rodrigues, Leonardo, Marques Coutinho, Barbara, Moreira, Carolina Lavigne, Daccach Marques, Vanessa, Munoz Beamud, Francisco, Gragera Martínez, Álvaro, Borrachero, Cristina, Losada López, Inés Asunción, Cisneros Barroso, Eugenia, Rodríguez Rodríguez, Adrián, Sanz, Monica, Rigo Oliver, Elena, González Moreno, Juan, Gamez Martinez, Jose M, Descals, Cristina, Uson, Mercedes, Jose Vega, Francisco, Figuerola, Antoni, Montala, Carles, Waddington-Cruz, Márcia, Dias da Silva, Moises, Gervais de Santa Rosa, Renata, Pinto, Luiz Felipe, Pinto, Marcus Vinicius, Cardoso Berensztejn, Amanda, Barroso, Fabio, Lautre, Andrea, Orellana, Lucas G, González-Duarte Briseño, Maria Alejandra, Cárdenas-Soto, Karla, Jiménez López, Brenda Poled, Pérez-Castañeda, Sandra Lorena, Cantú Brito, Carlos Gerardo, Rivera de la Parra, David, Hernandez Reyes, Jose Pablo, del Mar Saniger Alba, Maria, Criollo Mora, Elia, Parman, Yesim, Rezzan, Kus Jülide, Sahin, Erdi, Serbest, Nail G, Durmus, Hacer, Cakar, Arman, Tugal Tutkun, Nuriye Ilknur, Karamursel, Sacit, Elitok, Ali, Sirin Inan, Nermin G, Altinkurt, Emre, Polydefkis, Michael, Ye, Jing, Allen, Adriane C, Chaudhry, Vinay, Jarrett, Raquel, Bressler, Neil, Burks, Kathleen L, Liu, Qingfeng, Khoshnoodi, Mohammad, Judge, Daniel P, Vista, Geno, Shah, Syed Mahmood, Hamaguchi, Hirotoshi, Oda, Junko, Fukase, Emi, Taniguchi, Ikuko, Oda, Tetsuya, Endo, Hironobu, Shimomura, Masahiro, Katanazaka, Kimitaka, Koto, Shusuke, Nakano, Takahiro, Scheid, Christof, Zueiter, Andreas, Pester, Lars, Walter, Doreen, Özdemir, Betül, Frenzel, Lukas F, Holtick, Udo, Oh, Jeeyoung, Kim, Hee Jin, Shin, Hyun Jin, Choi, Kyomin, Yamashita, Taro, Ueda, Mitsuharu, Masuda, Teruaki, Misumi, Yohei, Ueda, Akihiko, Nakahara, Keiichi, Yorita, Akiko, Tsuruhisa, Seiko, Taniwaki, Takayuki, Harada, Masaya, Moritaka, Taiga, Sakurada, Naonori, Mauricio, Elizabeth A, Baskin, Amber, Dimberg, Elliot, Dispenzieri, Angela, Fonder, Amie, Hobbs, Miriam, Russell, Stephen J, Dyck, Peter, Gonsalves, Wilson, Leung, Nelson, Witzig, Thomas E, Zeldenrust, Steven R, Hwa, Lisa, Kapoor, Prashant, Kumar, Shaji K, Lin, Yi, Lust, John A, Rajkumar, Vincent S, Dingli, David, Gertz, Morie A, Go, Ronald, Hayman, Suzanne R, Dalia, Samir, Carrillo, Esmeralda, Gorevic, Peter, Mason, Garnette, Chao, Chi-Chao, Lee, Ming-Jen, Su, Jen-Jen, Hsieh, Sung-Tsang, Tsai, Li-Kai, Yeh, Shin-Joe, Yang, Chih-Chao, Ajroud-Driss, Senda Ajroud-Driss, Casey, Patricia, Joslin, Benjamin C, Freimer, Miriam, Sankey, Alison, Kenepp, Amanda, Heintzman, Sarah, LoRusso, Samantha, Hokezu, Youichi, Kim, Byoung-Joon, Kim, JuHyeon, Lee, Ga Yeon, Cho, Eun Bin, Jeon, Eun-Seok, Min, Ju-Hong, Seok, Jin Myoung, Lee, Hye Lim, Park, Jae Hong, Sekijima, Yoshiki, Miyazawa, Chinatsu, Kato, Nagaaki, Kishida, Dai, Hineno, Akiyo, Kodaira, Minori, Yoshinaga, Tsuneaki, Miyahara, Teruyoshi, Imai, Akira, Matsumoto, Kazuhiko, Lin, Kon-Ping, Lee, Yi-Chung, Wixner, Jonas, Falk, Malin, Pilebro, Bjorn, Suhr, Ole, Lindqvist, Per, Soderberg, Karin, Pedrosa-Domellöf, Fatima, Anan, Intissar, Nordh, Erik, Tournev, Ivaylo, Zhelyazkova-Glaveeva, Sashka, Cherneva, Zheyna, Sarafov, Staiko, Chamova, Teodora, Cherninkova-Gopina, Sylvia, Schmidt, Hartmut H, Friebel, Frauke, Zibert, Andree, Mihailovic, Natasa, Schubert, Friederike, Vorona, Elena, Lahme, Larissa, Huesing-Kabar, Anna, Schilling, Matthias, Kabar, Iyad, Gillmore, Julian D, Martinez-Naharro, Ana, Chacko, Liza, Cohen, Oliver, Law, Steven, Rezk, Tamer, Lachmann, Helen J, Quan, Dianna, Blume, Brianna, Dixon, Stacy, Low, Soon Chai, Chan, Soo Looi, Lim, He Eng Li, Goh, Khean Jin, Mezei, Michelle M, Kraus, Deborah, Jack, Kristin, Wade, N. Kevin, Lopate, Glenn, Zwijack, Brittany, Florence, Julaine, Sommerville, R. Brian, Stewart, Graeme, Ryder, Julie, Mekhael, Linda, Taylor, Mark, Suan, Daniel, Wells, Karen, Stone, Paula, Itoya, Amenze, Owusu-Sekyere, Mercy, Thai, Desmond, Chahine, Ilonah, Pedrosa, Salve, Do, Thi Hoa (Therese), González-Duarte, Alejandra, Kyriakides, Theodoros, Ajroud-Driss, Senda, Mauricio, Elizabeth, Brannagan, Thomas H, III, Aldinc, Emre, Wang, Jing Jing, White, Matthew T, Vest, John, Berber, Erhan, and Sweetser, Marianne T

Published
2021
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11. Frequent detection of IFN-gamma-producing memory effector and effector T cells in patients with progressive multifocal leukoencephalopathy.

Author

de Goër de Herve, Marie-Ghislaine, Dekeyser, Manon, Hendel-Chavez, Houria, Maillart, Elisabeth, Labeyrie, Céline, Adams, David, Moreau, Thibault, Lubetzki, Catherine, Papeix, Caroline, Stankoff, Bruno, Gasnault, Jacques, and Taoufik, Yassine

Subjects
PROGRESSIVE multifocal leukoencephalopathy, IMMUNOLOGIC memory, T cells, CENTRAL nervous system, DEMYELINATION, JOHN Cunningham virus
Abstract

Introduction: Progressive Multifocal Leukoencephalopathy (PML) is a rare and deadly demyelinating disease caused by JC virus (JCV) replication in the central nervous system. PML occurs exclusively in patients with severe underlying immune deficiencies, including AIDS and hematological malignancies. PML has also emerged as a significant threat to patients on potent new immunosuppressive biologics, including natalizumab in multiple sclerosis. Methods: Here, we developed an IFN-g release assay (IGRA) that mainly detects JCV-specific effector memory T cells and effectors T cells in the blood. Results: This assay was frequently positive in patients with active PML (with a positive JCV PCR in CSF) of various underlying immunosuppression causes (84% sensitivity). Only 3% of healthy donors had a positive response (97% specificity). The frequency of positivity also increased in multiple sclerosis patients according to the time on natalizumab (up to 36% in patients treated for more than 48 months, who are considered at a higher risk of PML). Discussion: The results show this assay's frequent or increased positivity in patients with PML or an increased risk of PML, respectively. The assay may help to stratify the risk of PML. [ABSTRACT FROM AUTHOR]

Published
2024
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12. Detailed clinical, physiological and pathological phenotyping can impact access to disease-modifying treatments in ATTR carriers.

Author

Beauvais, Diane, Labeyrie, Céline, Cauquil, Cécile, Francou, Bruno, Eliahou, Ludivine, Not, Adeline, Echaniz-Laguna, Andoni, Adam, Clovis, Slama, Michel S., Benmalek, Anouar, Leonardi, Luca, Rouzet, François, Adams, David, Algalarrondo, Vincent, and Beaudonnet, Guillemette

Subjects
CARDIAC amyloidosis, MAGNETIC resonance neurography, CARDIAC magnetic resonance imaging, GLOBAL longitudinal strain
Published
2024
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13. Electrophysiological features of chronic inflammatory demyelinating polyradiculoneuropathy associated with IgG4 antibodies targeting neurofascin 155 or contactin 1 glycoproteins

Author

Kouton, Ludivine, Boucraut, José, Devaux, Jérome, Rajabally, Yusuf A., Adams, David, Antoine, Jean Christophe, Bourdain, Frédéric, Brodovitch, Alexandre, Camdessanché, Jean-Philippe, Cauquil, Cécile, Ciron, Jonathan, Dubard, Thierry, Echaniz-Laguna, Andoni, Grapperon, Aude-Marie, Juntas-Morales, Raul, Kremer, Laurent, Kuntzer, Thierry, Labeyrie, Céline, Lanfranco, Luca, Léger, Jean-Marc, Maisonobe, Thierry, Mavroudakis, Nicolas, Mecharles-Darrigol, Sylvie, Merle, Philippe, Noury, Jean-Baptiste, Rouaud, Violaine, Tard, Céline, Théaudin, Marie, Vallat, Jean-Michel, Viala, Karine, Attarian, Shahram, and Delmont, Emilien

Published
2020
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14. Indications de l’autogreffe de cellules souches hématopoïétiques dans la polyradiculonévrite inflammatoire démyélinisante chronique : recommandations de la Société francophone de greffe de moelle et de thérapie cellulaire (SFGM-TC)

Author

Puyade, Mathieu, Labeyrie, Céline, Badoglio, Manuela, Cintas, Pascal, Guenounou, Sarah, Lansiaux, Pauline, Marjanovic, Zora, Nicolas, Guillaume, Pomies, Amélie, Terriou, Louis, Torregrosa Diaz, Jose-Miguel, Baron, Clément, Castilla Llorente, Cristina, Yakoub-Agha, Ibrahim, and Farge, Dominique

Published
2020
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15. Antibodies against the node of Ranvier: a real-life evaluation of incidence, clinical features and response to treatment based on a prospective analysis of 1500 sera

Author

Delmont, Emilien, Brodovitch, Alexandre, Kouton, Ludivine, Allou, Thibaut, Beltran, Stéphane, Brisset, Marion, Camdessanché, Jean Philippe, Cauquil, Cécile, Cirion, Jonathan, Dubard, Thierry, Echaniz-Laguna, Andoni, Grapperon, Aude-Marie, Jauffret, Joëlle, Juntas-Morales, Raul, Kremer, Laurent Daniel, Kuntzer, Thierry, Labeyrie, Céline, Lanfranco, Lucas, Maisonobe, Thierry, Mavroudakis, Nicolas, Mecharles-Darrigol, Sylvie, Nicolas, Guillaume, Noury, Jean-Baptiste, Perie, Maud, Rajabally, Yusuf A., Remiche, Gauthier, Rouaud, Violaine, Tard, Céline, Salort-Campana, Emmanuelle, Verschueren, Annie, Viala, Karine, Wang, Adrien, Attarian, Shahram, and Boucraut, José

Published
2020
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16. Acute Clinical Events Identified as Relapses With Stable Magnetic Resonance Imaging in Multiple Sclerosis

Author

Gavoille, Antoine, Rollot, Fabien, Casey, Romain, Kerbrat, Anne, Le Page, Emmanuelle, Bigaut, Kevin, Mathey, Guillaume, Michel, Laure, Ciron, Jonathan, Ruet, Aurelie, Maillart, Elisabeth, Labauge, Pierre, Zephir, Hélène, Papeix, Caroline, Defer, Gilles, Lebrun-Frenay, Christine, Moreau, Thibault, Berger, Eric, Stankoff, Bruno, Clavelou, Pierre, Thouvenot, Eric, Heinzlef, Olivier, Pelletier, Jean, Al-Khedr, Abdullatif, Casez, Olivier, Bourre, Bertrand, Cabre, Philippe, Wahab, Abir, Magy, Laurent, Camdessanché, Jean-Philippe, Doghri, Inès, Moulin, Solène, Ben-Nasr, Haifa, Labeyrie, Céline, Hankiewicz, Karolina, Neau, Jean-Philippe, Pottier, Corinne, Nifle, Chantal, Manchon, Eric, Lapergue, Bertrand, Wiertlewski, Sandrine, De Sèze, Jérôme, Vukusic, Sandra, and Laplaud, David Axel

Abstract

IMPORTANCE: Understanding the association between clinically defined relapses and radiological activity in multiple sclerosis (MS) is essential for patient treatment and therapeutic development. OBJECTIVE: To investigate clinical events identified as relapses but not associated with new T2 lesions or gadolinium-enhanced T1 lesions on brain and spinal cord magnetic resonance imaging (MRI). DESIGN, SETTING, AND PARTICIPANTS: This multicenter observational cohort study was conducted between January 2015 and June 2023. Data were extracted on June 8, 2023, from the French MS registry. All clinical events reported as relapses in patients with relapsing-remitting MS were included if brain and spinal cord MRI was performed within 12 and 24 months before the event, respectively, and 50 days thereafter with gadolinium injection. EXPOSURES: Events were classified as relapses with active MRI (RAM) if a new T2 lesion or gadolinium-enhanced T1 lesion appeared on brain or spinal cord MRI or as acute clinical events with stable MRI (ACES) otherwise. MAIN OUTCOMES AND MEASURES: Factors associated with ACES were investigated; patients with ACES and RAM were compared regarding Expanded Disability Status Scale (EDSS) course, relapse rate, confirmed disability accrual (CDA), relapse-associated worsening (RAW), progression independent of relapse activity (PIRA), and transition to secondary progressive (SP) MS, and ACES and RAM rates under each disease-modifying therapy (DMT) were estimated. RESULTS: Among 31 885 clinical events, 637 in 608 patients (493 [77.4%] female; mean [SD] age, 35.8 [10.7] years) were included. ACES accounted for 166 (26.1%) events and were more likely in patients receiving highly effective DMTs, those with longer disease duration (odds ratio [OR], 1.04; 95% CI, 1.01-1.07), or those presenting with fatigue (OR, 2.14; 95% CI, 1.15-3.96). ACES were associated with significant EDSS score increases, lower than those found for RAM. Before the index event, patients with ACES experienced significantly higher rates of relapse (relative rate [RR], 1.21; 95% CI, 1.01-1.46), CDA (hazard ratio [HR], 1.54; 95% CI, 1.13-2.11), and RAW (HR, 1.72; 95% CI, 1.20-2.45). Patients with ACES were at significantly greater risk of SP transition (HR, 2.58; 95% CI, 1.02-6.51). Although RAM rate decreased with DMTs according to their expected efficacy, ACES rate was stable across DMTs. CONCLUSIONS AND RELEVANCE: The findings in this study introduce the concept of ACES in MS, which accounted for one-fourth of clinical events identified as relapses.

Published
2024
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17. Hereditary transthyretin amyloidosis in middle-aged and elderly patients with idiopathic polyneuropathy: a nationwide prospective study.

Author

Fargeot, Guillaume, Echaniz-Laguna, Andoni, Labeyrie, Céline, Svahn, Juliette, Camdessanché, Jean-Philippe, Cintas, Pascal, Chanson, Jean-Baptiste, Esselin, Florence, Piedvache, Céline, Verstuyft, Céline, Genestet, Steeve, Lagrange, Emmeline, Magy, Laurent, Péréon, Yann, Sacconi, Sabrina, Signate, Aissatou, Nadaj-Pakleza, Aleksandra, Taithe, Frédéric, Viala, Karine, and Tard, Céline

Subjects
OLDER patients, CARDIAC amyloidosis, POLYNEUROPATHIES, TRANSTHYRETIN, NERVE conduction studies, AMYLOIDOSIS
Abstract

Hereditary transthyretin amyloidosis (ATTRv) is an adult-onset autosomal dominant disease resulting from TTR gene pathogenic variants. ATTRv often presents as a progressive polyneuropathy, and effective ATTRv treatments are available. In this 5 year-long (2017–2021) nationwide prospective study, we systematically analysed the TTR gene in French patients with age >50 years with a progressive idiopathic polyneuropathy. 553 patients (70% males) with a mean age of 70 years were included. A TTR gene pathogenic variant was found in 15 patients (2.7%), including the Val30Met TTR variation in 10 cases. In comparison with patients with no TTR gene pathogenic variants (n = 538), patients with TTR pathogenic variants more often presented with orthostatic hypotension (53 vs. 21%, p =.007), significant weight loss (33 vs 11%, p =.024) and rapidly deteriorating nerve conduction studies (26 vs. 8%, p =.03). ATTRv diagnosis led to amyloid cardiomyopathy diagnosis in 11 cases, ATTRv specific treatment in all cases and identification of 15 additional ATTRv cases among relatives. In this nationwide prospective study, we found ATTRv in 2.7% of patients with age >50 years with a progressive polyneuropathy. These results are highly important for the early identification of patients in need of disease-modifying treatments. [ABSTRACT FROM AUTHOR]

Published
2024
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18. Detailed clinical, physiological and pathological phenotyping can impact access to disease-modifying treatments in ATTR carriers

Author

Beauvais, Diane, primary, Labeyrie, Céline, additional, Cauquil, Cécile, additional, Francou, Bruno, additional, Eliahou, Ludivine, additional, Not, Adeline, additional, Echaniz-Laguna, Andoni, additional, Adam, Clovis, additional, Slama, Michel S, additional, Benmalek, Anouar, additional, Leonardi, Luca, additional, Rouzet, François, additional, Adams, David, additional, Algalarrondo, Vincent, additional, and Beaudonnet, Guillemette, additional

Published
2023
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19. High-Efficacy Therapy Discontinuation vs Continuation in Patients 50 Years and Older With Nonactive MS

Author

Jouvenot, Guillaume, Courbon, Guilhem, Lefort, Mathilde, Rollot, Fabien, Casey, Romain, Le Page, Emmanuelle, Michel, Laure, Edan, Gilles, de Seze, Jérome, Kremer, Laurent, Bigaut, Kevin, Vukusic, Sandra, Mathey, Guillaume, Ciron, Jonathan, Ruet, Aurélie, Maillart, Elisabeth, Labauge, Pierre, Zephir, Hélène, Papeix, Caroline, Defer, Gilles, Lebrun-Frenay, Christine, Moreau, Thibault, Laplaud, David Axel, Berger, Eric, Stankoff, Bruno, Clavelou, Pierre, Thouvenot, Eric, Heinzlef, Olivier, Pelletier, Jean, Al-Khedr, Abdullatif, Casez, Olivier, Bourre, Bertrand, Cabre, Philippe, Wahab, Abir, Magy, Laurent, Camdessanché, Jean-Philippe, Doghri, Ines, Moulin, Solène, Ben-Nasr, Haifa, Labeyrie, Céline, Hankiewicz, Karolina, Neau, Jean-Philippe, Pottier, Corinne, Nifle, Chantal, Collongues, Nicolas, and Kerbrat, Anne

Abstract

IMPORTANCE: A recent randomized clinical trial concluded that discontinuing medium-efficacy therapy might be a reasonable option for older patients with nonactive multiple sclerosis (MS), but there is a lack of data on discontinuing high-efficacy therapy (HET). In younger patients, the discontinuation of natalizumab and fingolimod is associated with a risk of rebound of disease activity. OBJECTIVE: To determine whether discontinuing HET in patients 50 years and older with nonactive MS is associated with an increased risk of relapse compared with continuing HET. DESIGN, SETTING, AND PARTICIPANTS: This observational cohort study used data from 38 referral centers from the French MS registry (Observatoire Français de la Sclérose en Plaques [OFSEP] database). Among 84704 patients in the database, data were extracted for 1857 patients 50 years and older with relapsing-remitting MS treated by HET and with no relapse or magnetic resonance imaging activity for at least 2 years. After verification of the medical records, 1620 patients were classified as having discontinued HET or having remained taking treatment and were matched 1:1 using a dynamic propensity score (including age, sex, disease phenotype, disability, treatment of interest, and time since last inflammatory activity). Patients were included from February 2008 to November 2021, with a mean (SD) follow-up of 5.1 (2.9) years. Data were extracted in June 2022. EXPOSURES: Natalizumab, fingolimod, rituximab, and ocrelizumab. MAIN OUTCOMES AND MEASURES: Time to first relapse. RESULTS: Of 1620 included patients, 1175 (72.5%) were female, and the mean (SD) age was 54.7 (4.8) years. Among the 1452 in the HET continuation group and 168 in the HET discontinuation group, 154 patients in each group were matched using propensity scores (mean [SD] age, 57.7 [5.5] years; mean [SD] delay since the last inflammatory activity, 5.6 [3.8] years; mean [SD] follow-up duration after propensity score matching, 2.5 [2.1] years). Time to first relapse was significantly reduced in the HET discontinuation group compared with the HET continuation group (hazard ratio, 4.1; 95% CI, 2.0-8.5; P < .001) but differed between HETs, with a hazard ratio of 7.2 (95% CI, 2.1-24.5; P = .001) for natalizumab, 4.5 (95% CI, 1.3-15.5; P = .02) for fingolimod, and 1.1 (95% CI, 0.3-4.8; P = .85) for anti-CD20 therapy. CONCLUSION AND RELEVANCE: As in younger patients, in patients 50 years and older with nonactive MS, the risk of relapse increased significantly after stopping HETs that impact immune cell trafficking (natalizumab and fingolimod). There was no significant increase in risk after stopping HETs that deplete B-cells (anti-CD20 therapy). This result may inform decisions about stopping HETs in clinical practice.

Published
2024
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21. Comparative effectiveness of teriflunomide vs dimethyl fumarate in multiple sclerosis

Author

Laplaud, David-Axel, Casey, Romain, Barbin, Laetitia, Debouverie, Marc, De Sèze, Jérôme, Brassat, David, Wiertlewski, Sandrine, Brochet, Bruno, Pelletier, Jean, Vermersch, Patrick, Edan, Gilles, Lebrun-Frenay, Christine, Clavelou, Pierre, Thouvenot, Eric, Camdessanché, Jean-Philippe, Tourbah, Ayman, Stankoff, Bruno, Al Khedr, Abdullatif, Cabre, Philippe, Lubetzki, Catherine, Papeix, Caroline, Berger, Eric, Heinzlef, Olivier, Debroucker, Thomas, Moreau, Thibault, Gout, Olivier, Bourre, Bertrand, Wahab, Abir, Labauge, Pierre, Magy, Laurent, Defer, Gilles, Guennoc, Anne-Marie, Maubeuge, Nicolas, Labeyrie, Céline, Patry, Ivania, Nifle, Chantal, Casez, Olivier, Michel, Laure, Rollot, Fabien, Leray, Emmanuelle, Vukusic, Sandra, Foucher, Yohann, Fontaine, B., Marignier, R., Durand-Dubief, F., Mathey, G., Le Page, E., Peaureaux-Averseng, D., Ouallet, J.C., Ruet, A., Collongues, N., Hautecoeur, P., Zephir, H., Maillard, E., Cohen, M., Derache, N., Branger, P., Ayrignac, X., Carra-Dalliere, C., Fromont, A., Chamard-Witkowski, L., Taithe, F., Moisset, X., Audoin, B., Rico-Lamy, A., Castelnovo, G., Giannesini, C., Fagniez, O., Bensa, C., Gueguen, A., Kasonde, I. Tabellah, De Vilmarest, A., Montcuquet, A., Vaillants, M., Beltran, S., Creange, A., Ayache, S., Abdellaoui, M., Pottier, C., Slesari, I., Deburghraeve, V., Neau, J.P., Servan, J., Pico, F., Henry, C., and Hankiewicz, K.

Published
2019
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23. Anti‐disialosyl‐immunoglobulin M chronic autoimmune neuropathies: a nationwide multicenter retrospective study

Author

Peillet, Claire, primary, Adams, David, additional, Attarian, Shahram, additional, Bouhour, Françoise, additional, Cauquil, Cécile, additional, Cassereau, Julien, additional, Chanson, Jean‐Baptiste, additional, Cintas, Pascal, additional, Creange, Alain, additional, Delmont, Emilien, additional, Fargeot, Guillaume, additional, Genestet, Steeve, additional, Gueguen, Antoine, additional, Kaminsky, Anne Laure, additional, Kuntzer, Thierry, additional, Labeyrie, Céline, additional, Michaud, Maud, additional, Pereon, Yann, additional, Puma, Angela, additional, Viala, Karine, additional, Chretien, Pascale, additional, Adam, Clovis, additional, and Echaniz‐Laguna, Andoni, additional

Published
2022
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26. Skin amyloid deposits and nerve fiber loss as markers of neuropathy onset and progression in hereditary transthyretin amyloidosis

Author

Leonardi, Luca, primary, Adam, Clovis, additional, Beaudonnet, Guillemette, additional, Beauvais, Diane, additional, Cauquil, Cécile, additional, Not, Adeline, additional, Morassi, Olivier, additional, Benmalek, Anouar, additional, Trassard, Olivier, additional, Echaniz‐Laguna, Andoni, additional, Adams, David, additional, and Labeyrie, Céline, additional

Published
2022
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27. Amyloidosis from the patient perspective: the French daily impact of amyloidosis study

Author

Damy, Thibaud, primary, Adams, David, additional, Bridoux, Frank, additional, Grateau, Gilles, additional, Planté-Bordeneuve, Violaine, additional, Ghiron, Yves, additional, Farrugia, Agnès, additional, Pelcot, Françoise, additional, Taieb, Charles, additional, Labeyrie, Céline, additional, Jaccard, Arnaud, additional, and Georgin-Lavialle, Sophie, additional

Published
2022
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28. Neurological complications induced by immune checkpoint inhibitors: a comprehensive descriptive case-series unravelling high risk of long-term sequelae

Author

Plaçais, Léo, primary, Michot, Jean-Marie, additional, Champiat, Stéphane, additional, Romano-Martin, Patricia, additional, Baldini, Capucine, additional, Joao, Maria Silva, additional, Marabelle, Aurélien, additional, Voisin, Anne-Laure, additional, Not, Adeline, additional, Labeyrie, Céline, additional, Beaudonnet, Guillemette, additional, Laparra, Ariane, additional, Maria, Alexandre T J, additional, Masseau, Agathe, additional, Dehette, Stéphanie, additional, Deleporte, Amélie, additional, Echaniz-Laguna, Andoni, additional, Denier, Christian, additional, Adams, David, additional, Lambotte, Olivier, additional, Noel, Nicolas, additional, and Cauquil, Cécile, additional

Published
2021
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29. The effectiveness of natalizumab vs fingolimod–A comparison of international registry studies

Author

Andersen, Johanna B, primary, Sharmin, Sifat, additional, Lefort, Mathilde, additional, Koch-Henriksen, Nils, additional, Sellebjerg, Finn, additional, Sørensen, Per Soelberg, additional, Hilt Christensen, Claudia C, additional, Rasmussen, Peter V, additional, Jensen, Michael B, additional, Frederiksen, Jette L, additional, Bramow, Stephan, additional, Mathiesen, Henrik K, additional, Schreiber, Karen I, additional, Horakova, Dana, additional, Havrdova, Eva K, additional, Alroughani, Raed, additional, Izquierdo, Guillermo, additional, Eichau, Sara, additional, Ozakbas, Serkan, additional, Patti, Francesco, additional, Onofrj, Marco, additional, Lugaresi, Alessandra, additional, Terzi, Murat, additional, Grammond, Pierre, additional, Grand Maison, Francois, additional, Yamout, Bassem, additional, Prat, Alexandre, additional, Girard, Marc, additional, Duquette, Pierre, additional, Boz, Cavit, additional, Trojano, Maria, additional, McCombe, Pamela, additional, Slee, Mark, additional, Lechner-Scott, Jeannette, additional, Turkoglu, Recai, additional, Sola, Patrizia, additional, Ferraro, Diana, additional, Granella, Franco, additional, Shaygannejad, Vahid, additional, Prevost, Julie, additional, Skibina, Olga, additional, Solaro, Claudio, additional, Karabudak, Rana, additional, Wijmeersch, Bart V, additional, Csepany, Tunde, additional, Spitaleri, Daniele, additional, Vucic, Steve, additional, Casey, Romain, additional, Debouverie, Marc, additional, Edan, Gilles, additional, Ciron, Jonathan, additional, Ruet, Aurélie, additional, Sèze, Jérôme D, additional, Maillart, Elisabeth, additional, Zephir, Hélène, additional, Labauge, Pierre, additional, Defer, Gilles, additional, Lebrun, Christine, additional, Moreau, Thibault, additional, Berger, Eric, additional, Clavelou, Pierre, additional, Pelletier, Jean, additional, Stankoff, Bruno, additional, Gout, Olivier, additional, Thouvenot, Eric, additional, Heinzlef, Olivier, additional, Al-Khedr, Abdullatif, additional, Bourre, Bertrand, additional, Casez, Olivier, additional, Cabre, Philippe, additional, Montcuquet, Alexis, additional, Wahab, Abir, additional, Camdessanché, Jean-Philippe, additional, Marousset, Aude, additional, Patry, Ivania, additional, Hankiewicz, Karolina, additional, Pottier, Corinne, additional, Maubeuge, Nicolas, additional, Labeyrie, Céline, additional, Nifle, Chantal, additional, Leray, Emmanuelle, additional, Laplaud, David A, additional, Butzkueven, Helmut, additional, Kalincik, Tomas, additional, Vukusic, Sandra, additional, and Magyari, Melinda, additional

Published
2021
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30. Genotype–phenotype correlation in French patients with myelin protein zero gene‐related inherited neuropathy

Author

Subréville, Marie, primary, Bonello‐Palot, Nathalie, additional, Yahiaoui, Douniazed, additional, Beloribi‐Djefaflia, Sadia, additional, Fernandes, Sara, additional, Stojkovic, Tanya, additional, Cassereau, Julien, additional, Péréon, Yann, additional, Echaniz‐Laguna, Andoni, additional, Violleau, Marie‐Hélène, additional, Soulages, Antoine, additional, Louis, Sarah Léonard, additional, Masingue, Marion, additional, Magot, Armelle, additional, Delmont, Emilien, additional, Sacconi, Sabrina, additional, Adams, David, additional, Labeyrie, Céline, additional, Genestet, Steeve, additional, Noury, Jean‐Baptiste, additional, Chanson, Jean‐Baptiste, additional, Lévy, Nicolas, additional, Juntas‐Morales, Raul, additional, Tard, Céline, additional, Sole, Guilhem, additional, and Attarian, Shahram, additional

Published
2021
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31. sj-pdf-1-msj-10.1177_1352458520981300 – Supplemental material for Determinants of therapeutic lag in multiple sclerosis

Author

Izanne Roos, Leray, Emmanuelle, Frascoli, Federico, Casey, Romain, J William L Brown, Horakova, Dana, Havrdova, Eva Kubala, Debouverie, Marc, Trojano, Maria, Patti, Francesco, Izquierdo, Guillermo, Eichau, Sara, Edan, Gilles, Prat, Alexandre, Girard, Marc, Duquette, Pierre, Onofrj, Marco, Lugaresi, Alessandra, Grammond, Pierre, Ciron, Jonathan, Ruet, Aurélie, Ozakbas, Serkan, Seze, Jérôme De, Louapre, Céline, Zephir, Hélène, Sá, Maria José, Sola, Patrizia, Ferraro, Diana, Labauge, Pierre, Defer, Gilles, Bergamaschi, Roberto, Lebrun-Frenay, Christine, Boz, Cavit, Cartechini, Elisabetta, Moreau, Thibault, Laplaud, David, Lechner-Scott, Jeannette, Francois Grand’Maison, Gerlach, Oliver, Terzi, Murat, Granella, Franco, Alroughani, Raed, Iuliano, Gerardo, Pesch, Vincent Van, Wijmeersch, Bart Van, Spitaleri, Daniele LA, Soysal, Aysun, Berger, Eric, Prevost, Julie, Aguera-Morales, Eduardo, McCombe, Pamela, Triviño, Tamara Castillo, Clavelou, Pierre, Pelletier, Jean, Turkoglu, Recai, Stankoff, Bruno, Gout, Olivier, Thouvenot, Eric, Heinzlef, Olivier, Sidhom, Youssef, Gouider, Riadh, Tunde Csepany, Bourre, Bertrand, Abdullatif Al Khedr, Casez, Olivier, Cabre, Philippe, Montcuquet, Alexis, Wahab, Abir, Jean-Philippe Camdessanche, Maurousset, Aude, Patry, Ivania, Hankiewicz, Karolina, Pottier, Corinne, Maubeuge, Nicolas, Labeyrie, Céline, Nifle, Chantal, Coles, Alasdair, Malpas, Charles B, Vukusic, Sandra, Butzkueven, Helmut, and Kalincik, Tomas

Subjects
FOS: Clinical medicine, 111702 Aged Health Care, FOS: Health sciences, 110904 Neurology and Neuromuscular Diseases
Abstract

Supplemental material, sj-pdf-1-msj-10.1177_1352458520981300 for Determinants of therapeutic lag in multiple sclerosis by Izanne Roos, Emmanuelle Leray, Federico Frascoli, Romain Casey, J William L Brown, Dana Horakova, Eva Kubala Havrdova, Marc Debouverie, Maria Trojano, Francesco Patti, Guillermo Izquierdo, Sara Eichau, Gilles Edan, Alexandre Prat, Marc Girard, Pierre Duquette, Marco Onofrj, Alessandra Lugaresi, Pierre Grammond, Jonathan Ciron, Aurélie Ruet, Serkan Ozakbas, Jérôme De Seze, Céline Louapre, Hélène Zephir, Maria José Sá, Patrizia Sola, Diana Ferraro, Pierre Labauge, Gilles Defer, Roberto Bergamaschi, Christine Lebrun-Frenay, Cavit Boz, Elisabetta Cartechini, Thibault Moreau, David Laplaud, Jeannette Lechner-Scott, Francois Grand’Maison, Oliver Gerlach, Murat Terzi, Franco Granella, Raed Alroughani, Gerardo Iuliano, Vincent Van Pesch, Bart Van Wijmeersch, Daniele LA Spitaleri, Aysun Soysal, Eric Berger, Julie Prevost, Eduardo Aguera-Morales, Pamela McCombe, Tamara Castillo Triviño, Pierre Clavelou, Jean Pelletier, Recai Turkoglu, Bruno Stankoff, Olivier Gout, Eric Thouvenot, Olivier Heinzlef, Youssef Sidhom, Riadh Gouider, Tunde Csepany, Bertrand Bourre, Abdullatif Al Khedr, Olivier Casez, Philippe Cabre, Alexis Montcuquet, Abir Wahab, Jean-Philippe Camdessanche, Aude Maurousset, Ivania Patry, Karolina Hankiewicz, Corinne Pottier, Nicolas Maubeuge, Céline Labeyrie, Chantal Nifle, Alasdair Coles, Charles B Malpas, Sandra Vukusic, Helmut Butzkueven and Tomas Kalincik in Multiple Sclerosis Journal

Published
2021
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32. The effectiveness of natalizumab vs fingolimod–A comparison of international registry studies

Author

Andersen, Johanna B., Sharmin, Sifat, Lefort, Mathilde, Koch-Henriksen, Nils, Sellebjerg, Finn, Sørensen, Per Soelberg, Hilt Christensen, Claudia C., Rasmussen, Peter V., Jensen, Michael B., Frederiksen, Jette L., Bramow, Stephan, Mathiesen, Henrik K., Schreiber, Karen I., Horakova, Dana, Havrdova, Eva K., Alroughani, Raed, Izquierdo, Guillermo, Eichau, Sara, Ozakbas, Serkan, Patti, Francesco, Onofrj, Marco, Lugaresi, Alessandra, Terzi, Murat, Grammond, Pierre, Grand Maison, Francois, Yamout, Bassem, Prat, Alexandre, Girard, Marc, Duquette, Pierre, Boz, Cavit, Trojano, Maria, McCombe, Pamela, Slee, Mark, Lechner-Scott, Jeannette, Turkoglu, Recai, Sola, Patrizia, Ferraro, Diana, Granella, Franco, Shaygannejad, Vahid, Prevost, Julie, Skibina, Olga, Solaro, Claudio, Karabudak, Rana, Wijmeersch, Bart V., Csepany, Tunde, Spitaleri, Daniele, Vucic, Steve, Casey, Romain, Debouverie, Marc, Edan, Gilles, Ciron, Jonathan, Ruet, Aurélie, Sèze, Jérôme D., Maillart, Elisabeth, Zephir, Hélène, Labauge, Pierre, Defer, Gilles, Lebrun, Christine, Moreau, Thibault, Berger, Eric, Clavelou, Pierre, Pelletier, Jean, Stankoff, Bruno, Gout, Olivier, Thouvenot, Eric, Heinzlef, Olivier, Al-Khedr, Abdullatif, Bourre, Bertrand, Casez, Olivier, Cabre, Philippe, Montcuquet, Alexis, Wahab, Abir, Camdessanché, Jean Philippe, Marousset, Aude, Patry, Ivania, Hankiewicz, Karolina, Pottier, Corinne, Maubeuge, Nicolas, Labeyrie, Céline, Nifle, Chantal, Leray, Emmanuelle, Laplaud, David A., Butzkueven, Helmut, Kalincik, Tomas, Vukusic, Sandra, Magyari, Melinda, Andersen, Johanna B., Sharmin, Sifat, Lefort, Mathilde, Koch-Henriksen, Nils, Sellebjerg, Finn, Sørensen, Per Soelberg, Hilt Christensen, Claudia C., Rasmussen, Peter V., Jensen, Michael B., Frederiksen, Jette L., Bramow, Stephan, Mathiesen, Henrik K., Schreiber, Karen I., Horakova, Dana, Havrdova, Eva K., Alroughani, Raed, Izquierdo, Guillermo, Eichau, Sara, Ozakbas, Serkan, Patti, Francesco, Onofrj, Marco, Lugaresi, Alessandra, Terzi, Murat, Grammond, Pierre, Grand Maison, Francois, Yamout, Bassem, Prat, Alexandre, Girard, Marc, Duquette, Pierre, Boz, Cavit, Trojano, Maria, McCombe, Pamela, Slee, Mark, Lechner-Scott, Jeannette, Turkoglu, Recai, Sola, Patrizia, Ferraro, Diana, Granella, Franco, Shaygannejad, Vahid, Prevost, Julie, Skibina, Olga, Solaro, Claudio, Karabudak, Rana, Wijmeersch, Bart V., Csepany, Tunde, Spitaleri, Daniele, Vucic, Steve, Casey, Romain, Debouverie, Marc, Edan, Gilles, Ciron, Jonathan, Ruet, Aurélie, Sèze, Jérôme D., Maillart, Elisabeth, Zephir, Hélène, Labauge, Pierre, Defer, Gilles, Lebrun, Christine, Moreau, Thibault, Berger, Eric, Clavelou, Pierre, Pelletier, Jean, Stankoff, Bruno, Gout, Olivier, Thouvenot, Eric, Heinzlef, Olivier, Al-Khedr, Abdullatif, Bourre, Bertrand, Casez, Olivier, Cabre, Philippe, Montcuquet, Alexis, Wahab, Abir, Camdessanché, Jean Philippe, Marousset, Aude, Patry, Ivania, Hankiewicz, Karolina, Pottier, Corinne, Maubeuge, Nicolas, Labeyrie, Céline, Nifle, Chantal, Leray, Emmanuelle, Laplaud, David A., Butzkueven, Helmut, Kalincik, Tomas, Vukusic, Sandra, and Magyari, Melinda

Abstract

Background: Natalizumab and fingolimod were the first preparations recommended for disease breakthrough in priorly treated relapsing-remitting multiple sclerosis. Of three published head-to-head studies two showed that natalizumab is the more effective to prevent relapses and EDSS worsening. Methods: By re-analyzing original published results from MSBase, France, and Denmark using uniform methodologies, we aimed at identifying the effects of differences in methodology, in the MS-populations, and at re-evaluating the differences in effectiveness between the two drugs. We gained access to copies of the individual amended databases and pooled all data. We used uniform inclusion/exclusion criteria and statistical methods with Inverse Probability Treatment Weighting. Results: The pooled analyses comprised 968 natalizumab- and 1479 fingolimod treated patients. The on-treatment natalizumab/fingolimod relapse rate ratio was 0.77 (p=0.004). The hazard ratio (HR) for a first relapse was 0.82 (p=0.030), and the HR for sustained EDSS improvement was 1.4 (p=0.009). There were modest differences between each of the original published studies and the replication study, but the conclusions of the three original studies remained unchanged: in two of them natalizumab was more effective, but in the third there was no difference between natalizumab and fingolimod. Conclusion: The results were largely invariant to the epidemiological and statistical methods but differed between the MS populations. Generally, the advantage of natalizumab was confirmed.

Published
2021

34. Therapeutic education programme in TTR-FAP

Author

Théaudin, Marie, Cauquil, Cécile, Antonini, Teresa, Algalarrondo, Vincent, Labeyrie, Céline, Aycaguer, Sophie, Kubezyk, Marie, Lemoine, Colombe, Nonnez, Géraldine, Morier, Agnès, Kouchi, Nadia, Darras, Amandine, Coste, Nathalie, Clément, Mireille, and Adams, David

Published
2015
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35. Comparative effectiveness of natalizumab and fingolimod in subgroups of patients with relapsing-remitting multiple sclerosis from three international cohorts (2943)

Author

Sharmin, Sifat, primary, Lefort, Mathilde, additional, Andersen, Johanna Balslev, additional, Leray, Emmanuelle, additional, Horakova, Dana, additional, Havrdova, Eva, additional, Alroughani, Raed, additional, Ayuso, Guillermo Izquierdo, additional, Ozakbas, Serkan, additional, Patti, Francesco, additional, Onofrj, Marco, additional, Lugaresi, Alessandra, additional, Terzi, Murat, additional, Grammond, Pierre, additional, Grand’Maison, Francois, additional, Yamout, Bassem, additional, Prat, Alexandre, additional, Girard, Marc, additional, Duquette, Pierre, additional, Boz, Cavit, additional, Trojano, Maria, additional, McCombe, Pamela, additional, Slee, Mark, additional, Lechner-Scott, Jeannette, additional, Turkoglu, Recai, additional, Sola, Patrizia, additional, Ferraro, Diana, additional, Granella, Franco, additional, Prevost, Julie, additional, Maimone, Davide, additional, Skibina, Olga, additional, Buzzard, Katherine, additional, Van der Walt, Anneke, additional, Van Wijmeersch, Bart, additional, Csepany, Tunde, additional, Spitaleri, Daniele Litterio A., additional, Vucic, Ostoja (Steve), additional, Casey, Romain, additional, Debouverie, Marc, additional, Edan, Gilles, additional, Ciron, Jonathan, additional, Ruet, Aurélie, additional, De Sèze, Jérôme, additional, Maillart, Elisabeth, additional, Zephir, Hélène, additional, Labauge, Pierre, additional, Defer, Gilles, additional, Lebrun-Frénay, Christine, additional, Moreau, Thibault, additional, Berger, Eric, additional, Clavelou, Pierre, additional, Pelletier, Jean, additional, Stankoff, Bruno, additional, Gout, Olivier, additional, Thouvenot, Eric, additional, Heinzlef, Olivier, additional, Al-Khedr, Abullatif, additional, Bourre, Bertrand, additional, Casez, Olivier, additional, Cabre, Philippe, additional, Montcuquet, Alexis, additional, Wahab, Abir, additional, Camdessanché, Jean-Philippe, additional, Maurousset, Aude, additional, Patry, Ivania, additional, Hankiewicz, Karolina, additional, Pottier, Corinne, additional, Maubeuge, Nicolas, additional, Labeyrie, Céline, additional, Nifle, Chantal, additional, Laplaud, David, additional, Koch-Henriksen, Nils, additional, Sellebjerg, Finn Thorup, additional, Soerensen, Per Soelberg, additional, Pfleger, Claudia Christina, additional, Rasmussen, Peter Vestergaard, additional, Jensen, Michael Broksgaard, additional, Frederiksen, Jette Lautrup, additional, Bramow, Stephan, additional, Mathiesen, Henrik Kahr, additional, Schreiber, Karen Ingrid, additional, Magyari, Melinda, additional, Vukusic, Sandra, additional, Butzkueven, Helmut, additional, and Kalincik, Tomas, additional

Published
2021
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36. Inflammatory demyelinating polyneuropathies and lymphoma: clues to diagnosis and therapy

Author

Noel, Nicolas, primary, Beaudonnet, Guillemette, additional, Cauquil, Cécile, additional, Laurenge, Alice, additional, De Menthon, Mathilde, additional, Labeyrie, Céline, additional, Chrétien, Pascale, additional, Adam, Clovis, additional, Hacein-Bey-Abina, Salima, additional, Goujard, Cécile, additional, Lambotte, Olivier, additional, and Adams, David, additional

Published
2021
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37. Determinants of therapeutic lag in multiple sclerosis

Author

Roos, Izanne, primary, Leray, Emmanuelle, additional, Frascoli, Federico, additional, Casey, Romain, additional, Brown, J William L, additional, Horakova, Dana, additional, Havrdova, Eva Kubala, additional, Debouverie, Marc, additional, Trojano, Maria, additional, Patti, Francesco, additional, Izquierdo, Guillermo, additional, Eichau, Sara, additional, Edan, Gilles, additional, Prat, Alexandre, additional, Girard, Marc, additional, Duquette, Pierre, additional, Onofrj, Marco, additional, Lugaresi, Alessandra, additional, Grammond, Pierre, additional, Ciron, Jonathan, additional, Ruet, Aurélie, additional, Ozakbas, Serkan, additional, De Seze, Jérôme, additional, Louapre, Céline, additional, Zephir, Hélène, additional, Sá, Maria José, additional, Sola, Patrizia, additional, Ferraro, Diana, additional, Labauge, Pierre, additional, Defer, Gilles, additional, Bergamaschi, Roberto, additional, Lebrun-Frenay, Christine, additional, Boz, Cavit, additional, Cartechini, Elisabetta, additional, Moreau, Thibault, additional, Laplaud, David, additional, Lechner-Scott, Jeannette, additional, Grand’Maison, Francois, additional, Gerlach, Oliver, additional, Terzi, Murat, additional, Granella, Franco, additional, Alroughani, Raed, additional, Iuliano, Gerardo, additional, Van Pesch, Vincent, additional, Van Wijmeersch, Bart, additional, Spitaleri, Daniele LA, additional, Soysal, Aysun, additional, Berger, Eric, additional, Prevost, Julie, additional, Aguera-Morales, Eduardo, additional, McCombe, Pamela, additional, Castillo Triviño, Tamara, additional, Clavelou, Pierre, additional, Pelletier, Jean, additional, Turkoglu, Recai, additional, Stankoff, Bruno, additional, Gout, Olivier, additional, Thouvenot, Eric, additional, Heinzlef, Olivier, additional, Sidhom, Youssef, additional, Gouider, Riadh, additional, Csepany, Tunde, additional, Bourre, Bertrand, additional, Al Khedr, Abdullatif, additional, Casez, Olivier, additional, Cabre, Philippe, additional, Montcuquet, Alexis, additional, Wahab, Abir, additional, Camdessanche, Jean-Philippe, additional, Maurousset, Aude, additional, Patry, Ivania, additional, Hankiewicz, Karolina, additional, Pottier, Corinne, additional, Maubeuge, Nicolas, additional, Labeyrie, Céline, additional, Nifle, Chantal, additional, Coles, Alasdair, additional, Malpas, Charles B, additional, Vukusic, Sandra, additional, Butzkueven, Helmut, additional, and Kalincik, Tomas, additional

Published
2021
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39. Quality of life outcomes in APOLLO, the phase 3 trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis

Author

Obici, Laura, primary, Berk, John L., additional, González-Duarte, Alejandra, additional, Coelho, Teresa, additional, Gillmore, Julian, additional, Schmidt, Hartmut H.-J., additional, Schilling, Matthias, additional, Yamashita, Taro, additional, Labeyrie, Céline, additional, Brannagan, Thomas H., additional, Ajroud-Driss, Senda, additional, Gorevic, Peter, additional, Kristen, Arnt V., additional, Franklin, Jaclyn, additional, Chen, Jihong, additional, Sweetser, Marianne T., additional, Wang, Jing Jing, additional, and Adams, David, additional

Published
2020
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42. Conjunctival lymphangiectasia as a biomarker of severe systemic disease in Ser77Tyr hereditary transthyretin amyloidosis.

Author

Bunod, Roxane, Adams, David, Cauquil, Cécile, Francou, Bruno, Labeyrie, Céline, Bourenane, Halima, Adam, Clovis, Algalarrondo, Vincent, Slama, Michel, Darce-Bello, Martha, Barreau, Emmanuel, Labetoulle, Marc, and Rousseau, Antoine

Abstract

Aims To investigate the relationship between the ophthalmic and systemic phenotypes in patients with hereditary transthyretin amyloidosis with the S77Y mutation (ATTRS77Y). Methods In this cross-sectional study, patients with genetically confirmed ATTRS77Y amyloidosis were enrolled. All patients underwent complete neurological examination, including staging with the Neuropathy Impairment Score (NIS), Polyneuropathy Disability (PND) score; complete cardiological evaluation, including echocardiography, cardiac MRI and/or cardiac scintigraphy and complete ophthalmic evaluation, including slit lamp examination and fundus examination. Ocular ancillary tests (fluorescein and indocyanine green angiography, and anterior segment optical coherence tomography) were performed in cases with abnormal findings. The Kruskal-Wallis test was used for quantitative outcomes and Fisher's exact test for qualitative outcomes. Statistical significance was indicated by p<0.05 (two tailed). Results The study sample was composed of 24 ATTRS77Y patients. The mean patient age was 58.4±12.4 years. None of the patients presented with amyloid deposits in the anterior chamber, secondary glaucoma or vitreous amyloidosis. Retinal angiopathy was observed in four patients, complicated with retinal ischaemia in one patient. Conjunctival lymphangiectasia (CL) was detected in 13 patients (54%), associated with perilymphatic amyloid deposits. The presence of CL was statistically associated with more severe neurological disease (NIS=43.3±31.9 vs 18.9 ±20.4; PND=2.6±1.0 vs 1.4±0.7 in patients with and without CL, respectively; both p<0.05) and amyloid cardiomyopathy (p=0.002). Conclusion In ATTRS77Y patients, CL is common and could serve as a potential biomarker for severe systemic disease. There were neither anterior chamber deposits, secondary glaucoma nor vitreous deposits in ATTRS77Y patients. [ABSTRACT FROM AUTHOR]

Published
2020
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45. IRM plexique dans les CISP

Author

Fargeot, Guillaume, primary, Labeyrie, Céline, additional, Viala, Karine, additional, Leger, Jean-Marc, additional, Theaudin, Marie, additional, Vandendries, Christophe, additional, and Adams, David, additional

Published
2018
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46. Indications et suivi des autogreffes de cellules souches hématopoïétiques dans les maladies auto-immunes et auto-inflammatoires : recommandations de la Société francophone de greffe de moelle et de thérapie cellulaire (SFGM-TC)

Author

Pugnet, Grégory, primary, Castilla-Llorente, Christina, additional, Puyade, Mathieu, additional, Terriou, Louis, additional, Badoglio, Manuela, additional, Deligny, Christophe, additional, Guillaume-Jugnot, Perrine, additional, Labeyrie, Céline, additional, Benzidia, Ilham, additional, Faivre, Hélène, additional, Lansiaux, Pauline, additional, Marjanovic, Zora, additional, Bourhis, Jean-Henri, additional, Faucher, Catherine, additional, Furst, Sabine, additional, Huynh, Anne, additional, Martin, Thierry, additional, Vermersch, Patrick, additional, Yakoub-Agha, Ibrahim, additional, and Farge, Dominique, additional

Published
2017
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