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1. Examining the environmental risk factors of progressive-onset and relapsing-onset multiple sclerosis: recruitment challenges, potential bias, and statistical strategies

Author

Li, Ying, Saul, Alice, Taylor, Bruce, Ponsonby, Anne-Louise, Simpson-Yap, Steve, Blizzard, Leigh, Broadley, Simon, Lechner-Scott, Jeannette, Karabudak, Rana, Patti, Francesco, Eichau, Sara, Onofrj, Marco, Ozakbas, Serkan, Horakova, Dana, Kubala Havrdova, Eva, Grand’Maison, Francois, Alroughani, Raed, Gerlach, Oliver, Amato, Maria Pia, Altintas, Ayse, Girard, Marc, Duquette, Pierre, Blanco, Yolanda, Ramo-Tello, Cristina, Laureys, Guy, Kalincik, Tomas, Khoury, Samia J., Shaygannejad, Vahid, Etemadifar, Masoud, Singhal, Bhim, Mrabet, Saloua, Foschi, Matteo, Habek, Mario, John, Nevin, Hughes, Stella, McCombe, Pamela, Ampapa, Radek, van der Walt, Anneke, Butzkueven, Helmut, de Gans, Koen, McGuigan, Chris, Oreja-Guevara, Celia, Sa, Maria Jose, Petersen, Thor, Al-Harbi, Talal, Sempere, Angel Perez, Van Wijmeersch, Bart, Grigoriadis, Nikolaos, Prevost, Julie, Gray, Orla, Castillo-Triviño, Tamara, Macdonell, Richard, Lugaresi, Alessandra, Sajedi, Seyed Aidin, and van der Mei, Ingrid

Published
2024
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3. Clinical diagnostic utility of transcranial magnetic stimulation in neurological disorders. Updated report of an IFCN committee

Author

Vucic, Steve, Stanley Chen, Kai-Hsiang, Kiernan, Matthew C., Hallett, Mark, Benninger, David.H., Di Lazzaro, Vincenzo, Rossini, Paolo M, Benussi, Alberto, Berardelli, Alfredo, Currà, Antonio, Krieg, Sandro M, Lefaucheur, Jean-Pascal, Long Lo, Yew, Macdonell, Richard A, Massimini, Marcello, Rosanova, Mario, Picht, Thomas, Stinear, Cathy M, Paulus, Walter, Ugawa, Yoshikazu, Ziemann, Ulf, and Chen, Robert

Published
2023
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5. Electroclinical biomarkers of autoimmune encephalitis

Author

Wesselingh, Robb, Broadley, James, Buzzard, Katherine, Tarlinton, David, Seneviratne, Udaya, Kyndt, Chris, Stankovich, Jim, Sanfilippo, Paul, Nesbitt, Cassie, D'Souza, Wendyl, Macdonell, Richard, Butzkueven, Helmut, O'Brien, Terence J., and Monif, Mastura

Published
2022
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6. Effectiveness of autologous haematopoietic stem cell transplantation versus natalizumab in progressive multiple sclerosis.

Author

Kalincik, Tomas, Sharmin, Sifat, Roos, Izanne, Massey, Jennifer, Sutton, Ian, Withers, Barbara, Freedman, Mark S, Atkins, Harold, Krasulova, Eva, Havrdova, Eva Kubala, Trneny, Marek, Kozak, Tomas, Burman, Joachim, Macdonell, Richard, Torkildsen, Øivind, Bø, Lars, Lehmann, Anne Kristine, Sharrack, Basil, and Snowden, John

Subjects
HEMATOPOIETIC stem cell transplantation, MEDICAL sciences, MEDICAL research, HEMATOPOIETIC stem cells, RESPIRATORY infections, FEBRILE neutropenia
Published
2024
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7. Routine CSF parameters as predictors of disease course in multiple sclerosis: an MSBase cohort study

Author

Dekeyser, Cathérine, primary, Hautekeete, Matthias, additional, Cambron, Melissa, additional, Van Pesch, Vincent, additional, Patti, Francesco, additional, Kuhle, Jens, additional, Khoury, Samia, additional, Lechner Scott, Jeanette, additional, Gerlach, Oliver, additional, Lugaresi, Alessandra, additional, Maimone, Davide, additional, Surcinelli, Andrea, additional, Grammond, Pierre, additional, Kalincik, Tomas, additional, Habek, Mario, additional, Willekens, Barbara, additional, Macdonell, Richard, additional, Lalive, Patrice, additional, Csepany, Tunde, additional, Butzkueven, Helmut, additional, Boz, Cavit, additional, Tomassini, Valentina, additional, Foschi, Matteo, additional, Sánchez-Menoyo, José Luis, additional, Altintas, Ayse, additional, Mrabet, Saloua, additional, Iuliano, Gerardo, additional, Sa, Maria Jose, additional, Alroughani, Raed, additional, Karabudak, Rana, additional, Aguera-Morales, Eduardo, additional, Gray, Orla, additional, de Gans, Koen, additional, van der Walt, Anneke, additional, McCombe, Pamela A, additional, Deri, Norma, additional, Garber, Justin, additional, Al-Asmi, Abdullah, additional, Skibina, Olga, additional, Duquette, Pierre, additional, Cartechini, Elisabetta, additional, Spitaleri, Daniele, additional, Gouider, Riadh, additional, Soysal, Aysun, additional, Van Hijfte, Liesbeth, additional, Slee, Mark, additional, Amato, Maria Pia, additional, Buzzard, Katherine, additional, and Laureys, Guy, additional

Published
2024
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8. Safety and efficacy of MD1003 (high-dose biotin) in patients with progressive multiple sclerosis (SPI2): a randomised, double-blind, placebo-controlled, phase 3 trial

Author

Reingold, Stephen, Duquette, Pierre, Derfuss, Tobias, Fazekas, Franz, Sormani, Maria Pia, Lisak, Robert P., Graves, Jennifer, Krieger, Stephen, Zabad, Rana K., Newsome, Scott, Barton, Joshua, MacDonell, Richard, Marriott, Mark, De Klippel, Nina, Laureys, Guy, Willekens, Barbara, Devonshire, Virginia, Freedman, Mark, Girard, J Marc, Giacomini, Paul, McKelvey, Roger, Selchen, Daniel, Vorobeychik, Galina, Witkowski, Ludivine, Ampapa, Radek, Preiningerova, Jana Lizrova, Meluzinova, Eva, Talab, Radomir, Vachova, Marta, Aktas, Orhan, Buttmann, Mathias, Birte, Elias-Hamp, Kuempfel, Tania, Friedemann, Paul, Rau, Daniela, Reifschneider, Gerd, Sokolowski, Piotr, Tumani, Hayrettin, Satori, Maria, Pozzilli, Carlo, Klosek, Agata, Koscielniak, Jozef, Waldemar, Fryze, Zajda, Malgorzata, Gonzalez, Rafael Arroyo, Ayuso, Guillermo Izquierdo, Sanchez, Victoria Fernandez, Guevara, Celia Oreja, Rodriguez, Jose Enrique Martinez, Montalban, Xavier, Ramio-Torrenta, Lluis, Brundin, Lou, Lycke, Jan, Terzi, Murat, Guadagno, Joe, Mahad, Don, Pace, Adrian, Schmierer, Klaus, Toosy, Ahmed, Webb, Stewart, Agius, Mark, Amezcua, Lilyana, Apperson, Michelle, Bagert, Bridget, Bandari, Daniel, Bernitsas, Evanthia, Calkwood, Jonathan, Carter, Jonathan, Cohen, Bruce, Conway, Devon, Cooper, Joanna, Corboy, John, Coyle, Patricia, Cree, Bruce, Freedman, Mitchel, Ford, Corey, Fox, Edward, Goldman, Myla, Greenberg, Benjamin, Kita, Mariko, Leist, Thomas, Lynch, Sharon, Miller, Aaron, Moses, Harold, Naismith, Robert, Picone, Mary Ann, Perminder, Bhatia, Rae-Grant, Alexander, Rammohan, Kottil, Reder, Anthony, Riley, Claire, Robertson, Derrick, Rowe, Vernon, Saidha, Shiv, Samkoff, Lawrence, Severson, Christopher, Smoot, Kyle, Stoll, Sharon, Trudell, Randall, Weinstock-Guttman, Bianca, Yathiraj, Sanjay, Cree, Bruce A C, Cutter, Gary, Wolinsky, Jerry S, Freedman, Mark S, Comi, Giancarlo, Giovannoni, Gavin, Hartung, Hans-Peter, Arnold, Douglas, Kuhle, Jens, Block, Valerie, Munschauer, Frederick E, Sedel, Frédéric, and Lublin, Fred D

Published
2020
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10. Dominant NARS1 mutations causing axonal Charcot–Marie–Tooth disease expand NARS1-associated diseases

Author

Beijer, Danique, primary, Marte, Sheila, additional, Li, Jiaxin C, additional, De Ridder, Willem, additional, Chen, Jessie Z, additional, Tadenev, Abigail L D, additional, Miers, Kathy E, additional, Deconinck, Tine, additional, Macdonell, Richard, additional, Marques, Wilson, additional, De Jonghe, Peter, additional, Pratt, Samia L, additional, Meyer-Schuman, Rebecca, additional, Züchner, Stephan, additional, Antonellis, Anthony, additional, Burgess, Robert W, additional, and Baets, Jonathan, additional

Published
2024
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11. Vitamin D did not reduce multiple sclerosis disease activity after a clinically isolated syndrome

Author

Butzkueven, Helmut, primary, Ponsonby, Anne-Louise, additional, Stein, Mark S, additional, Lucas, Robyn M, additional, Mason, Deborah, additional, Broadley, Simon, additional, Kilpatrick, Trevor, additional, Lechner-Scott, Jeannette, additional, Barnett, Michael, additional, Carroll, William, additional, Mitchell, Peter, additional, Hardy, Todd A, additional, Macdonell, Richard, additional, McCombe, Pamela, additional, Lee, Andrew, additional, Kalincik, Tomas, additional, van der Walt, Anneke, additional, Lynch, Chris, additional, Abernethy, David, additional, Willoughby, Ernest, additional, Barkhof, Frederik, additional, MacManus, David, additional, Clarke, Michael, additional, Andrew, Julie, additional, Morahan, Julia, additional, Zhu, Chao, additional, Dear, Keith, additional, and Taylor, Bruce V, additional

Published
2023
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13. Vitamin D did not reduce multiple sclerosis disease activity after a clinically isolated syndrome.

Author

Butzkueven, Helmut, Ponsonby, Anne-Louise, Stein, Mark S, Lucas, Robyn M, Mason, Deborah, Broadley, Simon, Kilpatrick, Trevor, Lechner-Scott, Jeannette, Barnett, Michael, Carroll, William, Mitchell, Peter, Hardy, Todd A, Macdonell, Richard, McCombe, Pamela, Lee, Andrew, Kalincik, Tomas, van der Walt, Anneke, Lynch, Chris, Abernethy, David, and Willoughby, Ernest

Subjects
MULTIPLE sclerosis, DIETARY supplements, VITAMINS, VITAMIN D, DISEASE relapse
Abstract

Low serum levels of 25-hydroxyvitamin D [25(OH)D] and low sunlight exposure are known risk factors for the development of multiple sclerosis. Add-on vitamin D supplementation trials in established multiple sclerosis have been inconclusive. The effects of vitamin D supplementation to prevent multiple sclerosis is unknown. We aimed to test the hypothesis that oral vitamin D3 supplementation in high-risk clinically isolated syndrome (abnormal MRI, at least three T2 brain and/or spinal cord lesions), delays time to conversion to definite multiple sclerosis, that the therapeutic effect is dose-dependent, and that all doses are safe and well tolerated. We conducted a double-blind trial in Australia and New Zealand. Eligible participants were randomized 1:1:1:1 to placebo, 1000, 5000 or 10 000 international units (IU) of oral vitamin D3 daily within each study centre (n = 23) and followed for up to 48 weeks. Between 2013 and 2021, we enrolled 204 participants. Brain MRI scans were performed at baseline, 24 and 48 weeks. The main study outcome was conversion to clinically definite multiple sclerosis based on the 2010 McDonald criteria defined as either a clinical relapse or new brain MRI T2 lesion development. We included 199 cases in the intention-to-treat analysis based on assigned dose. Of these, 116 converted to multiple sclerosis by 48 weeks (58%). Compared to placebo, the hazard ratios (95% confidence interval) for conversion were 1000 IU 0.87 (0.50, 1.50); 5000 IU 1.37 (0.82, 2.29); and 10 000 IU 1.28 (0.76, 2.14). In an adjusted model including age, sex, latitude, study centre and baseline symptom number, clinically isolated syndrome onset site, presence of infratentorial lesions and use of steroids, the hazard ratios (versus placebo) were 1000 IU 0.80 (0.45, 1.44); 5000 IU 1.36 (0.78, 2.38); and 10 000 IU 1.07 (0.62, 1.85). Vitamin D3 supplementation was safe and well tolerated. We did not demonstrate reduction in multiple sclerosis disease activity by vitamin D3 supplementation after a high-risk clinically isolated syndrome. [ABSTRACT FROM AUTHOR]

Published
2024
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16. Natalizumab treatment shows low cumulative probabilities of confirmed disability worsening to EDSS milestones in the long-term setting

Author

Correale, Jorge, Caride, Alejandro, Deri, Norma H., Ballario, Carlos, Broadley, Simon, Kneebone, Chris, Barnett, Michael, Pollard, John, Hodgkinson, Suzanne, Kermode, Allan, Macdonell, Richard, King, John, Butzkueven, Helmut, Lechner-Scott, Jeannette, Saines, Noel, Slee, Mark, Plummer, Chris, Willekens, Barbara, Vanopdenbosch, Ludo, Belachew, Shibeshih, Phan-Ba, Rémy, Delvaux, Valérie, Bissay, Veronique, Debruyne, Jan, Decoo, Danny, Crols, Roeland, Symons, Anoek, Nagels, Guy, Van Pesch, Vincent, Sindic, Christian, Dubois, Benedicte, Medaer, Robert, D'Hooghe, Marie, Guillaume, Daniel, De Smet, Eric, Seeldrayers, Pierrette, Lysandropoulos, Andreas, Vokaer, Mathieu, Geens, Karine, Willems, Christina, Denayer, Pierre, Bureau, Michel, Retif, Cecile, Dupuis, Michel, Bouquiaux, Olivier, Vanderdonckt, Patrick, van Landegem, William, Caekebeke, Jo, Van Ingelghem, Erwin, Peeters, Katelijne, Gerard, Pascale, de Noordhout, Alain Maertens, Desfontaines, Philippe, Urbain, Etienne, Declercq, Inge, Van Wijmeersch, Bart, Vanroose, Erwin, Wibail, Alain, Barthomolé, Emmanuel, Ursell, Melanie, Sweet, Margaret Elizabeth, Howse, David, Jichici, Draga, Shawush, Melad, Namaka, Mike, Traboulsee, Anthony, Hashimoto, Stan, Lo, Raymond, Marchetti, Paul, Lapierre, Yves, Jacques, Francois, MacLean, Gregg, Bhan, Virender, Duquette, Pierre, Stewart, Bradley, Paulseth, John, Kremenchutzky, Marcelo, Vorobeychik, Galina, O'Connor, Paul, Grand'Maison, François, Havrdova, Eva, Meluzinová, Eva, Valis, Martin, Talab, Radomír, Stourac, Pavel, Zapletalová, Olga, Dufek, Michal, Sládková, Vladimíra, Novotna, Alena, Vancurová, Romana, Lhotaková, Libuse, Fiedler, Jiri, Vachova, Marta, Dolezil, David, Stetkarova, Ivana, Rehankova, Adela, Psenica, Petr, Ulehlova, Veronika, Feketova, Sona, Skoda, Ondrej, Färkkilä, Markus, Taneli, Sarasoja, Koivisto, Keijo, Seppä, Juha Matti, Airas, Laura, Elovaara, Irina, Hartikainen, Päivi, Pirttila, Tuula, Louchart, Pierre, Ille, Olivier, Thenint, Jean philippe, Godet, Etienne, Vioud, Marcel Maillet, Colamarino, Renato, Gugenheim, Michel, Grimaud, Jerome, Kopf, Audrey, Billy, Christophe, Huttin, Bernard, Borsotti, Jean paul, Devos, Philippe, Kendjuo, Jean bertin N, Verier, Albert, Chapuis, Stephane, Daluzeau, Nathalie, Angibaud, Gilles, Uriot, Marie-Sylvie Artaud, Ziegler, François, Sellal, François, Moulignier, Antoine, Lavenu, Isabelle, Ismail, Samir, Devy, Richard, Suceveanu, Manuel, Wagner, Marc, Marcel, Sebastien, Derouiche, Faycal, Mostoufizadehghalamfarsa, Sohrab, Delalande, Sophie, Ruggieri, Irene, Van Nieuwenhuyse, Catherine Bossu, Nifle, Chantal, Ondze, Basile, Vasilescu, Carmen Gurau, Vongsouthi, Cyrille, Coustans, Marc, Anne, Olivier, Amevigbe, Josephine, Servan, Jerome, Merienne, Marc, Eck, Philippe, Berroir, Stephane, Busson, Philippe, Barroso, Bruno, Larrieu, Jean-Marc, Giendaj, Catherine Louvet, Malkoun, Imad, Hautecoeur, Patrick, Kwiatkowski, Arnaud, Pouliquen, Andre, Garrigues, Guillaume, Delerue, Olivier, Giraud, Pierric, Gere, Julien, Vaunaize, Jean, Dereeper, Olivier, Seiller, Nicolas, Alsassa, Roger, Vlaicu, Mihaela, Neuville, Veronique, Faucheux, Jean Marc, Bernady, Patricia, Fanjaud, Guy, Viallet, François, Schroeter, Michael, Schlemilch-Paschen, Sylke, Lange, Thomas, Bohr, Kin-Arno, Jendroska, Klaus, Rehkopf, Elisabeth, Bergmann, Arnfin, Kleinschnitz, Christoph, Postert, Thomas, Scholz, Peter, Mauz, Uwe, Stratmann, Hubert, Siefjediers, Veneta, Prantl, Martin, Gehring, Klaus, Zellner, Ruth, Junge, Kathrin, Zellner, Anton, Bacay, Valerina, Schlegel, Eugen, Polzer, Udo, Strauss, Erik, Link, Andreas, Stenzel, Christoph, Freidel, Matthias, Drews, Joachim, Neudert, Christian, Schmitz, Frank, Jaeger, Joachim, Masri, Said, Heuberger, Wolfgang, Trausch, Beate, Ruhnke, Oliver, Scarel, Serena, Bach, Kathlen, Ernst, Michael, Landefeld, Harald, Richter, Nils, Schmidt, Stephan, Krause, Michaela, Dressel, Alezander, Ruth, Roland, Anvari, Kerstin, Gossling, Jens, Schenk, Christoph, Tiedge, Oliver, Bode, Lutz, Eder, Hans-Thomas, Pfeffer, Oliver, Krug, Reinhard, Lassek, Christoph, Fleischer, Eberhard, Meuth, Sven, Klotz, Luisa Hildegard, Peglau, Ines, Kukowski, Borries, Herting, Birgit, Guthke, Kersten, Schierenbeck, Jurgen, Brockmeier, Bernd, Albrecht, Holger, Wuttke, Matthias, Augspach-Hofmann, Regine, Gunther, Stefan, Redbrake, Martin, Franke, Christian, Buchner, Klaus, Gratz, Thomas, Horn, Rolf, Doemges, Frank, Schreiber, Martin, Brosch, Thomas, Horn, Markus, Kittlitz, Matthias, Vulturius, Gabriele, Hinse, Paul, Malessa, Rolf, Wiehler, Stephan, Katsarava, Zaza, Kastrup, Oliver, Kausch, Ulrich, Gullekes, Martin, Fickinger, Markus, Wenzel, Wilhelm, Botefur, Ingolf C., Reifschneider, Gerd, Rauer, Sebastian, Lang, Michael, Harms, Lutz, Eckhardt, Ulrich, Cursiefen, Simone, Linker, Ralf, Angstwurm, Klemens, Haas, Judith, Schuetze, Ivo, Rohm, Eva, Stienker-Fisse, H., Sailer, Michael, Bohringer, Johannes, Maurer, Mathias, Bause, Eberhard, Wersching, Ronald, Dachsel, Reinhardt, Domke, Sylke, Hoffman, Frank, Tackenberg, Bjorn, Roch, Kerstin, Ziebold, Uwe, Kallmann, Boris, Buehler, Bernhard, Faiss, Judith, Faiss, Juergen, Schimrigk, Sebastian, Menges, Christian, Knop, Karl Christian, Koehler, Wolfgang, Siever, Arno, Bufler, Johannes, Gramsl, Georg, Kuhnler, Benedicta, Maschke, Matthias, Stogbauer, Florian, Staude, Lisa, Bethke, Florian, Bitsch, Andreas, Harmjanz, Arndt D., Windsheimer, Jorg, Kieseier, Bernd C., Berkenfeld, Ralf, Tumani, Hayrettin, Kirsch, Michael, Wildemann, Brigitte, Daniels, Regina, Gottwald, Klaus, Elias, Wolfgang-Gerhard, Hoffmann, Olaf, Schwab, Matthias, Pilz, Christopher, Klostermann, Fabian, Hellwig, Kerstin, Berthele, Achim, Bayas, Antonios, Molitor, Daniel, Grothe, Christoph, Wagner, Bert, Karageorgiou, Klimentini, Mitsikostas, Dimosthenis, Kodounis, Antonios, Plaitakis, Andreas, Papadimitriou, Alexandros, Grigoriadis, Nikolaos, Vlaikidis, Nikolaos, Koutlas, Evaggelos, Kyritsis, Athanassios, Papathanassopoulos, Panagiotis, Makris, Nikolaos, Tavernarakis, Antonios, Scarpini, Elio, Montanari, Enrico, Marrosu, Maria Giovanna, Trojano, Maria, Amato, Maria Pia, Rottoli, Mariarosa, Lugaresi, Alessandra, Florio, Ciro, Gasperini, Claudio, Grimaldi, Luigi, Millefiorini, Enrico, Koudriavtseva, Tatiana, Perla, Franco, Mantegazza, Renato, Bertolotto, Antonio, Ghezzi, Angelo, Aguilar, Sandra Quinones, Eisenberg, Eli Skromne, Lopez, Leondardo Llamas, Estudillo, Rocio Marquez, Schrijver, H.M., Wittebol, M.C., Baart, J.C., van Golde, A.E.L., Hengstman, G.J.D., Pop, P.H.M., Bos (Geldrop), M., Medaer, R., Schyns-Soeterboek, Angelique, van der Zwart, A., van Diepen, A.J.H., Verheul, G.A.M., Verhagen, W.I.M., Bos (Helmond), M., Witjes, R.J.G.M., Sinnige, L.G.F., van Munster, E.Th.L., Sanders, E.A.C.M., van Dijl, Ron, Hupperts, R.M.M., Frequin, S.T.F.M., Visser, L.H., Henselmans, J.M.L., Moll, J.W.B., Midgard, Rune, Myhr, Kjell Morten, Edland, Astrid, Telstad, Wenche, Hognestad, Tone, Lund, Christian, Hovdal, Harald, Kamaljit, Kaur, Schepel, Jan, Hogenesch, Roelfien Ida, Schüler, Stephan, Odeh, Francis, Alstadhaug, Karl B., Korsgaard, Olav, Farbu, Elisabeth, Ingvaldsen, Teis Barclay, Soares (SCO), Diana, Rente, José, Guerra, José Manuel Costa, Morganho, Armando, Leitão, António, de Sá, João, Sá, Maria José, Marques, Pinto, Veloso, Mário, Baptista, Miguel Viana, Szilasiová, Jarmila, Copikova-Cudrakova, Daniela, Prochazkova, Lubica, Klimová, Eleonóra, Donath, Vladimir, Brozman, Miroslav, Ramo, Cristina, Ruiz, Domingo Pérez, Hernández, Carmen Calles, Sola, María Eugenia Marzo, Moro, Roberto Suarez, Vidal, Jose Antonio, Rodríguez, Ana Belén Caminero, Ozaeta, Gisela Martin, Nadal, Jordi Batlle, Esquide, Amaya Alvarez de Arcaya, Urtaza, Javier Olascoaga, Martínez-Yélamos, Sergio, Arbizu, Txomin, Torrenta, Lluis Ramio i, Boggild, Mike, Wilson, Martin, Al-Araji, Adnan, Nicholas, Richard, Harrower, Timothy, Redmond, Ian, Wolf, Tilo, Osei-Bonsu, Michael, Mazibrada, Gordon, Rog, David, Cottrell, David, Constantinescu, Cris, Gray, Orla, Belhag, Mohamed, Shehu, Abdullah, Rashid, Waqar, Duddy, Martin, Kappos, Ludwig, Wiendl, Heinz, Spelman, Tim, Pellegrini, Fabio, Chen, Yi, Dong, Qunming, and Koendgen, Harold

Published
2018
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17. The professional practice and training of neurology in the Asian and Oceanian Region: A cross-sectional survey by the Asian and Oceanian Association of Neurology (AOAN)

Author

Roxas, Artemio, Jr, Mehndiratta, Man Mohan, Bornstein, Natan, Macdonell, Richard, Lim, Kheng Seang, Ng, Ping-Wing, Dashzeveg, Shuren, Mizusawa, Hidehiro, Esmatullah, Hamed, Wu, Shey Lin, Chen, Christopher, Kurniawan, Mohammad, Rha, Joung-Ho, Wasay, Mohammad, Poungvarin, Niphon, Gunatilake, Saman, and Thang, Nguyen Huy

Published
2017
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18. The risk of secondary progressive multiple sclerosis is geographically determined but modifiable

Author

Sharmin, Sifat, primary, Roos, Izanne, additional, Simpson-Yap, Steve, additional, Malpas, Charles, additional, Sánchez, Marina M, additional, Ozakbas, Serkan, additional, Horakova, Dana, additional, Havrdova, Eva K, additional, Patti, Francesco, additional, Alroughani, Raed, additional, Izquierdo, Guillermo, additional, Eichau, Sara, additional, Boz, Cavit, additional, Zakaria, Magd, additional, Onofrj, Marco, additional, Lugaresi, Alessandra, additional, Weinstock-Guttman, Bianca, additional, Prat, Alexandre, additional, Girard, Marc, additional, Duquette, Pierre, additional, Terzi, Murat, additional, Amato, Maria Pia, additional, Karabudak, Rana, additional, Grand’Maison, Francois, additional, Khoury, Samia J, additional, Grammond, Pierre, additional, Lechner-Scott, Jeannette, additional, Buzzard, Katherine, additional, Skibina, Olga, additional, van der Walt, Anneke, additional, Butzkueven, Helmut, additional, Turkoglu, Recai, additional, Altintas, Ayse, additional, Maimone, Davide, additional, Kermode, Allan, additional, Shalaby, Nevin, additional, Pesch, Vincent V, additional, Butler, Ernest, additional, Sidhom, Youssef, additional, Gouider, Riadh, additional, Mrabet, Saloua, additional, Gerlach, Oliver, additional, Soysal, Aysun, additional, Barnett, Michael, additional, Kuhle, Jens, additional, Hughes, Stella, additional, Sa, Maria J, additional, Hodgkinson, Suzanne, additional, Oreja-Guevara, Celia, additional, Ampapa, Radek, additional, Petersen, Thor, additional, Ramo-Tello, Cristina, additional, Spitaleri, Daniele, additional, McCombe, Pamela, additional, Taylor, Bruce, additional, Prevost, Julie, additional, Foschi, Matteo, additional, Slee, Mark, additional, McGuigan, Chris, additional, Laureys, Guy, additional, Hijfte, Liesbeth V, additional, de Gans, Koen, additional, Solaro, Claudio, additional, Oh, Jiwon, additional, Macdonell, Richard, additional, Aguera-Morales, Eduardo, additional, Singhal, Bhim, additional, Gray, Orla, additional, Garber, Justin, additional, Wijmeersch, Bart V, additional, Simu, Mihaela, additional, Castillo-Triviño, Tamara, additional, Sanchez-Menoyo, Jose L, additional, Khurana, Dheeraj, additional, Al-Asmi, Abdullah, additional, Al-Harbi, Talal, additional, Deri, Norma, additional, Fragoso, Yara, additional, Lalive, Patrice H, additional, Sinnige, L G F, additional, Shaw, Cameron, additional, Shuey, Neil, additional, Csepany, Tunde, additional, Sempere, Angel P, additional, Moore, Fraser, additional, Decoo, Danny, additional, Willekens, Barbara, additional, Gobbi, Claudio, additional, Massey, Jennifer, additional, Hardy, Todd, additional, Parratt, John, additional, and Kalincik, Tomas, additional

Published
2023
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19. Long-Term Safety and Effectiveness of Delayed-release Dimethyl Fumarate in Multiple Sclerosis Patients Treated in Routine Medical Practice, an updated analysis of the ESTEEM Study (P7-3.009)

Author

Pandey, Krupa, primary, Giles, Kathryn, additional, Balashov, Konstantin, additional, Macdonell, Richard, additional, Windsheimer, Jorg, additional, Martinez, Mikel, additional, Bozin, Ivan, additional, Jiang, Xiaotong, additional, Lyons, Jennifer, additional, Mokliatchouk, Oksana, additional, and Okai, Annette, additional

Published
2023
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20. Disability accrual in primary and secondary progressive multiple sclerosis

Author

Harding-Forrester, Sam, primary, Roos, Izanne, additional, Nguyen, Ai-Lan, additional, Malpas, Charles B, additional, Diouf, Ibrahima, additional, Moradi, Nahid, additional, Sharmin, Sifat, additional, Izquierdo, Guillermo, additional, Eichau, Sara, additional, Patti, Francesco, additional, Horakova, Dana, additional, Kubala Havrdova, Eva, additional, Prat, Alexandre, additional, Girard, Marc, additional, Duquette, Pierre, additional, Grand'Maison, Francois, additional, Onofrj, Marco, additional, Lugaresi, Alessandra, additional, Grammond, Pierre, additional, Ozakbas,, Serkan, additional, Amato, Maria Pia, additional, Gerlach, Oliver, additional, Sola, Patrizia, additional, Ferraro, Diana, additional, Buzzard, Katherine, additional, Skibina, Olga, additional, Lechner-Scott, Jeannette, additional, Alroughani, Raed, additional, Boz, Cavit, additional, Van Pesch, Vincent, additional, Cartechini, Elisabetta, additional, Terzi, Murat, additional, Maimone, Davide, additional, Ramo-Tello, Cristina, additional, Yamout, Bassem, additional, Khoury, Samia Joseph, additional, La Spitaleri, Daniele, additional, Sa, Maria Jose, additional, Blanco, Yolanda, additional, Granella, Franco, additional, Slee, Mark, additional, Butler, Ernest, additional, Sidhom, Youssef, additional, Gouider, Riadh, additional, Bergamaschi, Roberto, additional, Karabudak, Rana, additional, Ampapa, Radek, additional, Sánchez-Menoyo, José Luis, additional, Prevost, Julie, additional, Castillo-Trivino, Tamara, additional, McCombe, Pamela A, additional, Macdonell, Richard, additional, Laureys, Guy, additional, Van Hijfte, Liesbeth, additional, Oh, Jiwon, additional, Altintas, Ayse, additional, de Gans, Koen, additional, Turkoglu, Recai, additional, van der Walt, Anneke, additional, Butzkueven, Helmut, additional, Vucic, Steve, additional, Barnett, Michael, additional, Cristiano, Edgardo, additional, Hodgkinson, Suzanne, additional, Iuliano, Gerardo, additional, Kappos, Ludwig, additional, Kuhle, Jens, additional, Shaygannejad, Vahid, additional, Soysal, Aysun, additional, Weinstock-Guttman, Bianca, additional, Van Wijmeersch, Bart, additional, and Kalincik, Tomas, additional

Published
2023
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21. Early non-disabling relapses are important predictors of disability accumulation in people with relapsing-remitting multiple sclerosis

Author

Daruwalla, Cyrus, primary, Shaygannejad, Vahid, additional, Ozakbas, Serkan, additional, Havrdova, Eva Kubala, additional, Horakova, Dana, additional, Alroughani, Raed, additional, Boz, Cavit, additional, Patti, Francesco, additional, Onofrj, Marco, additional, Lugaresi, Alessandra, additional, Eichau, Sara, additional, Girard, Marc, additional, Prat, Alexandre, additional, Duquette, Pierre, additional, Yamout, Bassem, additional, Khoury, Samia J, additional, Sajedi, Seyed Aidin, additional, Turkoglu, Recai, additional, Altintas, Ayse, additional, Skibina, Olga, additional, Buzzard, Katherine, additional, Grammond, Pierre, additional, Karabudak, Rana, additional, van der Walt, Anneke, additional, Butzkueven, Helmut, additional, Maimone, Davide, additional, Lechner-Scott, Jeannette, additional, Soysal, Aysun, additional, John, Nevin, additional, Prevost, Julie, additional, Spitaleri, Daniele, additional, Ramo-Tello, Cristina, additional, Gerlach, Oliver, additional, Iuliano, Gerardo, additional, Foschi, Matteo, additional, Ampapa, Radek, additional, van Pesch, Vincent, additional, Barnett, Michael, additional, Shalaby, Nevin, additional, D’hooghe, Marie, additional, Kuhle, Jens, additional, Sa, Maria Jose, additional, Fabis-Pedrini, Marzena, additional, Kermode, Allan, additional, Mrabet, Saloua, additional, Gouider, Riadh, additional, Hodgkinson, Suzanne, additional, Laureys, Guy, additional, Van Hijfte, Liesbeth, additional, Macdonell, Richard, additional, Oreja-Guevara, Celia, additional, Cristiano, Edgardo, additional, McCombe, Pamela, additional, Sanchez-Menoyo, Jose Luis, additional, Singhal, Bhim, additional, Blanco, Yolanda, additional, Hughes, Stella, additional, Garber, Justin, additional, Solaro, Claudio, additional, McGuigan, Chris, additional, Taylor, Bruce, additional, de Gans, Koen, additional, Habek, Mario, additional, Al-Asmi, Abdullah, additional, Mihaela, Simu, additional, Castillo Triviño, Tamara, additional, Al-Harbi, Talal, additional, Rojas, Juan Ignacio, additional, Gray, Orla, additional, Khurana, Dheeraj, additional, Van Wijmeersch, Bart, additional, Grigoriadis, Nikolaos, additional, Inshasi, Jihad, additional, Oh, Jiwon, additional, Aguera-Morales, Eduardo, additional, Fragoso, Yara, additional, Moore, Fraser, additional, Shaw, Cameron, additional, Baghbanian, Seyed Mohammad, additional, Shuey, Neil, additional, Willekens, Barbara, additional, Hardy, Todd A, additional, Decoo, Danny, additional, sempere, Angel Perez, additional, Field, Deborah, additional, Wynford-Thomas, Ray, additional, Cunniffe, Nick G, additional, Roos, Izanne, additional, Malpas, Charles B, additional, Coles, Alasdair J, additional, Kalincik, Tomas, additional, and Brown, J William L, additional

Published
2023
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22. Variability of the response to immunotherapy among subgroups of patients with multiple sclerosis

Author

Diouf, Ibrahima, primary, Malpas, Charles B., additional, Sharmin, Sifat, additional, Roos, Izanne, additional, Horakova, Dana, additional, Havrdova, Eva Kubala, additional, Patti, Francesco, additional, Shaygannejad, Vahid, additional, Ozakbas, Serkan, additional, Izquierdo, Guillermo, additional, Eichau, Sara, additional, Onofrj, Marco, additional, Lugaresi, Alessandra, additional, Alroughani, Raed, additional, Prat, Alexandre, additional, Girard, Marc, additional, Duquette, Pierre, additional, Terzi, Murat, additional, Boz, Cavit, additional, Grand'Maison, Francois, additional, Hamdy, Sherif, additional, Sola, Patrizia, additional, Ferraro, Diana, additional, Grammond, Pierre, additional, Turkoglu, Recai, additional, Buzzard, Katherine, additional, Skibina, Olga, additional, Yamout, Bassem, additional, Altintas, Ayse, additional, Gerlach, Oliver, additional, van Pesch, Vincent, additional, Blanco, Yolanda, additional, Maimone, Davide, additional, Lechner‐Scott, Jeannette, additional, Bergamaschi, Roberto, additional, Karabudak, Rana, additional, Iuliano, Gerardo, additional, McGuigan, Chris, additional, Cartechini, Elisabetta, additional, Barnett, Michael, additional, Hughes, Stella, additional, Sa, Maria José, additional, Solaro, Claudio, additional, Kappos, Ludwig, additional, Ramo‐Tello, Cristina, additional, Cristiano, Edgardo, additional, Hodgkinson, Suzanne, additional, Spitaleri, Daniele, additional, Soysal, Aysun, additional, Petersen, Thor, additional, Slee, Mark, additional, Butler, Ernest, additional, Granella, Franco, additional, de Gans, Koen, additional, McCombe, Pamela, additional, Ampapa, Radek, additional, Van Wijmeersch, Bart, additional, van der Walt, Anneke, additional, Butzkueven, Helmut, additional, Prevost, Julie, additional, Sinnige, L. G. F., additional, Sanchez‐Menoyo, Jose Luis, additional, Vucic, Steve, additional, Laureys, Guy, additional, Van Hijfte, Liesbeth, additional, Khurana, Dheeraj, additional, Macdonell, Richard, additional, Gouider, Riadh, additional, Castillo‐Triviño, Tamara, additional, Gray, Orla, additional, Aguera‐Morales, Eduardo, additional, Al‐Asmi, Abdullah, additional, Shaw, Cameron, additional, Deri, Norma, additional, Al‐Harbi, Talal, additional, Fragoso, Yara, additional, Csepany, Tunde, additional, Perez Sempere, Angel, additional, Trevino‐Frenk, Irene, additional, Schepel, Jan, additional, Moore, Fraser, additional, and Kalincik, Tomas, additional

Published
2023
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23. Variability of the response to immunotherapy among subgroups of patients with multiple sclerosis

Author

Altıntaş, Ayşe (ORCID 0000-0002-8524-5087 & YÖK ID 11611), Diouf, Ibrahima; Malpas, Charles B.; Sharmin, Sifat; Roos, Izanne; Horakova, Dana; Havrdova, Eva Kubala; Patti, Francesco; Shaygannejad, Vahid; Ozakbas, Serkan; Izquierdo, Guillermo; Eichau, Sara; Onofrj, Marco; Lugaresi, Alessandra; Alroughani, Raed; Prat, Alexandre; Girard, Marc; Duquette, Pierre; Terzi, Murat; Boz, Cavit; Grand'Maison, Francois; Hamdy, Sherif; Sola, Patrizia; Ferraro, Diana; Grammond, Pierre; Turkoglu, Recai; Buzzard, Katherine; Skibina, Olga; Yamout, Bassem; Gerlach, Oliver; van Pesch, Vincent; Blanco, Yolanda; Maimone, Davide; Lechner-Scott, Jeannette; Bergamaschi, Roberto; Karabudak, Rana; Iuliano, Gerardo; McGuigan, Chris; Cartechini, Elisabetta; Barnett, Michael; Hughes, Stella; Sa, Maria Jose; Solaro, Claudio; Kappos, Ludwig; Ramo-Tello, Cristina; Cristiano, Edgardo; Hodgkinson, Suzanne; Spitaleri, Daniele; Soysal, Aysun; Petersen, Thor; Slee, Mark; Butler, Ernest; Granella, Franco; de Gans, Koen; McCombe, Pamela; Ampapa, Radek; Van Wijmeersch, Bart; van der Walt, Anneke; Butzkueven, Helmut; Prevost, Julie; Sinnige, L. G. F.; Sanchez-Menoyo, Jose Luis; Vucic, Steve; Laureys, Guy; Van Hijfte, Liesbeth; Khurana, Dheeraj; Macdonell, Richard; Gouider, Riadh; Castillo-Trivino, Tamara; Gray, Orla; Aguera-Morales, Eduardo; Al-Asmi, Abdullah; Shaw, Cameron; Deri, Norma; Al-Harbi, Talal; Fragoso, Yara; Csepany, Tunde; Sempere, Angel Perez; Trevino-Frenk, Irene; Schepel, Jan; Moore, Fraser; Kalincik, Tomas, Koç University Research Center for Translational Medicine (KUTTAM) / Koç Üniversitesi Translasyonel Tıp Araştırma Merkezi (KUTTAM), School of Medicine, Altıntaş, Ayşe (ORCID 0000-0002-8524-5087 & YÖK ID 11611), Diouf, Ibrahima; Malpas, Charles B.; Sharmin, Sifat; Roos, Izanne; Horakova, Dana; Havrdova, Eva Kubala; Patti, Francesco; Shaygannejad, Vahid; Ozakbas, Serkan; Izquierdo, Guillermo; Eichau, Sara; Onofrj, Marco; Lugaresi, Alessandra; Alroughani, Raed; Prat, Alexandre; Girard, Marc; Duquette, Pierre; Terzi, Murat; Boz, Cavit; Grand'Maison, Francois; Hamdy, Sherif; Sola, Patrizia; Ferraro, Diana; Grammond, Pierre; Turkoglu, Recai; Buzzard, Katherine; Skibina, Olga; Yamout, Bassem; Gerlach, Oliver; van Pesch, Vincent; Blanco, Yolanda; Maimone, Davide; Lechner-Scott, Jeannette; Bergamaschi, Roberto; Karabudak, Rana; Iuliano, Gerardo; McGuigan, Chris; Cartechini, Elisabetta; Barnett, Michael; Hughes, Stella; Sa, Maria Jose; Solaro, Claudio; Kappos, Ludwig; Ramo-Tello, Cristina; Cristiano, Edgardo; Hodgkinson, Suzanne; Spitaleri, Daniele; Soysal, Aysun; Petersen, Thor; Slee, Mark; Butler, Ernest; Granella, Franco; de Gans, Koen; McCombe, Pamela; Ampapa, Radek; Van Wijmeersch, Bart; van der Walt, Anneke; Butzkueven, Helmut; Prevost, Julie; Sinnige, L. G. F.; Sanchez-Menoyo, Jose Luis; Vucic, Steve; Laureys, Guy; Van Hijfte, Liesbeth; Khurana, Dheeraj; Macdonell, Richard; Gouider, Riadh; Castillo-Trivino, Tamara; Gray, Orla; Aguera-Morales, Eduardo; Al-Asmi, Abdullah; Shaw, Cameron; Deri, Norma; Al-Harbi, Talal; Fragoso, Yara; Csepany, Tunde; Sempere, Angel Perez; Trevino-Frenk, Irene; Schepel, Jan; Moore, Fraser; Kalincik, Tomas, Koç University Research Center for Translational Medicine (KUTTAM) / Koç Üniversitesi Translasyonel Tıp Araştırma Merkezi (KUTTAM), and School of Medicine

Abstract

Background and purpose: This study assessed the effect of patient characteristics on the response to disease-modifying therapy (DMT) in multiple sclerosis (MS). Methods: We extracted data from 61,810 patients from 135 centers across 35 countries from the MSBase registry. The selection criteria were: clinically isolated syndrome or definite MS, follow-up ? 1 year, and Expanded Disability Status Scale (EDSS) score ? 3, with ?1 score recorded per year. Marginal structural models with interaction terms were used to compare the hazards of 12-month confirmed worsening and improvement of disability, and the incidence of relapses between treated and untreated patients stratified by their characteristics. Results: Among 24,344 patients with relapsing MS, those on DMTs experienced 48% reduction in relapse incidence (hazard ratio [HR] = 0.52, 95% confidence interval [CI] = 0.45–0.60), 46% lower risk of disability worsening (HR = 0.54, 95% CI = 0.41–0.71), and 32% greater chance of disability improvement (HR = 1.32, 95% CI = 1.09–1.59). The effect of DMTs on EDSS worsening and improvement and the risk of relapses was attenuated with more severe disability. The magnitude of the effect of DMT on suppressing relapses declined with higher prior relapse rate and prior cerebral magnetic resonance imaging activity. We did not find any evidence for the effect of age on the effectiveness of DMT. After inclusion of 1985 participants with progressive MS, the effect of DMT on disability mostly depended on MS phenotype, whereas its effect on relapses was driven mainly by prior relapse activity. Conclusions: DMT is generally most effective among patients with lower disability and in relapsing MS phenotypes. There is no evidence of attenuation of the effect of DMT with age., This study was financially supported by the National Health and Medical Research Council of Australia (1129189, 1140766, 1080518).

Published
2023

25. Comparative effectiveness of cladribine tablets versus other oral disease-modifying treatments for multiple sclerosis: Results from MSBase registry

Author

Spelman, Tim, primary, Ozakbas, Serkan, additional, Alroughani, Raed, additional, Terzi, Murat, additional, Hodgkinson, Suzanne, additional, Laureys, Guy, additional, Kalincik, Tomas, additional, Van Der Walt, Anneke, additional, Yamout, Bassem, additional, Lechner-Scott, Jeannette, additional, Soysal, Aysun, additional, Kuhle, Jens, additional, Sanchez-Menoyo, Jose Luis, additional, Blanco Morgado, Yolanda, additional, Spitaleri, Daniele LA, additional, van Pesch, Vincent, additional, Horakova, Dana, additional, Ampapa, Radek, additional, Patti, Francesco, additional, Macdonell, Richard, additional, Al-Asmi, Abdullah, additional, Gerlach, Oliver, additional, Oh, Jiwon, additional, Altintas, Ayse, additional, Tundia, Namita, additional, Wong, Schiffon L, additional, and Butzkueven, Helmut, additional

Published
2022
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26. Comparative effectiveness of autologous hematopoietic stem cell transplant vs Fingolimod, Natalizumab, and Ocrelizumab in highly active relapsing-remitting multiple sclerosis

Author

Kalincik, Tomas, Sharmin, Sifat, Roos, Izanne, Freedman, Mark S., Atkins, Harold, Burman, Joachim, Massey, Jennifer, Sutton, Ian, Withers, Barbara, Macdonell, Richard, Grigg, Andrew, Torkildsen, Øivind, Bo, Lars, Lehmann, Anne Kristine, Havrdova, Eva Kubala, Krasulova, Eva, Trněný, Marek, Kozak, Tomas, van der Walt, Anneke, Butzkueven, Helmut, McCombe, Pamela, Skibina, Olga, Lechner-Scott, Jeannette, Willekens, Barbara, Cartechini, Elisabetta, Ozakbas, Serkan, Alroughani, Raed, Kuhle, Jens, Patti, Francesco, Duquette, Pierre, Lugaresi, Alessandra, Khoury, Samia J., Slee, Mark, Turkoglu, Recai, Hodgkinson, Suzanne, John, Nevin, Maimone, Davide, Sa, Maria Jose, van Pesch, Vincent, Gerlach, Oliver, Laureys, Guy, Van Hijfte, Liesbeth, Karabudak, Rana, Spitaleri, Daniele, Csepany, Tunde, Gouider, Riadh, Castillo-Triviño, Tamara, Taylor, Bruce, Sharrack, Basil, Snowden, John A., Horakova, Dana, Buzzard, Katherine, Terzi, Murat, Prat, Alexandre, Girard, Marc, Grammond, Pierre, Barnett, Michael, Stewart, Grace, Onofrj, Marco, Izquierdo, Guillermo, Eichau, Sara, Grand'Maison, Francois, Prevost, Julie, Van Wijmeersch, Bart, Amato, Maria Pia, Shaygannejad, Vahid, Boz, Cavit, Bolaños, Ricardo Fernandez, Soysal, Aysun, Ramo-Tello, Cristina, Solaro, Claudio, Gobbi, Claudio, Cabrera-Gomez, Jose Antonio, Roullet, Etienne, Zwanikken, Cees, Den braber-Moerland, Leontien, Deri, Norma, Saladino, Maria Laura, Cristiano, Edgardo, Rojas, Juan Ignacio, Vrech, Carlos, Shaw, Cameron, Shuey, Neil, Boggild, Mike, Tan, Ik Lin, Hardy, Todd, Decoo, Danny, Moore, Fraser, Oh, Jiwon, Lalive, Patrice, Ampapa, Radek, Petersen, Thor, Oreja-Guevara, Celia, Perez Sempere, Angel, Dominguez, Jose Andres, Besora, Sarah, Hughes, Stella, Gray, Orla, Grigoriadis, Nikolaos, Piroska, Imre, Rozsa, Csilla, Kasa, Krisztian, Simo, Magdolna, Kovacs, Krisztina, Sas, Attila, Dobos, Eniko, Rajda, Cecilia, McGuigan, Chris, Mason, Deborah, Schepel, Jan, Alkhaboori, Jabir, Rio, Maria Edite, Mihaela, Simu, Al-Harbi, Talal, Altintas, Ayse, Kister, Ilya, Marriott, Mark, Kilpatrick, Trevor, King, John, Nguyen, Ai-Lan, Dwyer, Chris, Monif, Mastura, Taylor, Lisa, Diamanti, Matteo, Chisari, Clara, Toscano, Simona, Salvatore, Lo Fermo, Larochelle, Catherine, De Luca, Giovanna, Di Tommaso, Valeria, Travaglini, Daniela, Pietrolongo, Erika, di Ioia, Maria, Farina, Deborah, Mancinelli, Luca, Hupperts, Raymond, Olascoaga, Javier, Saiz, Albert, Zivadinov, Robert, Benedict, Ralph, Verheul, Freek, Fabis-Pedrini, Marzena, Mrabet, Saloua, Garber, Justin, Sanchez-Menoyo, Jose Luis, Aguera-Morales, Eduardo, Blanco, Yolanda, Al-Asmi, Abdullah, Weinstock-Guttman, Bianca, Fragoso, Yara, de Gans, Koen, and Kermode, Allan

Subjects
Human medicine
Abstract

you are agreeing to our Cookie Policy | Continue JAMA Network HomeJAMA Neurology This Issue Views 2,357 Citations 0 60 Full Text Share Comment Original Investigation May 15, 2023 Comparative Effectiveness of Autologous Hematopoietic Stem Cell Transplant vs Fingolimod, Natalizumab, and Ocrelizumab in Highly Active Relapsing-Remitting Multiple Sclerosis Tomas Kalincik, MD, PhD1,2; Sifat Sharmin, PhD1,2; Izanne Roos, MBChB, PhD1,2; Mark S. Freedman, MD3; Harold Atkins, MD4; Joachim Burman, MD, PhD5; Jennifer Massey, MBBS, PhD6,7; Ian Sutton, MBBS, PhD6,8; Barbara Withers, MD, PhD7,9; Richard Macdonell, MD, PhD10,11; Andrew Grigg, MD, PhD11,12; Øivind Torkildsen, MD, PhD13; Lars Bo, MD, PhD13; Anne Kristine Lehmann, MD, PhD14; Eva Kubala Havrdova, MD, PhD15; Eva Krasulova, MD, PhD15; Marek Trněný, MD, PhD16; Tomas Kozak, MD, PhD17; Anneke van der Walt, MBBS, PhD18,19; Helmut Butzkueven, MBBS, PhD18,19; Pamela McCombe, MBBS20,21; Olga Skibina, MBBS18,22,23; Jeannette Lechner-Scott, MD, PhD24,25; Barbara Willekens, MD, PhD26,27; Elisabetta Cartechini, MD28; Serkan Ozakbas, MD29; Raed Alroughani, MD30; Jens Kuhle, MD, PhD31; Francesco Patti, MD32,33; Pierre Duquette, MD34; Alessandra Lugaresi, MD, PhD35,36; Samia J. Khoury, MD, PhD37; Mark Slee, MD, PhD38; Recai Turkoglu, MD39; Suzanne Hodgkinson, MD40; Nevin John, MD, PhD41,42; Davide Maimone, MD43; Maria Jose Sa, MD44; Vincent van Pesch, MD, PhD45,46; Oliver Gerlach, MD, PhD47,48; Guy Laureys, MD49; Liesbeth Van Hijfte, MD49; Rana Karabudak, MD50; Daniele Spitaleri, MD51; Tunde Csepany, MD, PhD52; Riadh Gouider, MD53,54; Tamara Castillo-Triviño, MD55; Bruce Taylor, MD, PhD56,57; Basil Sharrack, MD, PhD58; John A. Snowden, MD, PhD59; and the MSBase Study Group Collaborators; and the MSBase Study Group Authors Author Affiliations JAMA Neurol. 2023;80(7):702-713. doi:10.1001/jamaneurol.2023.1184 editorial comment iconEditorial Comment Key Points Question What is the comparative effectiveness of autologous hematopoietic stem cell transplant (AHSCT) vs individual most potent disease-modifying therapies for relapsing-remitting multiple sclerosis (MS), such as natalizumab or ocrelizumab? Findings In this observational comparative effectiveness study of 4915 individuals using a composite cohort from specialized MS centers and the MSBase international registry, the effectiveness of AHSCT was compared with 1 medium-efficacy and 2 high-efficacy disease-modifying therapies (fingolimod, natalizumab, and ocrelizumab) in patients with relapsing-remitting MS, high frequency of relapses, and moderate disability. Over 5 years, AHSCT was associated with substantially lower relapse rate than fingolimod and marginally lower relapse rate than natalizumab and was also associated with a higher rate of recovery from disability compared with fingolimod and natalizumab, but no evidence of difference in clinical outcomes between AHSCT and ocrelizumab was found at 3-year follow-up. Meaning The results indicate that in relapsing-remitting MS, the clinical effectiveness of AHSCT is considerably superior to fingolimod and marginally superior to natalizumab. Abstract Importance Autologous hematopoietic stem cell transplant (AHSCT) is available for treatment of highly active multiple sclerosis (MS). Objective To compare the effectiveness of AHSCT vs fingolimod, natalizumab, and ocrelizumab in relapsing-remitting MS by emulating pairwise trials. Design, Setting, and Participants This comparative treatment effectiveness study included 6 specialist MS centers with AHSCT programs and international MSBase registry between 2006 and 2021. The study included patients with relapsing-remitting MS treated with AHSCT, fingolimod, natalizumab, or ocrelizumab with 2 or more years study follow-up including 2 or more disability assessments. Patients were matched on a propensity score derived from clinical and demographic characteristics. Exposure AHSCT vs fingolimod, natalizumab, or ocrelizumab. Main outcomes Pairwise-censored groups were compared on annualized relapse rates (ARR) and freedom from relapses and 6-month confirmed Expanded Disability Status Scale (EDSS) score worsening and improvement. Results Of 4915 individuals, 167 were treated with AHSCT; 2558, fingolimod; 1490, natalizumab; and 700, ocrelizumab. The prematch AHSCT cohort was younger and with greater disability than the fingolimod, natalizumab, and ocrelizumab cohorts; the matched groups were closely aligned. The proportion of women ranged from 65% to 70%, and the mean (SD) age ranged from 35.3 (9.4) to 37.1 (10.6) years. The mean (SD) disease duration ranged from 7.9 (5.6) to 8.7 (5.4) years, EDSS score ranged from 3.5 (1.6) to 3.9 (1.9), and frequency of relapses ranged from 0.77 (0.94) to 0.86 (0.89) in the preceding year. Compared with the fingolimod group (769 [30.0%]), AHSCT (144 [86.2%]) was associated with fewer relapses (ARR: mean [SD], 0.09 [0.30] vs 0.20 [0.44]), similar risk of disability worsening (hazard ratio [HR], 1.70; 95% CI, 0.91-3.17), and higher chance of disability improvement (HR, 2.70; 95% CI, 1.71-4.26) over 5 years. Compared with natalizumab (730 [49.0%]), AHSCT (146 [87.4%]) was associated with marginally lower ARR (mean [SD], 0.08 [0.31] vs 0.10 [0.34]), similar risk of disability worsening (HR, 1.06; 95% CI, 0.54-2.09), and higher chance of disability improvement (HR, 2.68; 95% CI, 1.72-4.18) over 5 years. AHSCT (110 [65.9%]) and ocrelizumab (343 [49.0%]) were associated with similar ARR (mean [SD], 0.09 [0.34] vs 0.06 [0.32]), disability worsening (HR, 1.77; 95% CI, 0.61-5.08), and disability improvement (HR, 1.37; 95% CI, 0.66-2.82) over 3 years. AHSCT-related mortality occurred in 1 of 159 patients (0.6%). Conclusion In this study, the association of AHSCT with preventing relapses and facilitating recovery from disability was considerably superior to fingolimod and marginally superior to natalizumab. This study did not find evidence for difference in the effectiveness of AHSCT and ocrelizumab over a shorter available follow-up time.

Published
2023

27. Comparison Between Dimethyl Fumarate, Fingolimod, and Ocrelizumab After Natalizumab Cessation

Author

Zhu, Chao, Kalincik, Tomas, Horakova, Dana, Zhou, Zhen, Buzzard, Katherine, Skibina, Olga, Alroughani, Raed, Izquierdo, Guillermo, Eichau, Sara, Kuhle, Jens, Patti, Francesco, Grand'Maison, Francois, Hodgkinson, Suzanne, Grammond, Pierre, Lechner-Scott, Jeannette, Butler, Ernest, Prat, Alexandre, Girard, Marc, Duquette, Pierre, Macdonell, Richard A L, Weinstock-Guttman, Bianca, Ozakbas, Serkan, Slee, Mark, Sa, Maria Jose, Van Pesch, Vincent, Barnett, Michael, Van Wijmeersch, Bart, Gerlach, Oliver, Prevost, Julie, Terzi, Murat, Boz, Cavit, Laureys, Guy, Van Hijfte, Liesbeth, Kermode, Allan G, Garber, Justin, Yamout, Bassem, Khoury, Samia J, Merlo, Daniel, Monif, Mastura, Jokubaitis, Vilija, van der Walt, Anneke, Butzkueven, Helmut, MSBase Study Group, and UCL - SSS/IONS/CEMO - Pôle Cellulaire et moléculaire

Abstract

Natalizumab cessation is associated with a risk of rebound disease activity. It is important to identify the optimal switch disease-modifying therapy strategy after natalizumab to limit the risk of severe relapses. To compare the effectiveness and persistence of dimethyl fumarate, fingolimod, and ocrelizumab among patients with relapsing-remitting multiple sclerosis (RRMS) who discontinued natalizumab. In this observational cohort study, patient data were collected from the MSBase registry between June 15, 2010, and July 6, 2021. The median follow-up was 2.7 years. This was a multicenter study that included patients with RRMS who had used natalizumab for 6 months or longer and then were switched to dimethyl fumarate, fingolimod, or ocrelizumab within 3 months after natalizumab discontinuation. Patients without baseline data were excluded from the analysis. Data were analyzed from May 24, 2022, to January 9, 2023. Dimethyl fumarate, fingolimod, and ocrelizumab. Primary outcomes were annualized relapse rate (ARR) and time to first relapse. Secondary outcomes were confirmed disability accumulation, disability improvement, and subsequent treatment discontinuation, with the comparisons for the first 2 limited to fingolimod and ocrelizumab due to the small number of patients taking dimethyl fumarate. The associations were analyzed after balancing covariates using an inverse probability of treatment weighting method. Among 66 840 patients with RRMS, 1744 had used natalizumab for 6 months or longer and were switched to dimethyl fumarate, fingolimod, or ocrelizumab within 3 months of natalizumab discontinuation. After excluding 358 patients without baseline data, a total of 1386 patients (mean [SD] age, 41.3 [10.6] years; 990 female [71%]) switched to dimethyl fumarate (138 [9.9%]), fingolimod (823 [59.4%]), or ocrelizumab (425 [30.7%]) after natalizumab. The ARR for each medication was as follows: ocrelizumab, 0.06 (95% CI, 0.04-0.08); fingolimod, 0.26 (95% CI, 0.12-0.48); and dimethyl fumarate, 0.27 (95% CI, 0.12-0.56). The ARR ratio of fingolimod to ocrelizumab was 4.33 (95% CI, 3.12-6.01) and of dimethyl fumarate to ocrelizumab was 4.50 (95% CI, 2.89-7.03). Compared with ocrelizumab, the hazard ratio (HR) of time to first relapse was 4.02 (95% CI, 2.83-5.70) for fingolimod and 3.70 (95% CI, 2.35-5.84) for dimethyl fumarate. The HR of treatment discontinuation was 2.57 (95% CI, 1.74-3.80) for fingolimod and 4.26 (95% CI, 2.65-6.84) for dimethyl fumarate. Fingolimod use was associated with a 49% higher risk for disability accumulation compared with ocrelizumab. There was no significant difference in disability improvement rates between fingolimod and ocrelizumab. Study results show that among patients with RRMS who switched from natalizumab to dimethyl fumarate, fingolimod, or ocrelizumab, ocrelizumab use was associated with the lowest ARR and discontinuation rates, and the longest time to first relapse.

Published
2023

28. A plain language summary on the effectiveness of cladribine tablets compared with other oral treatments for multiple sclerosis: results from the MSBase registry

Author

Spelman, Tim, Ozakbas, Serkan, Alroughani, Raed, Terzi, Murat, Hodgkinson, Suzanne, Laureys, Guy, Kalincik, Tomas, Der Walt, Anneke Van, Yamout, Bassem, Lechner-Scott, Jeannette, Soysal, Aysun, Kuhle, Jens, Sanchez-Menoyo, Jose Luis, Morgado, Yolanda Blanco, Spitaleri, Daniele LA, Pesch, Vincent van, Horakova, Dana, Ampapa, Radek, Patti, Francesco, Macdonell, Richard, Al-Asmi, Abdullah, Gerlach, Oliver, Oh, Jiwon, Altintas, Ayse, Tundia, Namita, Wong, Schiffon L, Butzkueven, Helmut, MSBase Study Group, [missing], and UCL - SSS/IONS/CEMO - Pôle Cellulaire et moléculaire

Subjects
teriflunomide (Aubagio®), relapses, cladribine tablets, fingolimod (Gilenya®), Neurology (clinical), lay summary, dimethyl fumarate (Tecfidera®), multiple sclerosis, treatment options
Abstract

What is this summary about? Patient registries contain anonymous data from people who share the same medical condition. The MSBase registry contains information from over 80,000 people living with multiple sclerosis (MS) across 41 countries. Using information from the MSBase registry, the GLIMPSE (Generating Learnings In MultiPle SclErosis) study looked at real-life outcomes in 3475 people living with MS who were treated with cladribine tablets (Mavenclad®) compared with other oral treatments. What were the results? Results showed that people treated with cladribine tablets stayed on treatment for longer than other treatments given by mouth. They also had fewer relapses (also called flare ups of symptoms) than people who received a different oral treatment for their MS. What do the results mean? The results provide evidence that, compared with other oral treatments for MS, cladribine tablets are an effective medicine for people living with MS.

Published
2023

29. Disability accrual in primary and secondary progressive multiple sclerosis

Author

Harding-Forrester, Sam, Roos, Izanne, Nguyen, Ai-Lan, Malpas, Charles B, Diouf, Ibrahima, Moradi, Nahid, Sharmin, Sifat, Izquierdo, Guillermo, Eichau, Sara, Patti, Francesco, Horakova, Dana, Kubala Havrdova, Eva, Prat, Alexandre, Girard, Marc, Duquette, Pierre, Grand'Maison, Francois, Onofrj, Marco, Lugaresi, Alessandra, Grammond, Pierre, Ozakbas, Serkan, Amato, Maria Pia, Gerlach, Oliver, Sola, Patrizia, Ferraro, Diana, Buzzard, Katherine, Skibina, Olga, Lechner-Scott, Jeannette, Alroughani, Raed, Boz, Cavit, Van Pesch, Vincent, Cartechini, Elisabetta, Terzi, Murat, Maimone, Davide, Ramo-Tello, Cristina, Yamout, Bassem, Khoury, Samia Joseph, La Spitaleri, Daniele, Sa, Maria Jose, Blanco, Yolanda, Granella, Franco, Slee, Mark, Butler, Ernest, Sidhom, Youssef, Gouider, Riadh, Bergamaschi, Roberto, Karabudak, Rana, Ampapa, Radek, Sánchez-Menoyo, José Luis, Prevost, Julie, Castillo-Trivino, Tamara, McCombe, Pamela A, Macdonell, Richard, Laureys, Guy, Van Hijfte, Liesbeth, Oh, Jiwon, Altintas, Ayse, de Gans, Koen, Turkoglu, Recai, van der Walt, Anneke, Butzkueven, Helmut, Vucic, Steve, Barnett, Michael, Cristiano, Edgardo, Hodgkinson, Suzanne, Iuliano, Gerardo, Kappos, Ludwig, Kuhle, Jens, Shaygannejad, Vahid, Soysal, Aysun, Weinstock-Guttman, Bianca, Van Wijmeersch, Bart, Kalincik, Tomas, MSBase investigators, and UCL - SSS/IONS/CEMO - Pôle Cellulaire et moléculaire

Subjects
Psychiatry and Mental health, Surgery, Neurology (clinical), multiple sclerosis
Abstract

BackgroundSome studies comparing primary and secondary progressive multiple sclerosis (PPMS, SPMS) report similar ages at onset of the progressive phase and similar rates of subsequent disability accrual. Others report later onset and/or faster accrual in SPMS. Comparisons have been complicated by regional cohort effects, phenotypic differences in sex ratio and management and variable diagnostic criteria for SPMS.MethodsWe compared disability accrual in PPMS and operationally diagnosed SPMS in the international, clinic-based MSBase cohort. Inclusion required PPMS or SPMS with onset at age ≥18 years since 1995. We estimated Andersen-Gill hazard ratios for disability accrual on the Expanded Disability Status Scale (EDSS), adjusted for sex, age, baseline disability, EDSS score frequency and drug therapies, with centre and patient as random effects. We also estimated ages at onset of the progressive phase (Kaplan-Meier) and at EDSS milestones (Turnbull). Analyses were replicated with physician-diagnosed SPMS.ResultsIncluded patients comprised 1872 with PPMS (47% men; 50% with activity) and 2575 with SPMS (32% men; 40% with activity). Relative to PPMS, SPMS had older age at onset of the progressive phase (median 46.7 years (95% CI 46.2–47.3) vs 43.9 (43.3–44.4); pConclusionsWe demonstrate later onset of the progressive phase and slower disability accrual in SPMS versus PPMS. This may balance greater baseline disability in SPMS, yielding convergent disability trajectories across phenotypes. The different rates of disability accrual should be considered before amalgamating PPMS and SPMS in clinical trials.

Published
2023

30. sj-docx-1-msj-10.1177_13524585231151951 – Supplemental material for Early non-disabling relapses are important predictors of disability accumulation in people with relapsing-remitting multiple sclerosis

Author

Daruwalla, Cyrus, Shaygannejad, Vahid, Ozakbas, Serkan, Havrdova, Eva Kubala, Horakova, Dana, Alroughani, Raed, Boz, Cavit, Patti, Francesco, Onofrj, Marco, Lugaresi, Alessandra, Eichau, Sara, Girard, Marc, Prat, Alexandre, Duquette, Pierre, Yamout, Bassem, Khoury, Samia J, Sajedi, Seyed Aidin, Turkoglu, Recai, Altintas, Ayse, Skibina, Olga, Buzzard, Katherine, Grammond, Pierre, Karabudak, Rana, van der Walt, Anneke, Butzkueven, Helmut, Maimone, Davide, Lechner-Scott, Jeannette, Soysal, Aysun, John, Nevin, Prevost, Julie, Spitaleri, Daniele, Ramo-Tello, Cristina, Gerlach, Oliver, Iuliano, Gerardo, Foschi, Matteo, Ampapa, Radek, van Pesch, Vincent, Barnett, Michael, Shalaby, Nevin, D’hooghe, Marie, Kuhle, Jens, Sa, Maria Jose, Fabis-Pedrini, Marzena, Kermode, Allan, Mrabet, Saloua, Gouider, Riadh, Hodgkinson, Suzanne, Laureys, Guy, Van Hijfte, Liesbeth, Macdonell, Richard, Oreja-Guevara, Celia, Cristiano, Edgardo, McCombe, Pamela, Sanchez-Menoyo, Jose Luis, Singhal, Bhim, Blanco, Yolanda, Hughes, Stella, Garber, Justin, Solaro, Claudio, McGuigan, Chris, Taylor, Bruce, de Gans, Koen, Habek, Mario, Al-Asmi, Abdullah, Mihaela, Simu, Castillo Triviño, Tamara, Al-Harbi, Talal, Rojas, Juan Ignacio, Gray, Orla, Khurana, Dheeraj, Van Wijmeersch, Bart, Grigoriadis, Nikolaos, Inshasi, Jihad, Oh, Jiwon, Aguera-Morales, Eduardo, Fragoso, Yara, Moore, Fraser, Shaw, Cameron, Baghbanian, Seyed Mohammad, Shuey, Neil, Willekens, Barbara, Hardy, Todd A, Decoo, Danny, sempere, Angel Perez, Field, Deborah, Wynford-Thomas, Ray, Cunniffe, Nick G, Roos, Izanne, Malpas, Charles B, Coles, Alasdair J, Kalincik, Tomas, and Brown, J William L

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

Supplemental material, sj-docx-1-msj-10.1177_13524585231151951 for Early non-disabling relapses are important predictors of disability accumulation in people with relapsing-remitting multiple sclerosis by Cyrus Daruwalla, Vahid Shaygannejad, Serkan Ozakbas, Eva Kubala Havrdova, Dana Horakova, Raed Alroughani, Cavit Boz, Francesco Patti, Marco Onofrj, Alessandra Lugaresi, Sara Eichau, Marc Girard, Alexandre Prat, Pierre Duquette, Bassem Yamout, Samia J Khoury, Seyed Aidin Sajedi, Recai Turkoglu, Ayse Altintas, Olga Skibina, Katherine Buzzard, Pierre Grammond, Rana Karabudak, Anneke van der Walt, Helmut Butzkueven, Davide Maimone, Jeannette Lechner-Scott, Aysun Soysal, Nevin John, Julie Prevost, Daniele Spitaleri, Cristina Ramo-Tello, Oliver Gerlach, Gerardo Iuliano, Matteo Foschi, Radek Ampapa, Vincent van Pesch, Michael Barnett, Nevin Shalaby, Marie D’hooghe, Jens Kuhle, Maria Jose Sa, Marzena Fabis-Pedrini, Allan Kermode, Saloua Mrabet, Riadh Gouider, Suzanne Hodgkinson, Guy Laureys, Liesbeth Van Hijfte, Richard Macdonell, Celia Oreja-Guevara, Edgardo Cristiano, Pamela McCombe, Jose Luis Sanchez-Menoyo, Bhim Singhal, Yolanda Blanco, Stella Hughes, Justin Garber, Claudio Solaro, Chris McGuigan, Bruce Taylor, Koen de Gans, Mario Habek, Abdullah Al-Asmi, Simu Mihaela, Tamara Castillo Triviño, Talal Al-Harbi, Juan Ignacio Rojas, Orla Gray, Dheeraj Khurana, Bart Van Wijmeersch, Nikolaos Grigoriadis, Jihad Inshasi, Jiwon Oh, Eduardo Aguera-Morales, Yara Fragoso, Fraser Moore, Cameron Shaw, Seyed Mohammad Baghbanian, Neil Shuey, Barbara Willekens, Todd A Hardy, Danny Decoo, Angel Perez sempere, Deborah Field, Ray Wynford-Thomas, Nick G Cunniffe, Izanne Roos, Charles B Malpas, Alasdair J Coles, Tomas Kalincik and J William L Brown in Multiple Sclerosis Journal

Published
2023
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31. The risk of secondary progressive multiple sclerosis is geographically determined but modifiable

Author

Sharmin, Sifat, Roos, Izanne, Simpson-Yap, Steve, Malpas, Charles, Sánchez, Marina M, Ozakbas, Serkan, Horakova, Dana, Havrdova, Eva K, Patti, Francesco, Alroughani, Raed, Izquierdo, Guillermo, Eichau, Sara, Boz, Cavit, Zakaria, Magd, Onofrj, Marco, Lugaresi, Alessandra, Weinstock-Guttman, Bianca, Prat, Alexandre, Girard, Marc, Duquette, Pierre, Terzi, Murat, Amato, Maria Pia, Karabudak, Rana, Grand'Maison, Francois, Khoury, Samia J, Grammond, Pierre, Lechner-Scott, Jeannette, Buzzard, Katherine, Skibina, Olga, van der Walt, Anneke, Butzkueven, Helmut, Turkoglu, Recai, Altintas, Ayse, Maimone, Davide, Kermode, Allan, Shalaby, Nevin, Pesch, Vincent V, Butler, Ernest, Sidhom, Youssef, Gouider, Riadh, Mrabet, Saloua, Gerlach, Oliver, Soysal, Aysun, Barnett, Michael, Kuhle, Jens, Hughes, Stella, Maria J, Sa, Hodgkinson, Suzanne, Oreja-Guevara, Celia, Ampapa, Radek, Petersen, Thor, Ramo-Tello, Cristina, Spitaleri, Daniele, Mccombe, Pamela, Taylor, Bruce, Prevost, Julie, Foschi, Matteo, Slee, Mark, Mcguigan, Chris, Laureys, Guy, Hijfte, Liesbeth V, de Gans, Koen, Solaro, Claudio, Jiwon, Oh, Macdonell, Richard, Aguera-Morales, Eduardo, Singhal, Bhim, Gray, Orla, Garber, Justin, Wijmeersch, Bart V, Simu, Mihaela, Triviño, Tamara C, Sanchez-Menoyo, Jose L, Khurana, Dheeraj, Al-Asmi, Abdullah, Al-Harbi, Talal, Deri, Norma, Fragoso, Yara, Lalive, Patrice H, Sinnige, L G F, Shaw, Cameron, Shuey, Neil, Csepany, Tunde, Sempere, Angel P, Moore, Fraser, Decoo, Danny, Willekens, Barbara, Gobbi, Claudio, Massey, Jennifer, Hardy, Todd, Parratt, John, and Kalincik, Tomas

Subjects
secondary progressive multiple sclerosis, health expenditure, disease-modifying therapy, geography, latitude
Published
2023

32. Therapeutic approaches to disease modifying therapy for multiple sclerosis in adults: An Australian and New Zealand perspective Part 1 Historical and established therapies

Author

Broadley, Simon A., Barnett, Michael H., Boggild, Mike, Brew, Bruce J., Butzkueven, Helmut, Heard, Robert, Hodgkinson, Suzanne, Kermode, Allan G., Lechner-Scott, Jeannette, Macdonell, Richard A.L., Marriott, Mark, Mason, Deborah F., Parratt, John, Reddel, Stephen W., Shaw, Cameron P., Slee, Mark, Spies, Judith, Taylor, Bruce V., Carroll, William M., Kilpatrick, Trevor J., King, John, McCombe, Pamela A., Pollard, John D., and Willoughby, Ernest

Published
2014
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33. Therapeutic approaches to disease modifying therapy for multiple sclerosis in adults: An Australian and New Zealand perspective Part 3 Treatment practicalities and recommendations

Author

Broadley, Simon A., Barnett, Michael H., Boggild, Mike, Brew, Bruce J., Butzkueven, Helmut, Heard, Robert, Hodgkinson, Suzanne, Kermode, Allan G., Lechner-Scott, Jeannette, Macdonell, Richard A.L., Marriott, Mark, Mason, Deborah F., Parratt, John, Reddel, Stephen W., Shaw, Cameron P., Slee, Mark, Spies, Judith, Taylor, Bruce V., Carroll, William M., Kilpatrick, Trevor J., King, John, McCombe, Pamela A., Pollard, John D., and Willoughby, Ernest

Published
2014
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34. Therapeutic approaches to disease modifying therapy for multiple sclerosis in adults: An Australian and New Zealand perspective Part 2 New and emerging therapies and their efficacy

Author

Broadley, Simon A., Barnett, Michael H., Boggild, Mike, Brew, Bruce J., Butzkueven, Helmut, Heard, Robert, Hodgkinson, Suzanne, Kermode, Allan G., Lechner-Scott, Jeannette, Macdonell, Richard A.L., Marriott, Mark, Mason, Deborah F., Parratt, John, Reddel, Stephen W., Shaw, Cameron P., Slee, Mark, Spies, Judith, Taylor, Bruce V., Carroll, William M., Kilpatrick, Trevor J., King, John, McCombe, Pamela A., Pollard, John D., and Willoughby, Ernest

Published
2014
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35. Disease Reactivation After Cessation of Disease-Modifying Therapy in Patients With Relapsing-Remitting Multiple Sclerosis

Author

Roos, Izanne, Malpas, Charles, Leray, Emmanuelle, Casey, Romain, Horakova, Dana, Havrdova, Eva Kubala, Debouverie, Marc, Patti, Francesco, De Seze, Jerome, Izquierdo, Guillermo, Eichau, Sara, Edan, Gilles, Prat, Alexandre, Girard, Marc, Ozakbas, Serkan, Grammond, Pierre, Zephir, Helene, Ciron, Jonathan, Maillart, Elisabeth, Moreau, Thibault, Amato, Maria Pia, Labauge, Pierre, Alroughani, Raed, Buzzard, Katherine, Skibina, Olga, Terzi, Murat, Laplaud, David Axel, Berger, Eric, Grand'Maison, Francois, Lebrun-Frenay, Christine, Cartechini, Elisabetta, Boz, Cavit, Lechner-Scott, Jeannette, Clavelou, Pierre, Stankoff, Bruno, Prevost, Julie, Kappos, Ludwig, Pelletier, Jean, Shaygannejad, Vahid, Yamout, Bassem I, Khoury, Samia J, Gerlach, Oliver, Spitaleri, Daniele L A, Van Pesch, Vincent, Gout, Olivier, Turkoglu, Recai, Heinzlef, Olivier, Thouvenot, Eric, McCombe, Pamela Ann, Soysal, Aysun, Bourre, Bertrand, Slee, Mark, Castillo-Trivino, Tamara, Bakchine, Serge, Ampapa, Radek, Butler, Ernest Gerard, Wahab, Abir, Macdonell, Richard A, Aguera-Morales, Eduardo, Cabre, Philippe, Ben, Nasr Haifa, Van der Walt, Anneke, Laureys, Guy, Van Hijfte, Liesbeth, Ramo-Tello, Cristina M, Maubeuge, Nicolas, Hodgkinson, Suzanne, Sánchez-Menoyo, José Luis, Barnett, Michael H, Labeyrie, Celine, Vucic, Steve, Sidhom, Youssef, Gouider, Riadh, Csepany, Tunde, Sotoca, Javier, de Gans, Koen, Al-Asmi, Abdullah, Fragoso, Yara Dadalti, Vukusic, Sandra, Butzkueven, Helmut, Kalincik, Tomas, MSBase and OFSEP, UCL - SSS/IONS/CEMO - Pôle Cellulaire et moléculaire, University of Melbourne, Centre de Recherches sur l'Action Politique en Europe (ARENES), Université de Rennes (UR)-Institut d'Études Politiques [IEP] - Rennes-École des Hautes Études en Santé Publique [EHESP] (EHESP)-Centre National de la Recherche Scientifique (CNRS), Recherche sur les services et le management en santé (RSMS), Université de Rennes (UR)-École des Hautes Études en Santé Publique [EHESP] (EHESP)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS), École des Hautes Études en Santé Publique [EHESP] (EHESP), Centre d'Investigation Clinique [Rennes] (CIC), Université de Rennes (UR)-Hôpital Pontchaillou-Institut National de la Santé et de la Recherche Médicale (INSERM), Recherche en Pharmaco-épidémiologie et Recours aux Soins (REPERES), Université de Rennes (UR)-École des Hautes Études en Santé Publique [EHESP] (EHESP), Hospices Civils de Lyon (HCL), Centre de recherche en neurosciences de Lyon - Lyon Neuroscience Research Center (CRNL), Université Claude Bernard Lyon 1 (UCBL), Université de Lyon-Université de Lyon-Université Jean Monnet - Saint-Étienne (UJM)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS), Adaptation, mesure et évaluation en santé. Approches interdisciplinaires (APEMAC), Université de Lorraine (UL), CIC Strasbourg (Centre d’Investigation Clinique Plurithématique (CIC - P) ), Université de Strasbourg (UNISTRA)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Nouvel Hôpital Civil de Strasbourg-Hôpital de Hautepierre [Strasbourg], CHU Pontchaillou [Rennes], Centre d'épidémiologie des populations (CEP), Université de Bourgogne (UB)-Centre Régional de Lutte contre le cancer Georges-François Leclerc [Dijon] (UNICANCER/CRLCC-CGFL), UNICANCER-UNICANCER, CIC Plurithématique de Nantes, Institut National de la Santé et de la Recherche Médicale (INSERM)-Ministère des Affaires sociales et de la Santé-Direction générale de l'offre de soins (DGOS)-Centre hospitalier universitaire de Nantes (CHU Nantes), Centre de Recherche en Transplantation et Immunologie - Center for Research in Transplantation and Translational Immunology (U1064 Inserm - CR2TI), Institut National de la Santé et de la Recherche Médicale (INSERM)-Nantes Université - UFR de Médecine et des Techniques Médicales (Nantes Univ - UFR MEDECINE), Nantes Université - pôle Santé, Nantes Université (Nantes Univ)-Nantes Université (Nantes Univ)-Nantes Université - pôle Santé, Nantes Université (Nantes Univ)-Nantes Université (Nantes Univ), Unité de Recherche Clinique de la Côte d’Azur (URRIS UR2CA), Centre Hospitalier Universitaire de Nice (CHU Nice)-Université Côte d'Azur (UCA), Neuro-Dol (Neuro-Dol), Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Clermont Auvergne (UCA), Institut du Cerveau = Paris Brain Institute (ICM), Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Institut National de la Santé et de la Recherche Médicale (INSERM)-CHU Pitié-Salpêtrière [AP-HP], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)-Sorbonne Université (SU)-Sorbonne Université (SU)-Centre National de la Recherche Scientifique (CNRS), Hôpital de la Timone [CHU - APHM] (TIMONE), Institut de Génomique Fonctionnelle (IGF), and Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS)-Université de Montpellier (UM)

Subjects
Multiple Sclerosis, Relapsing-Remitting, Multiple Sclerosis, Fingolimod Hydrochloride, Recurrence, [SDV]Life Sciences [q-bio], Natalizumab, Humans, Female, Neurology (clinical), Immunosuppressive Agents, Research Article, Retrospective Studies
Abstract

Objectives:To evaluate the rate of return of disease activity after cessation of multiple sclerosis (MS) disease-modifying therapy.Methods:This was a retrospective cohort study from two large observational MS registries: MSBase and OFSEP. Patients with relapsing-remitting MS who had ceased a disease-modifying therapy and were followed up for the subsequent 12-months were included in the analysis. The primary study outcome was annualised relapse rate in the 12 months after disease-modifying therapy discontinuation stratified by patients who did, and did not, commence a subsequent therapy. The secondary endpoint was the predictors of first relapse and disability accumulation after treatment discontinuation.Results:14,213 patients, with 18,029 eligible treatment discontinuation epochs, were identified for seven therapies. Annualised rates of relapse (ARR) started to increase 2-months after natalizumab cessation (month 2-4 ARR, 95% confidence interval): 0.47, 0.43-0.51). Commencement of a subsequent therapy within 2-4 months reduced the magnitude of disease reactivation (mean ARR difference: 0.15, 0.08-0.22). After discontinuation of fingolimod, rates of relapse increased overall (month 1-2 ARR: 0.80, 0.70-0.89), and stabilised faster in patients who started a new therapy within 1-2 months (mean ARR difference: 0.14, -0.01-0.29). Magnitude of disease reactivation for other therapies was low, but reduced further by commencement of another treatment 1-10 months after treatment discontinuation. Predictors of relapse were higher relapse rate in the year before cessation, female sex, younger age and higher EDSS. Commencement of a subsequent therapy reduced both the risk of relapse (HR 0.76, 95%CI 0.72-0.81) and disability accumulation (0.73, 0.65-0.80).Conclusion:The rate of disease reactivation after treatment cessation differs among MS treatments, with the peaks of relapse activity ranging from 1 to 10 months in untreated cohorts that discontinued different therapies. These results suggest that untreated intervals should be minimised after stopping anti-trafficking therapies (natalizumab and fingolimod).Classification of evidence:This study provides class III that disease reactivation occurs within months of discontinuation of multiple sclerosis disease-modifying therapies. Risk of disease activity is reduced by commencement of a subsequent therapy.

Published
2022

36. Comparing switch to ocrelizumab, cladribine or natalizumab after fingolimod treatment cessation in multiple sclerosis

Author

Zhu, Chao, primary, Zhou, Zhen, additional, Roos, Izanne, additional, Merlo, Daniel, additional, Kalincik, Tomas, additional, Ozakbas,, Serkan, additional, Skibina, Olga, additional, Kuhle, Jens, additional, Hodgkinson, Suzanne, additional, Boz, Cavit, additional, Alroughani, Raed, additional, Lechner-Scott, Jeannette, additional, Barnett, Michael, additional, Izquierdo, Guillermo, additional, Prat, Alexandre, additional, Horakova, Dana, additional, Kubala Havrdova, Eva, additional, Macdonell, Richard, additional, Patti, Francesco, additional, Khoury, Samia Joseph, additional, Slee, Mark, additional, Karabudak, Rana, additional, Onofrj, Marco, additional, Van Pesch, Vincent, additional, Prevost, Julie, additional, Monif, Mastura, additional, Jokubaitis, Vilija, additional, van der Walt, Anneke, additional, and Butzkueven, Helmut, additional

Published
2022
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37. 2242 Comparison of the effectiveness of ocrelizumab vs interferon β, fingolimod and natalizumab on relapses in relapsing-remitting multiple sclerosis

Author

Roos, Izanne, primary, Sharmin, Sifat, additional, Lechner-Scott, Jeannette, additional, Buzzard, Katherine, additional, Skibina, Olga, additional, Walt, Anneke van der, additional, Butzkueven, Helmut, additional, Butler, Ernest, additional, MacDonell, Richard, additional, McCombe, Pamela, additional, Slee, Mark, additional, Malpas, Charles, additional, and Kalincik, Tomas, additional

Published
2022
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38. 2339 Neuropsychological outcomes in chronic autoimmune encephalitis – A study from the Australian autoimmune encephalitis consortium

Author

Griffith, Sarah, primary, Wesselingh, Robb, additional, Rushen, Tiffany, additional, Broadley, James, additional, O’Shea, Marie, additional, Kyndt, Chris, additional, Meade, Catherine, additional, Seneviratne, Udaya, additional, Reidy, Natalie, additional, Halliday, Amy, additional, Buzzard, Katherine, additional, Kalincik, Tomas, additional, D’Souza, Wendyl, additional, Macdonell, Richard, additional, Brodtmann, Amy, additional, Butzkueven, Helmut, additional, O’Brien, Terence, additional, Alpitsis, Rubina, additional, Malpas, Charles, additional, and Monif, Mastura, additional

Published
2022
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40. 2327 Latitude, UVR and multiple sclerosis severity

Author

Vitkova, Marianna, primary, Diouf, Ibrahima, additional, Malpas, Charles, additional, Barnett, Michael, additional, Hodgkinson, Suzanne, additional, Butler, Ernest, additional, Slee, Mark, additional, Taylor, Bruce, additional, Butzkueven, Helmut, additional, Lechner-Scott, Jeanette, additional, McCombe, Pamela, additional, Vucic, Steve, additional, Macdonell, Richard, additional, Shaw, Cameron, additional, and Kalincik, Tomas, additional

Published
2022
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41. 2330 Neuropsychological syndromes of autoimmune encephalitis – a retrospective study

Author

Griffith, Sarah, primary, Wesselingh, Robb, additional, Broadley, James, additional, O’Shea, Marie, additional, Kyndt, Chris, additional, Meade, Catherine, additional, Long, Brian, additional, Seneviratne, Udaya, additional, Reidy, Natalie, additional, Bourke, Robert, additional, Buzzard, Katherine, additional, D’Souza, Wendyl, additional, Macdonell, Richard, additional, Brodtmann, Amy, additional, Butzkueven, Helmut, additional, O’Brien, Terence, additional, Alpitsis, Rubina, additional, Malpas, Charles, additional, and Monif, Mastura, additional

Published
2022
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43. Treatment satisfaction in patients with relapsing-remitting multiple sclerosis initiated on teriflunomide in routine clinical practice: Australian observational data

Author

Hardy, Todd A, primary, Parratt, John, additional, Beadnall, Heidi, additional, Blum, Stefan, additional, Macdonell, Richard, additional, Beran, Roy G, additional, Shuey, Neil, additional, Lee, Andrew, additional, Carroll, William, additional, Shaw, Cameron, additional, Worrell, Richard, additional, Moody, Jana, additional, Sedhom, Mamdouh, additional, Barnett, Michael, additional, and Vucic, Steve, additional

Published
2022
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44. Confirmed disability progression as a marker of permanent disability in multiple sclerosis

Author

Sharmin, Sifat, primary, Bovis, Francesca, additional, Malpas, Charles, additional, Horakova, Dana, additional, Havrdova, Eva Kubala, additional, Izquierdo, Guillermo, additional, Eichau, Sara, additional, Trojano, Maria, additional, Prat, Alexandre, additional, Girard, Marc, additional, Duquette, Pierre, additional, Onofrj, Marco, additional, Lugaresi, Alessandra, additional, Grand'Maison, Francois, additional, Grammond, Pierre, additional, Sola, Patrizia, additional, Ferraro, Diana, additional, Terzi, Murat, additional, Gerlach, Oliver, additional, Alroughani, Raed, additional, Boz, Cavit, additional, Shaygannejad, Vahid, additional, van Pesch, Vincent, additional, Cartechini, Elisabetta, additional, Kappos, Ludwig, additional, Lechner‐Scott, Jeannette, additional, Bergamaschi, Roberto, additional, Turkoglu, Recai, additional, Solaro, Claudio, additional, Iuliano, Gerardo, additional, Granella, Franco, additional, Van Wijmeersch, Bart, additional, Spitaleri, Daniele, additional, Slee, Mark, additional, McCombe, Pamela, additional, Prevost, Julie, additional, Ampapa, Radek, additional, Ozakbas, Serkan, additional, Sanchez‐Menoyo, Jose Luis, additional, Soysal, Aysun, additional, Vucic, Steve, additional, Petersen, Thor, additional, de Gans, Koen, additional, Butler, Ernest, additional, Hodgkinson, Suzanne, additional, Sidhom, Youssef, additional, Gouider, Riadh, additional, Cristiano, Edgardo, additional, Castillo‐Triviño, Tamara, additional, Saladino, Maria Laura, additional, Barnett, Michael, additional, Moore, Fraser, additional, Rozsa, Csilla, additional, Yamout, Bassem, additional, Skibina, Olga, additional, van der Walt, Anneke, additional, Buzzard, Katherine, additional, Gray, Orla, additional, Hughes, Stella, additional, Sempere, Angel Perez, additional, Singhal, Bhim, additional, Fragoso, Yara, additional, Shaw, Cameron, additional, Kermode, Allan, additional, Taylor, Bruce, additional, Simo, Magdolna, additional, Shuey, Neil, additional, Al‐Harbi, Talal, additional, Macdonell, Richard, additional, Dominguez, Jose Andres, additional, Csepany, Tunde, additional, Sirbu, Carmen Adella, additional, Sormani, Maria Pia, additional, Butzkueven, Helmut, additional, and Kalincik, Tomas, additional

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2022
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46. Long-Term Safety and Effectiveness of Delayed-release Dimethyl Fumarate in Multiple Sclerosis Patients Treated in Routine Medical Practice (P7-4.009)

Author

Pandey, Krupa, primary, Giles, Kathryn, additional, Balashov, Konstantin, additional, Macdonell, Richard, additional, Windsheimer, Jorg, additional, Martinez, Mikel, additional, Bozin, Ivan, additional, Grimes, Nydije, additional, Jiang, Xiaotong, additional, Kapadia, Shivani, additional, Lyons, Jennifer, additional, Mokliatchouk, Oksana, additional, and Okai, Annette, additional

Published
2022
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47. Early non-disabling relapses are important predictors of disability accumulation in people with relapsing-remitting multiple sclerosis

Author

Rojas, Juan Ignacio, Gray, Orla, Khurana, Dheeraj, Van Wijmeersch, Bart, Grigoriadis, Nikolaos, Inshasi, Jihad, Oh, Jiwon, Aguera-Morales, Eduardo, Fragoso, Yara, Moore, Fraser, Shaw, Cameron, Baghbanian, Seyed Mohammad, Shuey, Neil, Willekens, Barbara, Hardy, Todd A., Decoo, Danny, Sempere, Angel Perez, Field, Deborah, Wynford-Thomas, Ray, Cunniffe, Nick G., Roos, Izanne, Malpas, Charles B., Coles, Alasdair J., Kalincik, Tomas, Brown, J. William L., MSBase Study Grp, MSBase Study Grp, Shaygannejad, Vahid, Daruwalla, Cyrus, ÖZAKBAŞ, SERKAN, Havrdova, Eva Kubala, Horakova, Dana, Alroughani, Raed, BOZ, CAVİT, Patti, Francesco, Onofrj, Marco, Lugaresi, Alessandra, Eichau, Sara, Girard, Marc, Prat, Alexandre, Duquette, Pierre, Yamout, Bassem, Khoury, Samia J., Sajedi, Seyed Aidin, Turkoglu, Recai, Altintas, Ayse, Skibina, Olga, Buzzard, Katherine, Grammond, Pierre, Karabudak, Rana, van der Walt, Anneke, Butzkueven, Helmut, Maimone, Davide, Lechner-Scott, Jeannette, Soysal, Aysun, John, Nevin, Prevost, Julie, Spitaleri, Daniele, Ramo-Tello, Cristina, Gerlach, Oliver, Iuliano, Gerardo, Foschi, Matteo, Ampapa, Radek, van Pesch, Vincent, Barnett, Michael, Shalaby, Nevin, D'hooghe, Marie, Kuhle, Jens, Sa, Maria Jose, Fabis-Pedrini, Marzena, Kermode, Allan, Mrabet, Saloua, Gouider, Riadh, Hodgkinson, Suzanne, Laureys, Guy, Van Hijfte, Liesbeth, Macdonell, Richard, Oreja-Guevara, Celia, Cristiano, Edgardo, McCombe, Pamela, Sanchez-Menoyo, Jose Luis, Singhal, Bhim, Blanco, Yolanda, Hughes, Stella, Garber, Justin, Solaro, Claudio, McGuigan, Chris, Taylor, Bruce, de Gans, Koen, Habek, Mario, Al-Asmi, Abdullah, Mihaela, Simu, Castillo Trivino, Tamara, Al-Harbi, Talal, MSBase Study Group, Clinical sciences, Neuroprotection & Neuromodulation, Neurology, Faculty of Medicine and Pharmacy, Daruwalla, Cyrus [0000-0002-2329-5329], Shaygannejad, Vahid [0000-0001-5511-509X], Horakova, Dana [0000-0003-1915-0036], Alroughani, Raed [0000-0001-5436-5804], Patti, Francesco [0000-0002-6923-0846], Lugaresi, Alessandra [0000-0003-2902-5589], Eichau, Sara [0000-0001-9159-3128], Duquette, Pierre [0000-0001-7231-1754], Sajedi, Seyed Aidin [0000-0002-6704-9787], van der Walt, Anneke [0000-0002-4278-7003], Lechner-Scott, Jeannette [0000-0002-3850-447X], Barnett, Michael [0000-0002-2156-8864], Oreja-Guevara, Celia [0000-0002-9221-5716], Habek, Mario [0000-0002-3360-1748], Castillo Triviño, Tamara [0000-0002-9249-3185], Inshasi, Jihad [0000-0001-5892-751X], Oh, Jiwon [0000-0001-5519-6088], Fragoso, Yara [0000-0001-8726-089X], Baghbanian, Seyed Mohammad [0000-0002-8138-7504], Hardy, Todd A [0000-0003-4145-3172], Decoo, Danny [0000-0001-7689-3114], Roos, Izanne [0000-0003-0371-3666], Kalincik, Tomas [0000-0003-3778-1376], Brown, J William L [0000-0002-7737-5834], Apollo - University of Cambridge Repository, and UCL - SSS/IONS/CEMO - Pôle Cellulaire et moléculaire

Subjects
Neuroscience(all), Multiple sclerosis, prognosis, multiple sclerosis, Prognosis, Multiple Sclerosis, Relapsing-Remitting, Neurology, Recurrence, Medicine and Health Sciences, Humans, Human medicine, Neurology (clinical)
Abstract

Background: The prognostic significance of non-disabling relapses in people with relapsing-remitting multiple sclerosis (RRMS) is unclear. Objective: To determine whether early non-disabling relapses predict disability accumulation in RRMS. Methods: We redefined mild relapses in MSBase as ‘non-disabling’, and moderate or severe relapses as ‘disabling’. We used mixed-effects Cox models to compare 90-day confirmed disability accumulation events in people with exclusively non-disabling relapses within 2 years of RRMS diagnosis to those with no early relapses; and any early disabling relapses. Analyses were stratified by disease-modifying therapy (DMT) efficacy during follow-up. Results: People who experienced non-disabling relapses within 2 years of RRMS diagnosis accumulated more disability than those with no early relapses if they were untreated ( n = 285 vs 4717; hazard ratio (HR) = 1.29, 95% confidence interval (CI) = 1.00–1.68) or given platform DMTs ( n = 1074 vs 7262; HR = 1.33, 95% CI = 1.15–1.54), but not if given high-efficacy DMTs ( n = 572 vs 3534; HR = 0.90, 95% CI = 0.71–1.13) during follow-up. Differences in disability accumulation between those with early non-disabling relapses and those with early disabling relapses were not confirmed statistically. Conclusion: This study suggests that early non-disabling relapses are associated with a higher risk of disability accumulation than no early relapses in RRMS. This risk may be mitigated by high-efficacy DMTs. Therefore, non-disabling relapses should be considered when making treatment decisions.

Published
2023

48. Confirmed disability progression as a marker of permanent disability in multiple sclerosis.

Author

UCL - SSS/IONS/CEMO - Pôle Cellulaire et moléculaire, UCL - (SLuc) Service de biochimie médicale, UCL - (SLuc) Service de neurologie, Sharmin, Sifat, Bovis, Francesca, Malpas, Charles, Horakova, Dana, Havrdova, Eva Kubala, Izquierdo, Guillermo, Eichau, Sara, Trojano, Maria, Prat, Alexandre, Girard, Marc, Duquette, Pierre, Onofrj, Marco, Lugaresi, Alessandra, Grand'Maison, Francois, Grammond, Pierre, Sola, Patrizia, Ferraro, Diana, Terzi, Murat, Gerlach, Oliver, Alroughani, Raed, Boz, Cavit, Shaygannejad, Vahid, van Pesch, Vincent, Cartechini, Elisabetta, Kappos, Ludwig, Lechner-Scott, Jeannette, Bergamaschi, Roberto, Turkoglu, Recai, Solaro, Claudio, Iuliano, Gerardo, Granella, Franco, Van Wijmeersch, Bart, Spitaleri, Daniele, Slee, Mark, McCombe, Pamela, Prevost, Julie, Ampapa, Radek, Ozakbas, Serkan, Sanchez-Menoyo, Jose Luis, Soysal, Aysun, Vucic, Steve, Petersen, Thor, de Gans, Koen, Butler, Ernest, Hodgkinson, Suzanne, Sidhom, Youssef, Gouider, Riadh, Cristiano, Edgardo, Castillo-Triviño, Tamara, Saladino, Maria Laura, Barnett, Michael, Moore, Fraser, Rozsa, Csilla, Yamout, Bassem, Skibina, Olga, van der Walt, Anneke, Buzzard, Katherine, Gray, Orla, Hughes, Stella, Sempere, Angel Perez, Singhal, Bhim, Fragoso, Yara, Shaw, Cameron, Kermode, Allan, Taylor, Bruce, Simo, Magdolna, Shuey, Neil, Al-Harbi, Talal, Macdonell, Richard, Dominguez, Jose Andres, Csepany, Tunde, Sirbu, Carmen Adella, Sormani, Maria Pia, Butzkueven, Helmut, Kalincik, Tomas, UCL - SSS/IONS/CEMO - Pôle Cellulaire et moléculaire, UCL - (SLuc) Service de biochimie médicale, UCL - (SLuc) Service de neurologie, Sharmin, Sifat, Bovis, Francesca, Malpas, Charles, Horakova, Dana, Havrdova, Eva Kubala, Izquierdo, Guillermo, Eichau, Sara, Trojano, Maria, Prat, Alexandre, Girard, Marc, Duquette, Pierre, Onofrj, Marco, Lugaresi, Alessandra, Grand'Maison, Francois, Grammond, Pierre, Sola, Patrizia, Ferraro, Diana, Terzi, Murat, Gerlach, Oliver, Alroughani, Raed, Boz, Cavit, Shaygannejad, Vahid, van Pesch, Vincent, Cartechini, Elisabetta, Kappos, Ludwig, Lechner-Scott, Jeannette, Bergamaschi, Roberto, Turkoglu, Recai, Solaro, Claudio, Iuliano, Gerardo, Granella, Franco, Van Wijmeersch, Bart, Spitaleri, Daniele, Slee, Mark, McCombe, Pamela, Prevost, Julie, Ampapa, Radek, Ozakbas, Serkan, Sanchez-Menoyo, Jose Luis, Soysal, Aysun, Vucic, Steve, Petersen, Thor, de Gans, Koen, Butler, Ernest, Hodgkinson, Suzanne, Sidhom, Youssef, Gouider, Riadh, Cristiano, Edgardo, Castillo-Triviño, Tamara, Saladino, Maria Laura, Barnett, Michael, Moore, Fraser, Rozsa, Csilla, Yamout, Bassem, Skibina, Olga, van der Walt, Anneke, Buzzard, Katherine, Gray, Orla, Hughes, Stella, Sempere, Angel Perez, Singhal, Bhim, Fragoso, Yara, Shaw, Cameron, Kermode, Allan, Taylor, Bruce, Simo, Magdolna, Shuey, Neil, Al-Harbi, Talal, Macdonell, Richard, Dominguez, Jose Andres, Csepany, Tunde, Sirbu, Carmen Adella, Sormani, Maria Pia, Butzkueven, Helmut, and Kalincik, Tomas

Abstract

The prevention of disability over the long term is the main treatment goal in multiple sclerosis (MS); however, randomized clinical trials evaluate only short-term treatment effects on disability. This study aimed to define criteria for 6-month confirmed disability progression events of MS with a high probability of resulting in sustained long-term disability worsening. In total, 14,802 6-month confirmed disability progression events were identified in 8741 patients from the global MSBase registry. For each 6-month confirmed progression event (13,321 in the development and 1481 in the validation cohort), a sustained progression score was calculated based on the demographic and clinical characteristics at the time of progression that were predictive of long-term disability worsening. The score was externally validated in the Cladribine Tablets Treating Multiple Sclerosis Orally (CLARITY) trial. The score was based on age, sex, MS phenotype, relapse activity, disability score and its change from baseline, number of affected functional system domains and worsening in six of the domains. In the internal validation cohort, a 61% lower chance of improvement was estimated with each unit increase in the score (hazard ratio 0.39, 95% confidence interval 0.29-0.52; discriminatory index 0.89). The proportions of progression events sustained at 5 years stratified by the score were 1: 72%; 2: 88%; 3: 94%; 4: 100%. The results of the CLARITY trial were confirmed for reduction of disability progression that was >88% likely to be sustained (events with score ˃1.5). Clinicodemographic characteristics of 6-month confirmed disability progression events identify those at high risk of sustained long-term disability. This knowledge will allow future trials to better assess the effect of therapy on long-term disability accrual.

Published
2022

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