Comparative Effectiveness of Autologous Hematopoietic Stem Cell Transplant vs Fingolimod, Natalizumab, and Ocrelizumab in Highly Active Relapsing-Remitting Multiple Sclerosis

作者：Tomáš Kalinčík, Sifat Sharmin, Izanne Roos, Mark Steven Freedman, Harold L. Atkins, Joachim Burman, Jennifer C. Massey, Ian C. Sutton, Barbara Withers, Richard A L Macdonell, Andrew Grigg, Øivind Torkildsen, Lars Bø, Anne Kristine Lehmann, Eva Havrdová, Eva Krasulová, Marek Trněný, Tomáš Kozák, Anneke van der Walt, Helmut Butzkueven, Pamela McCombe, Olga G. Skibina, Jeannette Lechner‐Scott, Barbara M. P. Willekens, Elisabetta Cartechini, Serkan Özakbaş, Raed Alroughani, Jens Kühle, Francesco Patti, Pierre Duquette, Alessandra Lugaresi, Samia Joseph Khoury, Mark Slee, Recai Türkoğlu, Suzanne J Hodgkinson, Nevin A. John, Davide Maimone, María José Sá, Vincent Van Pesch, Oliver H.H. Gerlach, Guy Laureys, Liesbeth Van Hijfte, Rana Karabudak, Daniele L.A. Spitaleri, Tünde Csépány, Riadh Gouider, Tamara Castillo‐Triviño, Bruce Taylor, Basil Sharrack, John Andrew Snowden, MSBase Study Group Collaborators, MSBase Study Group Authors, Dana Horáková, Katherine A. Buzzard, Murat Terzi, Alexandre Prat, Marc Girard, Pierre Grammond, Michael Harry Barnett, Grace Stewart, MARCO C. ONOFRJ, Guillermo Izquierdo, Sara Eichau, François Grand’Maison, Julie Prévost, Bart V Wijmeersch, Maria Pia Amato, Vahid Shaygannejad, Cavit Boz, Ricardo Fernandez Bolanos, Aysun Soysal, Cristina M. Ramo-Tello, Claudio Marcello Solaro, Claudio Gobbi, José Antonio Cabrera-Gómez, Etienne Roullet, Cees Zwanikken, Leontien Den Braber‐Moerland, Norma Deri, Maria Luisa Saladino, Edgardo Cristiano, Juan Ignacio Rojas, Cárlos Vrech, Cameron P. Shaw, Neil Shuey, Mike Boggild, Ik Lin Tan, Todd A. Hardy, D. Decoo, Fraser G. A. Moore, Jiwon Oh, Patrice H. Lalive, Radek Ampapa, Thor Petersen, Celia Oreja‐Guevara, Ángel Pérez Sempere, José Andrés Domínguez, Sarah Besora, Stella Hughes, Orla M. Gray, Nikolaos Grigoriadis, Piroska Imre, Csilla Rózsa, Krisztián Kása, Magdolna Simó, Krisztina Kovács, Attila Sas, Enikő Dobos, Cecília Rajda, Chris McGuigan, Deborah F. Mason, Jan Schepel, Jabir Alkhaboori, Maria Edite Rio, Simu Mihaela, Talal Al‐Harbi, Ayşe Altıntaş, Ilya Kister, Mark P. Marriott, Trevor John Kilpatrick, John O King, Ai‐Lan Nguyen, Chris Dwyer, Mastura Monif, Izanne Roos, Lisa Taylor, Matteo Diamanti, Clara Grazia Chisari, Simona Toscano, Lo Fermo Salvatore, Catherine Larochelle, Giovanna De Luca, Valeria di Tommaso, Daniela Travaglini, Erika Pietrolongo, Maria di Ioia, Deborah Farina, Luca Maria Mancinelli, Raymond Hupperts, Javier Olascoaga, Albert Saiz, Robert Zivadinov, Ralph H. B. Benedict, Freek Verheul, Marzena J. Fabis‐Pedrini, Saloua Mrabet, Justin Garber, José Luis Sánchez-Menoyo, Eduardo Agüera, Yolanda Blanco, Abdullah Rashid Al-Asmi, Bianca Weinstock‐Guttman, Yára Dadalti Fragoso, Koen de Gans, Allan G. Kermode · 发表于：JAMA Neurology · 年份：2023 · DOI：10.1001/jamaneurol.2023.1184 · 被引用次数：59 · 研究领域：Multiple Sclerosis Research Studies、Polyomavirus and related diseases、Systemic Sclerosis and Related Diseases

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 ...