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Comparative Effectiveness of Autologous Hematopoietic Stem Cell Transplant vs Fingolimod, Natalizumab, and Ocrelizumab in Highly Active Relapsing-Remitting Multiple Sclerosis.
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; Trnený, 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.
Afiliación
  • Kalincik T; Neuroimmunology Centre, Department of Neurology, Royal Melbourne Hospital, Melbourne, Victoria, Australia.
  • Sharmin S; CORe, Department of Medicine, University of Melbourne, Melbourne, Victoria, Australia.
  • Roos I; Neuroimmunology Centre, Department of Neurology, Royal Melbourne Hospital, Melbourne, Victoria, Australia.
  • Freedman MS; CORe, Department of Medicine, University of Melbourne, Melbourne, Victoria, Australia.
  • Atkins H; Neuroimmunology Centre, Department of Neurology, Royal Melbourne Hospital, Melbourne, Victoria, Australia.
  • Burman J; CORe, Department of Medicine, University of Melbourne, Melbourne, Victoria, Australia.
  • Massey J; University of Ottawa, Department of Medicine, Ottawa Hospital Research Institute, Ottawa, Ontario, Canada.
  • Sutton I; Ottawa Hospital Research Institute, University of Ottawa, Ottawa, Ontario, Canada.
  • Withers B; Department of Medical Sciences, Neurology, Uppsala University, Uppsala, Sweden.
  • Macdonell R; Department of Neurology, St Vincent's Hospital Sydney, Sydney, New South Wales, Australia.
  • Grigg A; St Vincent's Clinical School, University of New South Wales, Sydney, New South Wales, Australia.
  • Torkildsen Ø; Department of Neurology, St Vincent's Hospital Sydney, Sydney, New South Wales, Australia.
  • Bo L; University of Sydney, Sydney, New South Wales, Australia.
  • Lehmann AK; St Vincent's Clinical School, University of New South Wales, Sydney, New South Wales, Australia.
  • Havrdova EK; Department of Haematology, St Vincent's Hospital Sydney, Sydney, New South Wales, Australia.
  • Krasulova E; Department of Neurology, Austin Health, Melbourne, Victoria, Australia.
  • Trnený M; University of Melbourne, Melbourne, Victoria, Australia.
  • Kozak T; University of Melbourne, Melbourne, Victoria, Australia.
  • van der Walt A; Department of Haematology, Austin Health, Melbourne, Victoria, Australia.
  • Butzkueven H; Department of Neurology, Haukeland University Hospital, Bergen, Norway.
  • McCombe P; Department of Neurology, Haukeland University Hospital, Bergen, Norway.
  • Skibina O; Department of Haematology, Haukeland University Hospital, Bergen, Norway.
  • Lechner-Scott J; Department of Neurology and Center of Clinical Neuroscience, First Faculty of Medicine, Charles University in Prague and General University Hospital, Prague, Czech Republic.
  • Willekens B; Department of Neurology and Center of Clinical Neuroscience, First Faculty of Medicine, Charles University in Prague and General University Hospital, Prague, Czech Republic.
  • Cartechini E; Department of Haematology, First Faculty of Medicine, Charles University in Prague and General University Hospital, Prague, Czech Republic.
  • Ozakbas S; Department of Haematology, 3rd Faculty of Medicine, Charles University in Prague, and University Hospital Kralovske Vinohrady, Prague, Czech Republic.
  • Alroughani R; Department of Neurology, The Alfred Hospital, Melbourne, Victoria, Australia.
  • Kuhle J; Central Clinical School, Monash University, Melbourne, Victoria, Australia.
  • Patti F; Department of Neurology, The Alfred Hospital, Melbourne, Victoria, Australia.
  • Duquette P; Central Clinical School, Monash University, Melbourne, Victoria, Australia.
  • Lugaresi A; University of Queensland, Brisbane, Queensland, Australia.
  • Khoury SJ; Royal Brisbane and Women's Hospital, Brisbane, Queensland, Australia.
  • Slee M; Department of Neurology, The Alfred Hospital, Melbourne, Victoria, Australia.
  • Turkoglu R; Department of Neurology, Box Hill Hospital, Melbourne, Victoria, Australia.
  • Hodgkinson S; Monash University, Melbourne, Victoria, Australia.
  • John N; School of Medicine and Public Health, University Newcastle, Newcastle, New South Wales, Australia.
  • Maimone D; Department of Neurology, John Hunter Hospital, Hunter New England Health, Newcastle, New South Wales, Australia.
  • Sa MJ; Department of Neurology, Antwerp University Hospital, Edegem, Belgium.
  • van Pesch V; Translational Neurosciences Research Group, Faculty of Medicine and Health Sciences, University of Antwerp, Wilrijk, Belgium.
  • Gerlach O; UOC Neurologia, Azienda Sanitaria Unica Regionale Marche-AV3, Macerata, Italy.
  • Laureys G; Dokuz Eylul University, Konak, Izmir, Turkey.
  • Van Hijfte L; Division of Neurology, Department of Medicine, Amiri Hospital, Sharq, Kuwait.
  • Karabudak R; Neurologic Clinic and Policlinic, Departments of Medicine and Clinical Research, University Hospital and University of Basel, Basel, Switzerland.
  • Spitaleri D; Department of Medical and Surgical Sciences and Advanced Technologies, GF Ingrassia, Catania, Italy.
  • Csepany T; Multiple Sclerosis Center, University of Catania, Catania, Italy.
  • Gouider R; CHUM MS Center and Universite de Montreal, Montreal, Quebec, Canada.
  • Castillo-Triviño T; IRCCS Istituto delle Scienze Neurologiche di Bologna, Bologna, Italy.
  • Taylor B; Dipartimento di Scienze Biomediche e Neuromotorie, Università di Bologna, Bologna, Italy.
  • Sharrack B; Nehme and Therese Tohme Multiple Sclerosis Center, American University of Beirut Medical Center, Beirut, Lebanon.
  • Snowden JA; Flinders University, Adelaide, South Australia, Australia.
JAMA Neurol ; 80(7): 702-713, 2023 07 01.
Article en En | MEDLINE | ID: mdl-37437240
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.
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Trasplante de Células Madre Hematopoyéticas / Esclerosis Múltiple Recurrente-Remitente / Esclerosis Múltiple Límite: Adult / Female / Humans Idioma: En Revista: JAMA Neurol Año: 2023 Tipo del documento: Article País de afiliación: Australia
Texto completo
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Trasplante de Células Madre Hematopoyéticas / Esclerosis Múltiple Recurrente-Remitente / Esclerosis Múltiple Límite: Adult / Female / Humans Idioma: En Revista: JAMA Neurol Año: 2023 Tipo del documento: Article País de afiliación: Australia