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Clinical Measurement

MiToS and King’s staging as clinical outcome measures in ALS: a retrospective analysis of the FORTITUDE-ALS trial

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Pages 304-310 | Received 11 May 2022, Accepted 28 Nov 2022, Published online: 12 Dec 2022

References

  • Cedarbaum JM, Stambler N, Malta E, Fuller C, Hilt D, Thurmond B, et al. The ALSFRS-R: a revised ALS functional rating scale that incorporates assessments of respiratory function. BDNF ALS Study Group (Phase III). J Neurol Sci. 1999;169:13–21.
  • Tramacere I, Dalla Bella E, Chiò A, Mora G, Filippini G, Lauria G, et al. The MITOS system predicts long-term survival in amyotrophic lateral sclerosis. J Neurol Neurosurg Psychiatry. 2015;86:1180–5.
  • Chiò A, Hammond ER, Mora G, Bonito V, Filippini G. Development and evaluation of a clinical staging system for amyotrophic lateral sclerosis. J Neurol Neurosurg Psychiatry. 2015;86:38–44.
  • Roche JC, Rojas-Garcia R, Scott KM, Scotton W, Ellis CE, Burman R, et al. A proposed staging system for amyotrophic lateral sclerosis. Brain. 2012;135:847–52.
  • Balendra R, Jones A, Jivraj N, Knights C, Ellis CM, Burman R, et al. Estimating clinical stage of amyotrophic lateral sclerosis from the ALS Functional Rating Scale. Amyotroph Lateral Scler Frontotemporal Degener. 2014;15:279–84.
  • Fang T, Al Khleifat A, Stahl DR, Lazo La Torre C, Murphy C, Uk-Mnd Lical S, et al. Comparison of the King’s and MiToS staging systems for ALS. Amyotroph Lateral Scler Frontotemporal Degener. 2017;18:227–32.
  • Al-Chalabi A, Chiò A, Merrill C, Oster G, Bornheimer R, Agnese W, et al. Clinical staging in amyotrophic lateral sclerosis: analysis of Edaravone Study 19. J Neurol Neurosurg Psychiatry. 2021;92:165–71.
  • Andrews JA, Miller TM, Vijayakumar V, Stoltz R, James JK, Meng L, et al. CK-2127107 amplifies skeletal muscle response to nerve activation in humans. Muscle Nerve. 2018;57:729–34.
  • Shefner JM, Andrews JA, Genge A, Jackson C, Lechtzin N, Miller TM, et al. A phase 2, double-blind, randomized, dose-ranging trial of reldesemtiv in patients with ALS. Amyotroph Lateral Scler Frontotemporal Degener. 2021;22:287–99.
  • ClinicalTrials.gov. A study to evaluate the efficacy and safety of reldesemtiv in patients with amyotrophic lateral sclerosis (ALS) (COURAGE-ALS). https://clinicaltrials.gov/ct2/show/NCT04944784. Accessed March 29, 2022.
  • Dalla Bella E, Bersano E, Antonini G, Borghero G, Capasso M, Caponnetto C, et al. The unfolded protein response in amyotrophic later sclerosis: results of a phase 2 trial. Brain. 2021;144:2635–47.
  • Fang T, Al Khleifat A, Meurgey JH, Jones A, Leigh PN, Bensimon G, et al. Stage at which riluzole treatment prolongs survival in patients with amyotrophic lateral sclerosis: a retrospective analysis of data from a dose-ranging study. Lancet Neurol. 2018;17:416–22.
  • Kaplan EL, Meier P. Nonparametric estimation from incomplete observations. JAMA 1958;53:457–81.
  • Cox DR. Regression models and life‐tables. J R Stat Soc B. 1972;34:187–220.
  • Chiò A, Logroscino G, Hardiman O, Swingler R, Mitchell D, Beghi E, et al. Prognostic factors in ALS: a critical review. Amyotroph Lateral Scler. 2009;10:310–23.
  • U.S. Department of Health and Human Services, Food and Drug Administration, Center for Drug Evaluation and Research (CDER), and Center for Biologics Evaluation and Research (CBER). Amyotrophic lateral sclerosis: developing drugs for treatment guidance for industry. 2019. Available at: https://www.fda.gov/media/130964/download. Accessed November 10, 2022.
  • Edaravone (MCI-186) ALS 19 Study Group. Safety and efficacy of edaravone in well defined patients with amyotrophic lateral sclerosis: a randomised, double-blind, placebo-controlled trial. Lancet Neurol 2017;16:505–12.
  • ClinicalTrials.gov. Phase III trial of AMX0035 for amyotrophic lateral sclerosis treatment (Phoenix). https://clinicaltrials.gov/ct2/show/NCT05021536. Accessed July 21, 2022.