Deconstructing progression of amyotrophic lateral sclerosis in stages: a Markov modeling approach

References

• 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.
   PubMed Web of Science ®Google Scholar
• 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.
   PubMed Web of Science ®Google Scholar
• 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 Front Degener. 2017;18:227–32.
   PubMed Web of Science ®Google Scholar
• 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.
   PubMed Web of Science ®Google Scholar
• Bakker LA, Schröder CD, van Es MA, Westers P, Visser-Meily JMA, van den Berg LH. Assessment of the factorial validity and reliability of the ALSFRS-R: a revision of its measurement model. J Neurol. 2017;264:1413–20.
   PubMed Web of Science ®Google Scholar
• Franchignoni F, Mora G, Giordano A, Volanti P, Chiò A. Evidence of multidimensionality in the ALSFRS-R Scale: a critical appraisal on its measurement properties using Rasch analysis. J Neurol Neurosurg Psychiatry. 2013;84:1340–5.
   PubMed Web of Science ®Google Scholar
• Thakore NJ, Lapin BR, Pioro EP. Trajectories of impairment in amyotrophic lateral sclerosis: insights from the Pooled Resource Open-Access ALS Clinical Trials cohort. Muscle Nerve. 2018;57:937–45.
   PubMed Web of Science ®Google Scholar
• Atassi N, Berry J, Shui A, Zach N, Sherman A, Sinani E, et al. The PRO-ACT database: design, initial analyses, and predictive features. Neurology. 2014;83:1719–25.
   PubMed Web of Science ®Google Scholar
• 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 Front Degener. 2014;15:279–84.
   PubMed Web of Science ®Google Scholar
• Andersen PK, Keiding N. Multi-state models for event history analysis. Stat Methods Med Res. 2002;11:91–115.
   PubMed Web of Science ®Google Scholar
• Jackson C. Multi-state models for panel data: the msm package for R. J Stat Soft. 2011;38:1–29. Internet]. [cited 2018 May 17]. Available at: https://www.jstatsoft.org/article/view/v038i08
   Web of Science ®Google Scholar
• Gil J, Preux PM, Alioum A, Ketzoian C, Desport JC, Druet-Cabanac M, et al. Disease progression and survival in ALS: first multi-state model approach. Amyotroph Lateral Scler. 2007;8:224–9.
   PubMed Web of Science ®Google Scholar
• Sinaki M, Mulder DW. Rehabilitation techniques for patients with amyotrophic lateral sclerosis. Mayo Clin Proc. 1978;53:173–8.
   PubMed Web of Science ®Google Scholar
• Riviere M, Meininger V, Zeisser P, Munsat T. An analysis of extended survival in patients with amyotrophic lateral sclerosis treated with riluzole. Arch Neurol. 1998;55:526–8.
   PubMed Web of Science ®Google Scholar
• Abe K, Itoyama Y, Sobue G, Tsuji S, Aoki M, Doyu M, et al. Confirmatory double-blind, parallel-group, placebo-controlled study of efficacy and safety of edaravone (MCI-186) in amyotrophic lateral sclerosis patients. Amyotroph Lateral Scler Front Degener. 2014;15:610–7.
   PubMed Web of Science ®Google Scholar
• Jones AR, Jivraj N, Balendra R, Murphy C, Kelly J, Thornhill M, et al. Health utility decreases with increasing clinical stage in amyotrophic lateral sclerosis. Amyotroph Lateral Scler Front Degener. 2014;15:285–91.
   PubMed Web of Science ®Google Scholar
• Aalen OO, Farewell VT, De Angelis D, Day NE, Gill ON. A Markov model for HIV disease progression including the effect of HIV diagnosis and treatment: application to AIDS prediction in England and Wales. Statist Med. 1997;16:2191–210.
   PubMed Web of Science ®Google Scholar
• Tavakoli M, Malek M. The cost utility analysis of riluzole for the treatment of amyotrophic lateral sclerosis in the UK. J Neurol Sci. 2001;191:95–102.
   PubMed Web of Science ®Google Scholar
• Centre for Clinical Practice at NICE (UK). Appendix 9.7. Health economic evaluation. A cost-effectiveness analysis of non-invasive ventilation in motor neurone disease. [Internet]. National Institute for Health and Clinical Excellence (UK); (NICE Clinical Guidelines). Report No.: 105. 2010. Available at: https://www.ncbi.nlm.nih.gov/pubmedhealth/PMH0033020/. Accessed May 17, 2018.
   Google Scholar
• Watanabe H, Atsuta N, Nakamura R, Hirakawa A, Watanabe H, Ito M, et al. Factors affecting longitudinal functional decline and survival in amyotrophic lateral sclerosis patients. Amyotroph Lateral Scler Front Degener. 2015;16:230–6.
   PubMed Web of Science ®Google Scholar
• Westeneng HJ, Debray TPA, Visser AE, van Eijk RPA, Rooney JPK, Calvo A, et al. Prognosis for patients with amyotrophic lateral sclerosis: development and validation of a personalised prediction model. Lancet Neurol. 2018;17:423–33.
   PubMed Web of Science ®Google Scholar
• Balendra R, Jones A, Jivraj N, Steen IN, Young CA, Shaw PJ, et al. Use of clinical staging in amyotrophic lateral sclerosis for phase 3 clinical trials. J Neurol Neurosurg Psychiatry. 2015;86:45–9.
   PubMed Web of Science ®Google Scholar
• Faissol DM, Griffin PM, Swann JL. Bias in Markov models of disease. Math Biosci. 2009;220:143–56.
   PubMed Web of Science ®Google Scholar
• Titman AC, Sharples LD. Semi-Markov models with phase-type sojourn distributions. Biometrics. 2010;66:742–52.
   PubMed Web of Science ®Google Scholar
• Ravits JM, La Spada AR. ALS motor phenotype heterogeneity, focality, and spread: deconstructing motor neuron degeneration. Neurology. 2009;73:805–11.
   PubMed Web of Science ®Google Scholar
• 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.
   PubMed Web of Science ®Google Scholar
• Donohue MC, Gamst AC, Thomas RG, Xu R, Beckett L, Petersen RC, et al. The relative efficiency of time-to-threshold and rate of change in longitudinal data. Contemp Clin Trials. 2011;32:685–93.
   PubMed Web of Science ®Google Scholar
• Abe K, Aoki M, Tsuji S, Itoyama Y, Sobue G, Togo M, et al. 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.
   PubMed Web of Science ®Google Scholar